Unlocking Cell and Gene Therapies: Introducing VantagePoint™ – Lonrú Consulting’s Suite of Solutions For Growth
As cell and gene therapies (CGT) continue to revolutionize medicine, enabling technology companies are increasingly becoming a key pillar of progress. These companies develop critical platforms, tools, and technologies that facilitate the scalable, cost-effective, and precise production and delivery of groundbreaking treatments. At Lonrú Consulting, we understand that these enabling technologies are more than just support systems—they form part of the vital infrastructure required to democratize access to advanced therapies globally.
That’s why we’ve developed VantagePoint™, a comprehensive suite of solutions carefully crafted to propel enabling technology companies at every stage of their growth journey. VantagePoint™ encapsulates our deep industry expertise and advanced AI-driven analytics into a unified approach, uniquely positioning our clients to overcome challenges, identify opportunities, and accelerate commercialization within the dynamic CGT landscape.
Lonrú Consulting’s VantagePoint™: A Unified Suite of AI-powered Solutions Propelling Enabling Technology Companies at Every Stage of Growth.
The VantagePoint™ Advantage
VantagePoint™ addresses the fundamental areas critical to the growth and scalability of enabling technology providers:
VantagePoint™ Insights – Strategic market intelligence that provides real-time, actionable clarity, empowering your company to anticipate market trends, understand competitive landscapes, and make confident, informed decisions.
VantagePoint™ Validation – Strategic technology validation and partnering solutions, helping emerging technology providers navigate complexity with confidence, secure robust partnerships, and validate innovations strategically within the competitive CGT market.
VantagePoint™ Accelerate – Precision-driven go-to-market and business development strategies designed to fast-track commercialization, optimize financial planning, and accelerate market adoption, ensuring that innovative technologies reach the patients who need them, faster.
VantagePoint™ Illuminate – Tailored strategic positioning and messaging that clearly articulates your technology’s unique value, enhancing brand visibility, and effectively engaging stakeholders, from investors to industry partners.
VantagePoint™ Connect – Strategic stakeholder engagement services, leveraging deep market connections and relationship-building frameworks to align enabling technology companies with key translational networks, biotech firms, investors, patient advocates and industry leaders for impactful collaboration and accelerated growth.
VantagePoint™ Scale – Comprehensive commercial feasibility and strategic scaling support, delivering robust pricing strategies, detailed market entry roadmaps, and scalable growth frameworks that ensure sustainable, long-term commercial success.
Enabling Technologies: The Key to Democratizing Advanced Therapies
By optimizing the reach and placement of innovative enabling technologies, Lonrú Consulting helps to democratize the transformative potential of cell and gene therapies. These technologies significantly reduce costs, improve accessibility, and enhance treatment precision, ultimately allowing more patients across diverse geographic and economic contexts to benefit from cutting-edge treatments.
Lonrú Consulting: Your Partner in Transforming CGT
Lonrú Consulting was founded on the vision of illuminating opportunities within the CGT industry. With over 15 years of global experience in the life sciences sector, Lonrú Consulting, led by Dr. Caoimhe Nic An tSaoir, integrates scientific rigor with strategic acumen, uniquely positioning us to support enabling technology companies effectively.
We have specifically tailored our expertise to empower technology providers, understanding that their success directly translates into broader patient access to life-changing therapies. Through VantagePoint™, we provide clarity, strategic insight, and precise execution, driving your company's success while catalyzing the industry’s evolution.
At Lonrú, we believe the future of cell and gene therapy depends heavily on innovation in enabling technologies. Together, let’s illuminate the route to viable transformative therapies.
Let us illuminate your business—contact us to discover how the VantagePoint™ suite can accelerate your journey in cell and gene therapy.
Navigating Strategic Partnerships: How Platform Technology Providers Can Overcome Validation Challenges
At last week's Advanced Therapies UK meeting, one theme resonated clearly among platform technology companies supporting cell and gene therapy (CGT) production: the complex yet essential role of partnerships in validating their technologies. These strategic collaborations are critical, yet they often present significant hurdles, from scoping and initial engagement to execution.
Companies face multiple challenges when entering third-party partnerships. Firstly, the precise scoping of collaborations often proves difficult, leading to misaligned expectations and inefficient use of resources. Secondly, engaging suitable partners involves navigating complex negotiations and managing expectations around technology validation and intellectual property. Finally, executing these partnerships demands rigorous project management and clear, constant communication—areas where resource constraints frequently limit effectiveness.
Why are these partnerships vital? Successful validation through third-party collaborations provides crucial credibility, enhancing market trust and significantly boosting investor confidence. Moreover, strategic partnerships open doors to larger networks and new commercial opportunities, accelerating technology adoption and growth.
However, for many enabling technology companies, internal teams are deeply entrenched in R&D, fundraising efforts, or focused on sales execution and revenue generation, often leaving strategic partnership initiatives underserved. This is precisely where Lonrú Consulting steps in.
Launching Lonrú's VantagePoint™ Validation Platform: Illuminating strategic clarity for enabling technology companies in Cell & Gene Therapy.
At Lonrú, we specialize in illuminating critical strategic partnership needs. Our structured approach offers technology providers unparalleled visibility, strategic insight, and clarity when validating their enabling technologies. From clearly defined validation objectives and partner alignment to meticulous execution and impactful outcomes, our VantagePoint™ Validation platform ensures strategic clarity and precision in every partnership.
Explore more about our VantagePoint™ Validation platform and strategic partnership services here.
If you're an enabling technology company underpinning cell and gene therapy production or delivery, it's time to explore how strategic outsourcing can accelerate your journey. Let's illuminate your pathway to validation together.
Ready to discuss your strategic partnership needs? Connect with Lonrú Consulting today and let us help you turn your validation challenges into powerful opportunities.
Catalysing the COGs Revolution: The Role of Enabling Technologies in CGT
Lonrú is at Advanced Therapies UK this week and day one discussions highlighted emerging trends, groundbreaking innovations, and pressing challenges within the cell and gene therapy (CGT) landscape, placing a strong emphasis on enabling technologies and their critical role in addressing one of the industry's biggest hurdles—the Cost of Goods (COGs).
Enabling technology providers are emerging as key catalysts capable of significantly reducing COGs, addressing one of the industry's primary barriers to patient access. The keynote panel underscored how high manufacturing costs often prevent eligible patients from receiving approved therapies due to regional payer reluctance, emphasizing the urgent need for cost-effective solutions—precisely the type of innovation Lonrú champions.
A particularly compelling panel discussion, "Academic Translation: Advancing Cutting Edge Technologies," chaired by Lara Campana, from Resolution Therapeutics (a University of Edinburgh Spinout) delved deeper into the challenges faced by technology founders in establishing meaningful collaborations. When Lonrú posed the critical question, "Looking back, where do you see the biggest missed opportunities for enabling technologies or partnerships to accelerate development—and what would you do differently?" panelists candidly discussed the difficulties their teams encountered in demonstrating proof of concept of their technologies and managing complex industry collaborations, IP ownership, and partnership structuring.
These insights resonate strongly with Lonrú's expertise. We specialize in identifying, cultivating, and establishing strategic collaborations, effectively bridging the gap between academic innovation and commercial execution. Our tailored approach and advanced analytics help enabling technology providers navigate complexities, optimize commercialization pathways, and significantly lower COGs.
As the industry progresses, the imperative is clear: innovation must be paired with strategic partnerships to achieve tangible cost reductions and improve patient accessibility worldwide.
If you're ready to leverage your enabling technology for maximum impact, Lonrú is here to illuminate your path forward.
Illuminating CRISPR Clinical Trial Progress: A Lonrú Lens for Enabling Tools Providers
A recent Keystone meeting showcased a multitude of CRISPR-based clinical trials, underscoring the tremendous growth and development in the field. As excitement around CRISPR therapies intensifies, enabling tools providers—ranging from assay developers to manufacturing technology companies—are uniquely positioned to support these therapeutic programs in critical ways. In this post, we take a close look at some of the latest CRISPR clinical trial updates (therapeutic applications only) and introduce Lonrú Consulting’s new public dashboard designed to help enabling technology companies recognise emerging needs and opportunities.
CRISPR Therapeutics: What’s New from Keystone?
This year’s Precision Genome Engineering Keystone meeting confirmed that CRISPR-based therapies have made considerable strides, with an approved therapy now commercially launched and an expanded pool of proof-of-concept data and early clinical safety readouts for new indications. Key highlights include:
Emerging Pipeline Diversity
While oncology and monogenic hematological diseases continue to account for the majority of CRISPR clinical trials, diversity of clinical trial categories is expanding to include neurological disorders and metabolic diseases.
This broadening pipeline hints at a growing need for specialized vectors, delivery systems, and lab instrumentation that can facilitate gene editing across a wider range of cell types and patient populations.
Focus on Delivery and Efficiency
Many panels spotlighted the importance of refining both ex vivo and in vivo delivery modalities. This shift underscores the demand for robust viral and non-viral delivery tools, high-fidelity nucleases, and scalable manufacturing platforms—all areas where enabling technology providers can shine.
Quality and Regulatory Considerations
As CRISPR programs move through clinical phases, compliance with evolving global regulatory standards has become paramount. Tool providers offering GMP-ready reagents, automated QC processes, and validated assay kits stand to gain traction as sponsors seek to streamline their clinical workflows from pre-clinical development to clinical manufacture.
Why It Matters for Enabling Tools Providers
CRISPR sponsors rely on high-performance instrumentation, reagents, software, and manufacturing technologies to maintain clinical momentum. From single-cell analysis platforms that verify gene edits to advanced vector packaging systems, every stage of CRISPR therapy development demands specialized support.
Assay & Analytics
Tool developers can build or adapt products that precisely predict or measure gene-editing efficiency, off-target effects, and cell viability. Demand continues to grow for analytical solutions that can scale from early R&D to late-phase GMP settings.Automation & Scale-Up
As more candidates progress into mid- and late-stage trials, sponsors will need integrated, automated workflows—particularly for ex vivo engineering of immune cells and beyond. Vendors who provide modular, automated solutions can help sponsors reduce costs and improve reproducibility.Regulatory-Grade Toolkits
Enhanced guidelines around gene editing are emerging. GMP-certified reagents, standardized protocols, and robust documentation platforms offer sponsors peace of mind and help expedite regulatory approvals.
Introducing Lonrú’s CRISPR Clinical Trial Dashboard
In line with our mission to help technology providers identify strategic opportunities, Lonrú Consulting is releasing our new, publicly accessible CRISPR Clinical Trial Dashboard below and on our interactive dashboards page. This interactive database lets you:
Browse Active Sponsors and Investigational Targets
Instantly see which companies are leading in oncology, rare diseases, or other emerging applications—to map those areas back to their specialized tool needs.Filter by Phase, Status, and Indication
Determine where your tools and technologies can fit best based on trial phase (e.g., early-phase for novel assays vs. near-market for scalable manufacturing solutions).Pinpoint Potential Partnerships
Identify top CRISPR sponsors that may be seeking next-gen delivery methods, automation solutions, or advanced analytics to complement their therapeutic pipelines.
By consolidating and visualizing information on CRISPR therapeutic trials, we aim to give enabling tools providers an easy way to track potential leads, time new product launches, and tailor their solutions to fast-evolving sponsor needs.
How Enabling Tools Providers Can Use Lonrú’s Dashboards
Market Positioning
Understand which clinical areas (e.g., B-cell malignancies, sickle cell disease) are seeing the greatest CRISPR activity, and tailor your product roadmap or marketing accordingly.
Targeted Business Development
Pinpoint sponsors that match your capabilities—whether you offer reagents optimized for allogeneic cell therapies or automation equipment ideal for large-scale gene editing.
Competitive Benchmarking
Track your peers (other platform and tool vendors) to see how they are positioning their products across varying therapeutic trials. Use these insights to refine your messaging or to guide strategic investments in R&D.
Regulatory & Compliance Insights
Monitor how trials across different geographies progress through regulatory milestones. Align your quality management systems and documentation practices to meet sponsor expectations in each region.
Closing Reflections
Keystone illuminated just how quickly CRISPR therapeutics are evolving. For enabling tools providers, each new clinical milestone signals a rising need for more specialized, reliable, and scalable products. With sponsors juggling multiple priorities—speed, safety, cost efficiency, and regulatory compliance—there’s never been a better time to cement your role as a crucial partner in the CRISPR value chain.
Lonrú Consulting invites you to explore our dashboard and harness these data-driven insights. If you’d like to learn more about how we can help you position your technologies or amplify your market impact, we’re here to illuminate the path forward.
Ready to dive in?
Customize our CRISPR Clinical Trial Dashboard to uncover key trends and potential partnerships. Reach out at info@lonruconsulting.com or visit Lonrú Consulting to learn how our data analytics and strategic advisory can illuminate your next big opportunity.
At Lonrú Consulting, we believe in empowering the technologies behind the therapies. Together, let’s unlock the full potential of CRISPR and continue shaping the future of genomic medicine.
Bridging the Gap—Enabling Technology Providers & the Path to the Clinic
Lonrú is attending the Precision Genome Engineering: Translating the Human Genome to the Clinic meeting in Killarney this week. The meeting brings together the leading minds in gene editing, delivery systems, and therapeutic development to discuss the latest advancements shaping the future of precision medicine. From new CRISPR-based tools and enabling technologies to the challenges of translating innovation into the clinic, the conference highlights the critical steps needed to move from discovery to impact. Our poster presentation yesterday on streamlining preclinical-to-IND pathways generated interest, not just from therapeutic developers, but also from enabling technology providers eager to gain a clearer view of the challenges their partners and customers face in clinical translation.
Why Are Enabling Technology Providers Paying Attention?
While much of the conversation around gene editing focuses on therapeutic breakthroughs, every successful CRISPR-based therapy depends on the enabling technologies that support it—from delivery systems and genome engineering platforms to advanced analytical tools and scalable manufacturing solutions. Many enabling technology providers recognize that to best serve their customers, they need a deeper understanding of:
✔ Regulatory bottlenecks that delay clinical progression and impact product adoption.
✔ Common reasons for IND holds, including safety concerns, CMC issues, and assay validation gaps.
✔ Preclinical validation requirements that define what data therapeutic developers need to move forward.
By better aligning their solutions with these pain points, enabling technology providers can position themselves as indispensable partners in the journey from discovery to the clinic.
Beyond the Poster: Full Report & Decision Trees
Attendees we met with appreciated having access to our full report to dive deeper into the topics covered in the poster. In particular, the decision tree framework resonated with both therapeutic and technology developers, offering a structured approach to mitigating risks early in the development process.
For those who couldn’t attend the poster session—or who want to explore the findings in more detail—you can view and download the full report, poster, and decision trees here:
Lonrú’s full report, poster and decision tree framework are available for download.
The conversation doesn’t end here—if you’re an enabling technology provider looking to bridge the gap between research and clinical success, let’s connect. Lonrú Consulting specializes in strategic insights that help align enabling technologies with the needs of therapeutic developers, ensuring that innovation translates into impact.
Lonrú at Keystone: Precision Genome Engineering and the Future of CRISPR Clinical Translation
The upcoming Keystone Symposium on Precision Genome Engineering: Translating the Human Genome to the Clinic (March 3-6, 2025, in Killarney, Ireland) presents an exciting opportunity for the field of gene editing to converge on the critical scientific and regulatory advancements necessary for therapeutic translation. As a firm dedicated to empowering technology providers in the Cell and Gene Therapy (CGT) sector, Lonrú Consulting is eager to engage in discussions that address key industry challenges and pathways for accelerating CRISPR-based therapeutic approvals.
What We’re Looking Forward To
The Keystone agenda is packed with high-impact sessions, and several key themes stand out as particularly relevant for tool providers and regulatory strategists:
Regulatory Innovation and Harmonization to Expand Access to Precision Gene Therapies
The evolution of global regulatory frameworks remains one of the largest hurdles in transitioning CRISPR therapies from preclinical research to clinical implementation. We are particularly interested in sessions that discuss strategies to streamline Investigational New Drug (IND) and Clinical Trial Application (CTA) submissions, as we consider how tools providers may help therapeutic developers in this space.
From Target to IND: Preclinical Studies for ex vivo and in vivo Therapies
With the rise of in vivo genome-editing therapies alongside more established ex vivo approaches, ensuring safety and efficacy through robust preclinical models is paramount. Lonrú is keen to explore discussions on off-target risk mitigation, potency assays, and long-term safety assessments.
Genome Engineering in the Clinic: Advances in Therapeutic Applications
As CRISPR-based therapies move into human trials, the interplay between genome engineering technologies and clinical strategy becomes increasingly complex. We anticipate valuable insights into how industry leaders are addressing patient risk management, staggered dosing strategies, and immune response monitoring.
Lonrú’s Contribution: Bridging the Gap Between Preclinical and Clinical Translation
Lonrú’s poster evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays, we also introduce our decision tree framework, a tool to help developers navigate common pitfalls.
At Keystone, Lonrú will present our poster, “Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies.” This work evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays.
Our analysis emphasizes four strategic imperatives:
Early engagement with regulators to preemptively address potential concerns.
Enhanced off-target validation to minimize safety risks.
Optimized Chemistry, Manufacturing, and Controls (CMC) readiness to ensure scalable production.
Parallel global regulatory filings to accelerate approvals across different jurisdictions.
To support these strategies, we are unveiling our Decision-Tree Framework, a structured tool designed to help developers de-risk their preclinical-to-clinical transitions. This framework is segmented into three core areas:
Preclinical & Safety Considerations (off-target assessment, long-term safety studies, in vivo vs. ex vivo risk management)
CMC & Manufacturing Readiness (potency assay validation, lot-to-lot consistency, delivery system characterization)
Regulatory & Trial Design Strategy (harmonization of submission dossiers, risk mitigation planning, patient safety protocols)
Post-Conference Insights & Resources
Following the Keystone meeting, Lonrú will provide a comprehensive conference report, including key takeaways from the most impactful sessions and commentary on emerging industry trends.
Additionally, our Decision-Tree Framework and poster will be made available for download, offering a valuable resource for CRISPR developers seeking to refine their IND/CTA strategy.
Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies
We are thrilled to present our poster at Keystone, showcasing key insights into regulatory readiness, risk mitigation, and translational strategies. Proudly featuring insights from Lonrú Consulting and Johns Hopkins Carey Business School, our work highlights data-driven approaches to accelerating CRISPR-based therapeutic development.
For those attending Keystone, we welcome discussions on how Lonrú can support technology providers in navigating regulatory complexities and accelerating the path to clinic.
If you are interested in receiving any of the resources outlined here, please complete the form below and we’ll be in touch after the conference.
How the Focus of Cell and Gene Clinical Trials in the UK is Shifting
The UK remains a global hub for cell and gene therapy (CGT) innovation, but where is the field heading? What indications are seeing the most investment? How are sponsors adapting their pipelines?
At Lonrú Consulting, we apply data-driven insights to illuminate these questions, helping technology providers and innovators navigate the evolving CGT landscape. Our latest analysis of UK CGT clinical trials (2010–2024) highlights shifting therapeutic priorities, changing sponsor dynamics, and emerging areas of opportunity.
A Recent Decline in CGT Clinical Trials
While the UK has maintained strong CGT activity over the past decade, 2024 saw a decline in new trial initiations. A total of 26 new trials were initiated in 2024, compared to 31 in 2023, 32 in 2022, and 28 in 2021. This decline reflects several industry-wide challenges, including regulatory complexities that extend trial approval timelines, funding constraints that particularly affect early-stage biotech companies, and a strategic shift among sponsors toward refining pipelines post-pandemic.
Although this slowdown is worth monitoring, it may also indicate a shift in the field, with a move toward high-value, well-validated programs rather than a focus on sheer trial volume. Understanding how companies are adapting to this changing landscape is essential for stakeholders in the CGT ecosystem.
Explore Lonrú’s interactive dashboard examples to view UK CGT trial trends for further insights.
A Shifting Sponsor Landscape
The mix of sponsors in UK CGT trials is evolving. Large pharmaceutical companies, such as AbbVie and Novartis, are increasing their presence, signaling long-term investment in CGT. UK-based biotech firms remain active in driving first-in-human trials, though some, like Achilles Therapeutics, have recently pivoted away from clinical-stage programs. Academic institutions such as University College London (UCL) continue to play a critical role in early-stage innovation, particularly in rare diseases and regenerative medicine.
This diverse sponsor base reflects both the increasing commercial interest in CGT and the persistent role of academic centers in developing new approaches. The dynamic between large pharma, biotech startups, and research institutions will be key to shaping the next phase of clinical development.
The Evolution of Indications in CGT Trials
By categorizing CGT clinical trials into broader therapeutic areas, we can see shifts in industry focus.
Oncology remains the dominant area for CGT trials, but the landscape is evolving. Hematologic cancers, including multiple myeloma, leukemia, and lymphoma, continue to drive significant trial activity, largely fueled by CAR-T and other engineered cell therapies. Solid tumors, such as lung cancer, liver cancer, and glioblastoma, remain a major focus, though the pace of new trial initiation has been more gradual. Adoptive cell therapies, including tumor-infiltrating lymphocytes (TILs) and engineered T-cell receptors (TCRs), are emerging as alternatives to traditional CAR-T approaches, offering new avenues for immunotherapy.
Rare and genetic disorders have gained significant momentum in CGT research. Duchenne muscular dystrophy, cystic fibrosis, and spinal muscular atrophy have all seen increased trial activity in recent years. Many of these trials leverage gene therapy and gene editing approaches, reflecting industry confidence in in vivo genetic correction as a viable treatment strategy.
Neurological disorders are an emerging frontier for CGT. While historically underrepresented in CGT trials, conditions such as ALS, Parkinson’s disease, and Huntington’s disease are now receiving increased investment. Challenges remain in delivering therapies effectively across the blood-brain barrier, but clinical interest in gene therapies for neurodegenerative and neuromuscular conditions continues to rise.
Ophthalmology has consistently been a growth area in CGT. Retinal diseases, including retinitis pigmentosa and Leber’s congenital amaurosis, are among the top indications for AAV-based gene therapies. These conditions are well-suited for local gene delivery, reducing systemic risks while improving treatment precision.
Hematological conditions outside of cancer, such as sickle cell disease and hemophilia, continue to attract CGT investment. However, with recent regulatory approvals such as Casgevy for ex vivo gene editing in sickle cell disease, the number of new clinical trials in these areas may moderate over time as companies transition toward commercialization.
Strategic Takeaways for CGT Stakeholders
Understanding these trends is essential for stakeholders across the CGT ecosystem. Enabling technology providers, Biotech and pharmaceutical companies should align their pipeline strategies with the shift beyond oncology into neurology, ophthalmology, and rare diseases, where CGT applications are growing. Technology providers and contract development and manufacturing organizations (CDMOs) must adapt to an evolving landscape where scalable allogeneic cell therapies, in vivo gene editing, and AAV-based delivery are gaining prominence. Investors should look beyond oncology, as emerging CGT applications in neurological and metabolic disorders present new opportunities for value creation.
As the industry moves forward, the ability to navigate these macro trends will be critical for companies developing platforms and enabling technologies. Lonrú Consulting provides real-time data analytics, market intelligence, and commercialization strategies to help companies position themselves effectively in a competitive and evolving field.
The Critical Role of Academic Translational Centers in Advancing Cell and Gene Therapies
As the field of cell and gene therapy (CGT) matures, academic translational centers remain indispensable in driving innovation and bridging the gap between early research and clinical application. Recent efforts in the UK, Japan, Australia, Spain, the USA, Germany, and Ireland illustrate how dedicated translational frameworks, such as the RISE model, are shaping sustainable CGT ecosystems (Naresh et al., 2025). However, these efforts are now facing significant financial headwinds, in the United States, where drastic cuts to NIH indirect funding threaten to undercut decades of translational research momentum.
The RISE Framework: A Global Lens on CGT Translation
The RISE model (Resource sharing, Interdisciplinary collaboration, Sustainable financial models, and Education) underscores the importance of structuring academic environments to support the full translational pipeline of CGTs (Naresh et al., 2025). This approach is evident in:
United Kingdom: Institutions like UCL lead CGT innovation, leveraging partnerships with the Cell & Gene Therapy Catapult to scale new therapies. Lonrú’s interactive dashboard highlights how UK-based academic centers are leading in CGT trial sponsorship and development phases (Lonrú Consulting, 2025).
Japan: The PMDA's progressive regulatory framework, coupled with translational research centers, has propelled Japan to the forefront of regenerative medicine.
Germany and Spain: Public funding initiatives have strengthened hospital-integrated manufacturing capabilities, increasing trial accessibility.
Australia: Investments in non-viral vector platforms are accelerating local innovation.
Ireland: The recently proposed all-island CGT strategy signals the country’s commitment to fostering a unified research, clinical, and commercial environment (Lonrú Consulting, 2025).
Without vital support, translational momentum is at risk—academic labs remain the backbone of cell and gene therapy innovation, but funding challenges threaten to slow progress from discovery to patient impact.
Threats to Academic Research: The US Funding Crisis
While academic medical centers have historically been the foundation of CGT innovation, recent proposals in the US to cap indirect cost reimbursements represent a direct threat to translational research (NIH, 2025). These funds support vital infrastructure, including laboratories, safety measures, IT systems, and core personnel.
Johns Hopkins University, among others, has sounded the alarm (JHU, 2025):
“These abrupt and sweeping cuts in NIH funding pose an extraordinary challenge to the important and lifesaving work of our faculty, staff, and students. They jeopardize the longstanding and remarkable research partnership that was forged between the federal government and higher education at the conclusion of WWII and put at risk the future of the American research enterprise as a whole.”
The repercussions extend beyond academia—clinical trials reliant on NIH support, particularly in oncology, rare diseases, and regenerative medicine, now face an uncertain future. The loss of indirect funding could delay or halt up to 600 ongoing clinical trials at institutions like Johns Hopkins alone (JHU, 2025).
How Lonrú Supports Academic Translational Efforts
In an era where public funding faces unprecedented change, technology transfer offices (TTOs) must adapt. Lonrú Consulting provides strategic tools to enhance translational readiness in CGT, ensuring academic programs can efficiently navigate regulatory, funding, and commercialization hurdles.
Tools for Translational Readiness: AI-driven tools to de-risk preclinical to clinical translation for academic labs.
Market Positioning for Translational Centers: Interactive tools to identify funding opportunities and investor alignment, optimizing commercialization potential.
Sustainable Financial Models: Advisory support for securing diversified funding, including philanthropic partnerships, venture investments, and alternative grant strategies.
As the impact of policy changes over funding are realised, Lonrú remains committed to supporting the academic innovation ecosystem—helping institutions turn research into real-world therapies and ensuring that promising CGT advances do not languish in the 'Valley of Death.'
Final Thoughts: Translational Research as a Pillar of CGT Sustainability
As global governments and research institutions refine their CGT strategies, translational research must remain a priority. Whether through RISE-inspired frameworks or adaptive funding models, securing the future of CGT requires sustained investment and infrastructure support. Institutions, industry partners, and policymakers must work together to uphold the critical role of academic translational centers.
References
JHU. (2025). Our essential research partnership with the NIH. Office of the University President and Office of Johns Hopkins Medicine. Email communication
Lonrú Consulting. (2025, January 29). UK Cell and Gene Therapy Clinical Trials To Date. Interactive Dashboards. https://www.lonruconsulting.com/interactive-dashboards
Lonrú Consulting. (2025, February 5). Building an All-Island CGT Strategy: Ireland’s Opportunity to Deliver Cell and Gene Therapy. Lonrú's Lens. https://www.lonruconsulting.com/blog-1-1/building-an-all-island-cgt-strategy-irelands-opportunity-to-deliver-cell-and-gene-therapy
Naresh, N. U., Hammill, D., Kessel, M., Hajjar, R. J., & Yozwiak, N. (2025). As new cell and gene therapies emerge from academia, we must RISE to the opportunity. Nature Biotech, 43(January 2025), 143-146. https://www.nature.com/articles/s41587-024-02520-9
NIH. (2025, February 7). Supplemental Guidance to the 2024 NIH Grants Policy Statement: Indirect Cost Rates. Office of The Director, National Institutes of Health. https://grants.nih.gov/grants/guide/notice-files/NOT-OD-25-068.html
Building an All-Island CGT Strategy: Ireland’s Opportunity to Deliver Cell and Gene Therapy
Last week’s Cell and Gene Therapy in Ireland conference underscored a pivotal moment for the Irish life sciences sector. The key takeaway? Ireland is in a strong starting position to deliver an ambitious all-island CGT strategy, leveraging its existing infrastructure, research expertise, and growing investment landscape to build a sustainable, patient-centric ecosystem.
Why Now? Ireland’s Readiness to Deliver in CGT
With capital continuing to flow into advanced therapies, Ireland has a unique opportunity to integrate its North-South partnerships, its biopharma expertise, and its strategic geographic positioning to drive a cohesive CGT strategy that delivers for patients across the island and beyond.
Currently, Ireland’s cell and gene therapy (CGT) landscape is still in its early stages compared to established hubs like the UK, Japan, Spain, and the US. However, this presents an advantage: a modern, efficient system can be designed from the ground up that avoids legacy inefficiencies seen in other markets.
The IMPACT initiative, introduced by Professor Sakis Mantalaris during the conference and highlighted in the BPCI budget recommendation, proposes an integrated ecosystem that spans research, clinical application, manufacturing, and distribution. A key lesson from successful CGT hubs —such as Spain and Japan’s focus on non-viral engineering, and the UK’s predominant use of Lentivirus and AAV vectors—is the critical role of platform technologies in creating a sustainable, scalable ecosystem. As Ireland builds its CGT infrastructure, how essential will it be for stakeholders—including government bodies, academic institutions, biopharma companies, and clinical networks—to align around specific platform technologies to drive efficiencies, de-risk investment, and accelerate progress across the ecosystem?
What is the Strategy?
Ireland’s CGT strategy is outlined under IMPACT (Irish Medicines Centre for Personalised Advanced Cellular Therapeutics), which aims to:
Develop a coordinated research and clinical ecosystem spanning both Northern Ireland and the Republic.
Leverage Ireland’s existing strengths in biopharma manufacturing to accelerate CGT production at scale.
Expand clinical trial infrastructure to position Ireland as a leader in early-stage CGT research.
Enhance regulatory and market access frameworks to ensure patient-centric delivery.
These steps align with ensuring Ireland remains competitive in the global CGT space and delivering for patients across the island.
The Benefits of an Early-Stage, All-Island Strategy
While Ireland is entering the CGT ecosystem later than the UK and US, this early-stage positioning offers several advantages:
Strategic Design from the Start – Unlike legacy markets that must retrofit infrastructure, Ireland can build a modern, integrated CGT system from the ground up.
North-South Collaboration – A joint approach ensures access to talent, resources, and patient populations across the entire island, creating a single innovation network.
Geographic and Demographic Advantages – With a relatively small, yet diverse population, Ireland is well-suited for early-phase trials and real-world evidence generation.
Existing Biopharma Expertise – Ireland’s strong pharma sector can be leveraged to accelerate CGT manufacturing and attract further investment.
Learning from Global Success Stories
To understand how Ireland can scale CGT efficiently, it’s worth reflecting on how other nations have built their infrastructure:
The UK Model: Public-Private Alignment
The UK’s success in CGT was driven by:
Government-backed initiatives (e.g., the Cell & Gene Therapy Catapult).
Strong academic and hospital manufacturing networks.
Regulatory adaptability, positioning the UK as a leader in AAV/Lentivirus vector production.
Spain’s Early Investment in Public Health Infrastructure
Spain built regional CGT hubs integrated with public healthcare.
Strong clinical trial networks positioned it as a European leader in CAR-T therapies.
The US Approach: Venture-Backed Innovation
A high-risk, high-reward model enabled rapid CGT development.
Strong links between academia, startups, and biopharma companies fostered an ecosystem of continuous innovation.
Key Challenges for Ireland
Despite its strong starting position, Ireland must address critical barriers to ensure its CGT strategy delivers long-term impact:
Limited Clinical Trial Infrastructure – Investment in trial sites and patient access programs is needed.
High Cost of CGT Treatments – Reimbursement frameworks must be carefully designed to ensure equitable access.
Skills and Talent Gaps – More funding is needed for CGT-specific education and workforce training programs.
Regulatory Complexity – Ireland must establish a streamlined, innovation-friendly regulatory framework to attract CGT developers.
The Path Forward: Positioning Ireland as a CGT Leader
If Ireland successfully aligns its CGT strategy across research, clinical development, and manufacturing, it can position itself as a global leader in next-generation therapies.
What’s Next?
The momentum from the CGT in Ireland conference highlights a critical window for action. As Ireland advances its all-island CGT strategy, Lonrú Consulting will continue to support industry leaders, startups, and policymakers in navigating the complexity of CGT innovation.
Want to be part of the conversation? Get in touch to explore how we can collaborate in shaping Ireland’s future in CGT.
CGT Ecosystems: Ireland Has The Opportunity To Build From The Ground Up.
De-risking Cell and Gene Therapy Development: Global Regulatory Strategies for Streamlined Development
Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER), recently shared insights at the 2025 JP Morgan Healthcare Conference, outlining the FDA’s ongoing efforts to accelerate advances in cell and gene therapy (CGT). With significant breakthroughs in 2024—including approvals for tissue-engineered products, mesenchymal stem cell therapies, and solid tumor-targeting T-cells—the CGT sector continues to make significant progress. However, despite these advances, challenges in regulatory harmonization, market access, and cost-effectiveness persist, requiring strategic policy measures and industry collaboration to de-risk CGT development in the US and beyond.
The Current CGT Landscape in the US
The US remains a leader in CGT innovation, with the FDA approving 24 cell and gene therapies to date, and is predicted to review or receive applications for a record 15 new therapies in 2025.
Figure 1. Showing FDA-approved and pipeline cell and gene therapies.
Recognising the need for new approaches to regulating these innovative therapies, Dr. Peter Marks discussed several new initiatives underway at the FDA. Key initiatives include:
Risk-based regulatory frameworks: The FDA is exploring a new approach to human cells, tissues, and cellular-based products (HCTPs), addressing over- and under-regulation issues with a planned scientific workshop in February 2025.
Platform technologies like CRISPR: Marks emphasized the need for a regulatory shift, treating CRISPR as a platform tool rather than a product, which could streamline development and approval processes, potentially under the draft platform technology guidance.
Rare disease initiatives: Programs such as the Rare Disease Hub and collaborative efforts with the EMA are expected to accelerate treatment pathways for underserved conditions.
While these regulatory-focused initiatives are promising, ongoing efforts should also focus on reducing manufacturing bottlenecks, ensuring sustained funding for innovation, and fostering public-private partnerships to sustain momentum in the sector.
The UK Approach: A Strategic Roadmap for Growth
The UK’s CGT ecosystem is supported by institutions such as the Cell and Gene Therapy Catapult and the Advanced Therapy Treatment Centres (ATTC), which focus on scaling up manufacturing and integrating therapies into the National Health Service (NHS). Recent milestones include:
NHS adoption of CRISPR-based therapies: The approval of Casgevy for transfusion-dependent beta-thalassemia demonstrates the UK’s leadership in pioneering CGT adoption within public healthcare.
Regulatory flexibility: The Medicines and Healthcare products Regulatory Agency (MHRA) is actively harmonizing with global frameworks to enable smoother market entry.
By aligning with the UK’s emphasis on infrastructure and workforce development, other markets can strengthen their own pathways to commercialization.
Germany’s National Strategy: Balancing Innovation with Compliance
Germany is positioning itself as a European leader in CGT with the introduction of a National Strategy for Gene and Cell Therapies, launched in June 2024. This strategy focuses on:
Regulatory streamlining: Efforts to simplify legal pathways and align with the European Medicines Agency (EMA).
Investment in infrastructure: Enhancements to Germany’s biomanufacturing capabilities to ensure scalable production of CGT products.
Industry collaboration: Strong ties between government bodies and industry stakeholders to foster a competitive environment.
Other countries can learn from Germany’s comprehensive approach by expanding infrastructure investment and creating strategic alliances to enhance manufacturing scalability.
Japan’s Regulatory Flexibility: A Model for Rapid Market Access
Japan’s regulatory framework for regenerative medicine, governed by the Pharmaceuticals and Medical Devices Agency (PMDA), may represent one of the most progressive models globally. With a conditional and time-limited approval pathway, Japan has enabled rapid commercialization of therapies such as CAR-T cell treatments. Key elements of Japan's success include:
Accelerated approval pathways: Conditional approvals based on preliminary efficacy data, allowing early patient access.
Post-market surveillance: Rigorous follow-up requirements ensure long-term safety and efficacy.
Public-private collaboration: Strategic partnerships between government, academia, and industry have accelerated innovation.
To remain competitive, regulatory bodies in other regions could consider adopting Japan’s adaptive regulatory approach for certain high-priority therapies, thereby accelerating patient access without compromising safety.
Opportunities for Global Regulatory Synergy
Despite regional differences, there are clear opportunities to align regulatory processes across key markets:
Harmonized Regulatory Frameworks: The FDA, EMA, MHRA, and PMDA could collaborate on common standards for CGT approvals, reducing redundancies and facilitating global market entry.
Data-Sharing Initiatives: Establishing global databases, leveraging federated learning models to share real-world evidence can enhance regulatory decision-making and improve patient safety outcomes.
Streamlined Manufacturing Guidelines: Aligning quality and manufacturing standards globally can reduce costs and accelerate production scalability.
Stakeholders across the CGT ecosystem should work toward strengthening partnerships with international regulatory bodies to create a more unified approach to CGT approvals and commercialization.
A Call for Strategic Action
As CGT continues to revolutionize medicine, proactive steps are needed to de-risk development by:
Modernizing regulatory frameworks to accommodate platform technologies.
Increasing funding for infrastructure and workforce development.
Strengthening international regulatory cooperation to facilitate global market access.
Addressing cost barriers to ensure broad patient access.
By drawing from strategies across the US, the UK, Germany, and Japan, the global CGT sector can create a more resilient and competitive landscape, ensuring that transformative therapies reach patients faster and more efficiently.
Want to stay ahead in cell and gene therapy development? Explore Lonrú’s strategic solutions.
From Semiconductors to Genes: Engineering Precision in Advanced Therapies
Exploring insights from ARM's JPM 2025 panel discussion and the evolving quest for molecular-level understanding in cell and gene therapy.
Background
At this year’s JPMorgan Healthcare Conference, the ARM panel discussion on "Capital Markets and Commercial Insights: Navigating Opportunities and Challenges in CGTs" brought an unexpected but interesting comparison to light. Keith Crandall of Arch Ventures, drawing from his semiconductor and photonics background, made a compelling case for how the biotech industry—specifically cell and gene therapy (CGT)—can learn from the rigor and precision that revolutionized semiconductor manufacturing.
Crandall noted that in semiconductor production, every molecule is quality-checked, and processes are controlled down to the quantum level. In contrast, CGT manufacturing still grapples with variability and a limited characterization of biological components at a molecular level. According to Crandall, the key to unlocking CGT’s full potential lies in adopting semiconductor-level precision and embedding quality control (QC) from the outset—not as an afterthought.
The Semiconductor Blueprint: A Roadmap for CGT
The semiconductor industry underwent significant transformation from its early, experimental days to become one of the most exacting fields in modern manufacturing. The drivers of this shift included:
Standardisation and Process Control: Introduction of Statistical Process Control (SPC), Six Sigma, and rigorous certification processes (Anonyuo et al., 2024).
Deep Foundational Understanding: Mastery over materials and physics that enabled predictable, repeatable outcomes.
Leadership Evolution: Semiconductor firms appointed leaders with strong manufacturing backgrounds, shifting from leaders with academic experience.
Collaborative Ecosystems: Industry-wide knowledge-sharing through consortia like SEMATECH helped accelerate improvements across the board (Irwin & Klenow, 1996).
Precision at the Molecular Level: Lessons from Semiconductors for the Future of Cell and Gene Therapy
CGT: A Field in Search of Precision
Despite the substantial advancements in CGT, significant hurdles remain, particularly in manufacturing scalability and consistency. The inherent complexity of biological systems introduces unpredictable variances—an issue that semiconductor methodologies could help address.
Key Areas for CGT Innovation Inspired by Semiconductors
Molecular-Level Understanding
Advances in single-cell analysis and multi-omics technologies are helping bridge the knowledge gap in CGT. Spain, for example, has a network of research groups conducting single-cell RNA sequencing pre- and post-CAR-T cell therapy to better understand therapeutic persistence and variability (Rodriguez-Madoz et al., 2023). Similar efforts are emerging globally, including:MIT’s Center for Biomedical Innovation (USA): Exploring process control and predictive analytics in cell therapy manufacturing.
Fraunhofer Institute (Germany): Investigating automation and in-line analytics for bio-manufacturing.
Cell and Gene Therapy Catapult (UK): Driving standardisation and scalable process development across the industry.
Standardised QC Protocols
The standardisation of QC protocols in ATMP manufacturing is evolving through the adoption of real-time process controls, automation, and the promise of harmonised regulatory frameworks from bodies like the FDA, EMA, and PMDA.Automation and Closed Systems
Leveraging automation and robotics to minimise human intervention in CGT manufacturing aligns closely with semiconductor principles. Companies such as Ori Biotech and Autolomous are pioneering efforts in digital manufacturing platforms tailored for advanced therapies.Leadership Evolution
As CGT companies advance, there is a growing recognition of the need for leadership with expertise in industrial-scale manufacturing. Bridging the gap between clinical and operational expertise could unlock significant efficiencies and scalability.
The Future Outlook: Unlocking Precision in CGT Manufacturing
Crandall’s perspective underscores that precision in biological manufacturing will do more than just improve yields and consistency—it could fundamentally change how we approach cell engineering and advanced therapeutic manufacturing. By minimising variability and embedding precision from the outset, CGT manufacturers could realise therapies that are safer, more predictable, and ultimately more accessible to patients worldwide.
Conclusion
The intersection of semiconductor precision and CGT innovation presents an exciting frontier. While biology’s inherent complexity presents challenges, adopting rigorous QC frameworks and fostering cross-industry collaboration can accelerate CGT’s trajectory from bespoke treatments to widely available, cost-effective therapies.
Lonrú Consulting continues to explore and support these transformational shifts—illuminating the opportunities for CGT companies looking to navigate complexity with precision and clarity.
References
Anonyuo, S., Kwakye, J., & Ozowe, W. (2024, November 22). A review of quality control and process optimization in high-volume semiconductor manufacturing. World Journal of Engineering and Technology Research. https://zealjournals.com/wjetr/content/review-quality-control-and-process-optimization-high-volume-semiconductor-manufacturing
Irwin, D., & Klenow, P. (1996, November 12). Sematech: Purpose and Performance. Proceedings of the National Academy of Sciences. https://www.pnas.org/doi/full/10.1073/pnas.93.23.12739
Rodriguez-Madoz, J. R., Prosper, F., & et al. (2023, November 2). Sequential Scmultiomics of In Vivo CAR-T Cells Allows Characterization of Transcriptional Differences between Patients, and Identifies IL10 As a Potential Mechanism of Resistance to CAR-T Cells in MM. Blood, 142(Supplement 1), 3433. https://ashpublications.org/blood/article/142/Supplement%201/3433/499052/Sequential-Scmultiomics-of-In-Vivo-CAR-T-Cells
Illuminating the Future of Cell and Gene Therapies
Key Insights and Lonrú’s Role in Driving Ecosystem Collaboration
Bridging the Gaps in the Evolving CGT Landscape
The cell and gene therapy (CGT) industry is experiencing transformative growth, but with this progress comes a host of challenges. Insights shared during ARM’s State of the Industry Capital Markets and Commercial Insights panel revealed that success in CGT hinges on addressing key obstacles such as regulatory complexity, stakeholder engagement, and ecosystem collaboration. These challenges, if unaddressed, could hinder the sector’s ability to deliver groundbreaking therapies to patients worldwide.
ARM’s State of the Industry Briefing: Panelists discussing capital markets and commercial insights.
Key Themes and Challenges in CGT
Regulatory Complexity Across Regions
The CGT sector operates within highly regulated environments, with frameworks varying between regions like the U.S., Europe, and Asia. Panelists stressed the need for tools that help companies adapt their regulatory strategies to meet jurisdiction-specific requirements efficiently. In an earlier session, the FDA’s Dr. Peter Marks underscored early efforts to align regulatory requirements across geographies, aiming to reduce the burden on sponsors. We’ll explore this topic in detail in an upcoming blog.Ecosystem Collaboration: A Critical Imperative
The panel emphasised that the CGT industry’s growth depends on strong collaboration among biotechs, academic institutions, investors, and regulators. However, many companies struggle to create these connections due to siloed approaches or timing of communication strategy, many leaving it late in the development process.Stakeholder Engagement and Communication
Effective stakeholder engagement remains a top challenge, with companies needing to tailor their messages to resonate with specific audiences—investors, regulators, patients, or clinicians. A one-size-fits-all approach simply does not work in such a diverse ecosystem.Data Integration for Better Decision-Making
Companies face the challenge of integrating data, ranging from clinical trial results to real-world patient outcomes. Transforming this data into actionable insights that drive strategic decisions is crucial but often underdeveloped.Speed-to-Market and Competitive Pressure
The CGT sector needs streamlined operations and fast time-to-market. Key challenges include identifying eligible patients, fostering trust through early communication, establishing accessible treatment centers, and aligning stakeholders. Addressing these challenges is critical for successful product launches.
The panel also surfaced intriguing perspectives that warrant deeper exploration. Keith Crandell of ARCH Venture Partners drew parallels between the CGT industry and the semiconductor sector, suggesting that lessons from semiconductors’ QC’ing and standardisation could shape CGT’s future success. Click here for a blog post diving into this concept.
Lonrú Consulting: Enabling Collaboration and Tailored Engagement
At Lonrú Consulting, we understand that thriving in the CGT sector requires more than cutting-edge therapies—it demands a collaborative, data-driven approach to overcoming industry challenges. Here’s how we help address these key themes:
Driving Ecosystem Collaboration with Tailored Solutions
Lonrú’s AI/ML-powered solutions facilitate collaboration by providing frameworks that align stakeholders, streamline workflows, and foster co-creation. Our interactive dashboards offer a centralized view of shared objectives, enabling seamless partnerships between biotechs, academic institutions, and investors.
Enhancing Stakeholder Engagement
Lonrú specializes in crafting stakeholder-specific messaging, ensuring that communication resonates with diverse audiences. Whether it’s presenting data to regulators, showcasing ROI to investors, or educating clinicians, our solutions enable companies to deliver personalized, impactful narratives.
Turning Data into Actionable Insights
Our advanced data analytics solutions can be deployed to integrate clinical, market, and patient datasets to provide clear, actionable insights. This supports informed decision-making, helping clients navigate regulatory landscapes, anticipate market trends, and optimize therapeutic strategies.
Accelerating Speed-to-Market
By owning resource-intensive tasks such as market development research, product development strategy, and marketing collateral creation, Lonrú reduces bottlenecks in the development process. This allows companies to move faster without sacrificing quality or compliance.
Empowering Precision Communication
With Lonrú’s tailored AI-driven content generation, companies can create high-impact materials—from investor decks to engaging podcasts—quickly, effectively and without compromising on quality. This ensures that stakeholders receive the right message, at the right time, through the right channels.
Lonrú’s Vision: Illuminating Opportunities For Innovation
Lonrú Consulting is poised to empower CGT organisations by simplifying complexity, fostering collaboration, and enhancing communication. By leveraging advanced AI/ML solutions and industry expertise, we help clients build strong ecosystems, engage stakeholders meaningfully, and bring transformative therapies to market faster.
Ready to Illuminate Your Path?
Let Lonrú Consulting help you navigate the intricacies of CGT with tailored, innovative solutions. Contact us today to learn more about how we can accelerate your journey to success.
Charting the Trajectory of Cell and Gene Therapy from 2024 to 2025
What to Expect from the 2025 State of the Industry Briefing
This morning at Lonrú we’re looking back on 2024, and sharing our take on what to expect from today’s discussions at the Alliance for Regenerative Medicine’s (ARM) 2025 State of the Industry briefing.
The past year was marked by significant progress, including advancements in in vivo therapies, global regulatory milestones, and an ongoing push to address affordability and accessibility in cell and gene therapy (CGT). As the industry continues to mature, we anticipate that today’s briefing will highlight key inflection points and lay out the challenges and opportunities for the year ahead.
From in vivo innovations and manufacturing breakthroughs to expanded therapeutic targets and global market shifts, the CGT sector is poised for another transformative year. Here’s our take on what shaped 2024 and what may define the future.
2024: A Year of Momentum and Milestones
Breakthroughs Becoming the Norm
2024 began with optimism following a stormy 2023 in capital markets. Despite economic volatility throughout 2024, the CGT sector achieved major milestones: nine new therapies were approved in the U.S., and two in Europe. The FDA’s approval of two sickle cell gene therapies in late 2023 further underscored the field’s transformative potential. These developments heralded a “new normal” where groundbreaking therapies routinely became available to patients (ARM, 2024).
Advancing Cell Therapy
By February, the industry spotlight shifted toward engineered cell therapies. The approval of tumour-infiltrating lymphocyte (TIL) therapy for solid tumours represented a watershed moment, while investment in autoimmune applications highlighted the expanding scope of cell therapies beyond oncology (ARM, 2024).
Reframing the Value Narrative
August saw renewed focus on the affordability and accessibility of CGT. Frameworks like “Gene Therapy for Patients and Society” (GPS) and “CAR-T for Patients and Society” (CAR-PS) were developed to demonstrate the long-term value and cost-effectiveness of these therapies. Such efforts aimed to reshape public perception and healthcare decision-making (CMS, 2024).
New Gene Editing Frontiers
December capped the year with approvals for two adoptive cell therapies targeting solid tumours and the introduction of next-generation gene editing techniques like prime and epigenetic editing in clinical trials. These developments opened new possibilities for tackling previously untreatable conditions (ARM, 2024).
In Vivo Growth: A Rising Star
While much of 2024’s focus was on ex vivo therapies, in vivo approaches gained significant traction. These methods, which deliver genetic modifications directly within the patient’s body, promise to enhance precision and scalability. Key developments included:
CRISPR-Based In Vivo Therapies: Clinical trials demonstrated promising results in diseases like Duchenne muscular dystrophy and retinal disorders. These breakthroughs highlight the growing maturity of in vivo technologies (ARM, 2024).
Next-Generation Gene Editing: Techniques such as prime* and epigenetic editing, which offer higher precision and reduced off-target effects, entered early clinical stages (Tune Therapeutics, 2024) (O'Hanlon Cohrt, 2024).
Innovative Delivery Mechanisms: Advances in nanoparticle and viral vector technologies further improved the efficiency and safety of in vivo therapy delivery (ARM, 2024).
Looking ahead, in vivo growth will likely feature prominently in discussions about the next wave of CGT innovations, particularly in areas like systemic genetic disorders and regenerative medicine.
Predictions for 2025
Based on 2024’s trajectory, today’s State of the Industry briefing may shine a light on several critical themes:
1. Regulatory and Manufacturing Innovations
With the FDA’s expanded Office of Therapeutic Products, efforts to harmonise global regulatory frameworks and streamline manufacturing processes will take centre stage.
2. Expanding Therapeutic Horizons
Expect updates on the broadening range of disease targets, including autoimmune conditions and rare disorders, as clinical breakthroughs continue to multiply.
3. Addressing Affordability and Access
ARM’s ongoing advocacy for outcomes-based agreements and cost-efficiency frameworks will remain a focal point as the industry works to balance innovation with equitable patient access.
4. Continued In Vivo Innovation
Advancements in delivery systems and new data from clinical trials of in vivo therapies will likely feature heavily, positioning this approach as a cornerstone of future CGT strategies.
5. Ecosystem Collaboration
Fostering partnerships and collaboration across academia, biopharma, and regulatory bodies will underpin sustainable CGT growth.
Illuminating the Path Forward
2024 demonstrated that the CGT sector is no longer in its infancy. Breakthroughs are expected, and the focus has shifted toward refining processes, improving access, and expanding therapeutic potential. As 2025 unfolds, Lonrú Consulting will continue to illuminate the opportunities for our clients, providing data-driven insights and tailored strategies to help them navigate this complex and dynamic landscape.
*Prime Medicine’s clinical program PM359 is an ex-vivo engineered HSC product (O'Hanlon Cohrt, 2024). Prime editing technology holds promise as an in-vivo engineering platform (Newby & Liu, 2021).
References
ARM. (2024, January 8). Introduction and Industry Update - Cell & Gene State of the Industry Briefing 2024. YouTube. Retrieved January 13, 2025, from https://www.youtube.com/watch?v=8uyekVKDo8Y
ARM. (2024, April 30). SECTOR SNAPSHOT ADVANCES IN ENGINEERED CELL THERAPY. ARM - Industry updates. https://alliancerm.org/wp-content/uploads/2024/05/Sector-Snapshot-4.30.2024.pdf
ARM. (2024, December 30). Alliance for Regenerative Medicine - December 2024 Sector Snapshot. ARM Industry Updates. https://alliancerm.org/wp-content/uploads/2025/01/20250107-2024-Sector-Snapshot.pdf
CMS. (2024, March 7). Cell and Gene Therapy (CGT) Access Model. enters for Medicare & Medicaid Services. Retrieved January 13, 2025, from https://www.cms.gov/priorities/innovation/innovation-models/cgt
Newby, G., & Liu, D. (2021, November 1). In vivo somatic cell base editing and prime editing. Molecular Therapy, 29(11), 3107-3124.
O'Hanlon Cohrt, K. (2024, May 1). The FDA Has Cleared the First Clinical Trial Application for a Prime Editor. CRISPR Medicine News. https://crisprmedicinenews.com/news/the-fda-has-cleared-the-first-clinical-trial-application-for-a-prime-editor/
Tune Therapeutics. (2024, November 14). Tune Therapeutics Moves into Clinical Spotlight with TUNE-401: A First-in-Class Epigenetic Silencer for Hepatitis B. Investors and Media. https://tunetx.com/tune-therapeutics-moves-into-clinical-spotlight-with-tune-401-a-first-in-class-epigenetic-silencer-for-hepatitis-b/
Introducing Lonrú Consulting
Navigating Complexity
Illuminating Innovation
Empowering the Future of Cell and Gene Therapy
The cell and gene therapy (CGT) sector is experiencing unprecedented growth, diversification, and challenges (McKinsey & Co., 2022). At Lonrú Consulting, we’re here to help organisations navigate this complex landscape by providing tailored solutions that empower innovation, optimise efficiency, and drive impactful results.
Our Mission
Lonrú Consulting is dedicated to delivering data-driven insights and actionable strategies that address the unique challenges of the CGT industry. Through collaboration, advanced tools, and domain expertise, we guide clients toward sustainable growth and innovation in a highly competitive market.
Why Now for AI/ML?
The rapid evolution of AI/ML tools over the last 18 months has transformed how organisations address critical challenges across many industries. The wider adoption of AI/ML in the life sciences field has triggered both excitement and caution (Nature, 2023). On one hand, these tools promise to revolutionise processes such as drug discovery, regulatory navigation, and patient engagement (Hutson 2024). On the other hand, perceptions of these tools as “black boxes” with potential accuracy and reliability issues have made some organisations hesitant to fully commit (McKinsey & Co., 2023). Despite these concerns, the tide is shifting as the technology continues to improve and demonstrate measurable impact.
Perceptions in Life Sciences
Early adopters of AI/ML in life sciences report a 30% improvement in engagement and satisfaction scores when using AI/ML powered tools for content personalisation and insight generation. Many life sciences leaders emphasise the importance of clear AI strategies, with companies adopting customised solutions seeing twice the impact compared to those using off-the-shelf models (McKinsey & Co., 2023). However, challenges such as data privacy, algorithmic transparency, and the potential for misinformation (e.g., hallucinations in outputs) continue to shape perceptions (Nature Editorial, 2024).
Building Confidence in AI for CGT
Transparency and customisation are critical to unlocking the full potential of AI in life sciences. Companies with defined AI strategies report twice the impact compared to those without (McKinsey & Co. 2023). At Lonrú, we ensure that every AI-powered solution is tailored to meet our clients’ specific goals, fostering trust and maximizing outcomes.
How Lonrú Can Help
Our AI/ML-driven solutions evolve with client needs, ensuring relevance in the fast-paced CGT landscape. By blending cutting-edge technology with domain expertise, we address industry-specific challenges and enable our clients to:
Analyze Data Effectively: Transform complex datasets into actionable insights.
Optimize Workflows: Eliminate inefficiencies and boost productivity.
Facilitate Collaboration: Connect biotechs, academia, and investors to foster innovation and growth.
In an industry where speed, precision, and trust are paramount, Lonrú equips clients to navigate complexity confidently, seize emerging opportunities, and drive impactful innovation.
Stay updated on our capabilities and projects by connecting with Lonrú Consulting on LinkedIn using the link below.
References
Hutson, M. (2024, March 14th). Cutting to the chase. Health sciences. https://media.nature.com/original/magazine-assets/d41586-024-00753-x/d41586-024-00753-x.pdf
McKinsey & Co. (2022, November 16th). How AI can accelerate R&D for cell and gene therapies. Life Sciences. https://www.mckinsey.com/industries/life-sciences/our-insights/how-ai-can-accelerate-r-and-d-for-cell-and-gene-therapies
McKinsey & Co. (2023, May 6th). Early adoption of generative AI in commercial life sciences. McKinsey Direct. https://www.mckinsey.com/industries/life-sciences/our-insights/early-adoption-of-generative-AI-in-commercial-life-sciences#/
Nature. (2023, December 6). Science and the new age of AI. Immersive. https://www.nature.com/immersive/d41586-023-03017-2