Cell & gene therapy R&D trends and breakthrough innovations
This report identifies the latest trends in cell and gene therapy R&D, surfacing the top assets in development at research institutes and biotech companies around the world.
The trends and breakthroughs in the report are identified by analyzing the engagement of industry teams (BD, S&E, Open Innovation) using our online partnering platform to identify their next scientific partners.
The insights should provide scientific decision-makers with a roadmap of high-impact opportunities in an evolving and competitive landscape, pinpointing emerging technologies and potential partners to advance cell and gene therapy R&D.
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Key challenges for CGT R&D
Cell and gene therapies have come a long way since their inception thirty years ago and are now one of the key frontiers of medical research. In, 2017 the FDA approved Kymriah, the first CAR-T cell therapy, and Luxturna, the first directly administered gene therapy. Since then, the pipeline of cell & gene therapies has expanded rapidly, with hundreds of candidates in development across oncology, rare disease, cardiovascular, ophthalmology, and beyond.
Both approaches hold promise to treat previously untreated diseases, and there has been a string of successes in recent years. These include approval of the first CRISPR-based gene therapies to treat sickle cell disease, and the continued deployment of CAR-T therapy in cancer treatments.
Although both cell and gene therapies have broad potential, their full medical and commercial promise has yet to be fully realised. The field is still maturing, with many therapies still in clinical trials. But as new technologies emerge, improving therapeutic precision, overcoming delivery challenges, and addressing regulatory and manufacturing hurdles are all being advanced.
R&D Challenge #1
Gaining ground in an emerging modality
While interest from industry in cell and gene therapies is high, no single player has mastered the space. This means there isn’t a tried and tested recipe for overcoming challenges with delivery, scalability, and long-term safety. Advancements in CRISPR, stem cell engineering, and viral vectors are promising, but innovation and investment are crucial to for commercialization and the benefits to be felt more widely.
R&D Challenge #2
The complexities of working with living entities
Biological entities are highly complex and extremely delicate, making their manipulation challenging for R&D. Their variability and sometimes unpredictable dynamics can lead to issues with consistency and scalability. Patient compatibility can also be challenging, with the possibility of adverse immune responses. Despite these challenges, working with live entities that have fine-tuned their function through evolution is also what makes cell and gene therapies so promising.
R&D Challenge #3
Delivery technologies
Efficiently and precisely delivering genetic material or engineered cells to the right tissues and organs remains a scientific and technical hurdle, especially for in vivo therapies. For that reason, we have chosen to include enabling and delivery technologies in this report.
Top Cell and Gene Therapy innovations
Review the highest performing cell and gene therapy technologies and assets from our online partnering platform.
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1. Regenerating therapeutic T cells in vivo
Chimeric antigen receptor (CAR) T-cell therapies have revolutionized the treatment of cancer, but their limited lifespan post-infusion poses a challenge and patients require multiple rounds of therapy to maintain remission. The ideal solution would be a self-renewing source of CAR-expressing T cells, generated within a patient.
Researchers at the City University of New York have developed a compact gene control system, derived from the TCR-alpha locus control region (LCR), directing therapeutic gene expression specifically to T cells. These “mini-LCR” cassettes are small enough to fit into standard gene transfer vectors and maintain full regulatory function, ensuring consistent and safe expression only in engineered T cells. This enables long-term, in vivo production of therapeutic T cells from stem cell transplants, improving the durability and safety of cancer immunotherapies.
2. Expressing gene therapies for Alzheimer’s through the brain
A major challenge in developing gene therapies for neurodegenerative diseases is that the therapeutic effects are confined to the injection site. This constraint is especially problematic for diseases such as Alzheimer’s, where pathological changes are widespread and diffuse.
Work from scientists at Western University introduces an AAV-based gene therapy vector that enables the expressed therapeutic protein to spread through the brain. By fusing the protein with a cell-penetrating peptide and engineering it for direct nuclear uptake, the therapy achieves widespread delivery to both transduced and neighbouring cells. Early preclinical models show near-complete clearance of beta-amyloid plaques, suggesting a potent new strategy for Alzheimer’s and other brain-wide disorders.
3. Incorporating tertiary design into mRNA therapeutics
Despite their merits, mRNA therapeutics and vaccines still face persistent challenges with molecular instability and inefficient protein translation. Current RNA design tools improve structural stability using secondary structure folding predictions but fall short in optimizing the more complex tertiary configurations that influence RNA performance in the body.
Researchers working at Houston Methodist Research Institute have developed what they call ‘Translatable RNA Origami’; the first mRNA design method incorporating tertiary structure. Their computer-aided approach yields RNA molecules that resist degradation and produce higher protein levels compared to existing tools such as LinearDesign. The result is a versatile platform for engineering more stable, potent RNA therapies and vaccines with broad applications across infectious disease, cancer, and rare genetic disorders.
4. Precision targeting for glioblastoma
Glioblastoma is notoriously resistant to treatment, largely due to the creation of a suppressive tumor microenvironment dominated by regulatory T cells (Tregs) and evasive immune pathways. Efforts to target the glioblastoma tumors directly with CAR T therapies have struggled with limited specificity and off-target toxicity, especially in the sensitive context of the brain.
Scientists at the Medical University of South Carolina have developed anti-GARP CAR T cells that target free, unbound GARP present on tumor and immune-suppressive cells in glioblastoma, but absent on healthy platelets. This design enables precise tumor and Treg targeting, reshaping the immune landscape to sustain anti-tumor activity without systemic toxicity. The approach holds promise not only for glioblastoma, but for other solid and hematologic cancers where GARP expression plays a role.
5. Reducing off-target effects with LNP delivery of mRNA therapies
Lipid nanoparticles (LNPs) used for mRNA therapies tend to accumulate in off-target organs like the liver and spleen, limiting safety and requiring high doses that often exceed tolerable limits. Moreover, conventional targeting strategies suffer from poor efficiency and high manufacturing complexity, constraining their broader therapeutic use.
Scientists at Monash University spinout, NovaRNA, have developed a next-generation LNP platform that enables the precise, tuneable delivery of mRNA using nanobody conjugation. Their system enhances targeting efficiency (>90%) and incorporates a bivalent design requiring dual antigen recognition, which dramatically reduces off-target effects. With improved safety, lower dosing requirements, and scalable manufacturing, the platform is primed for preclinical applications in oncology and autoimmune diseases.
6. A new alternative to the viral delivery of nucleic acids
Nucleic acids for gene therapy or vaccination are easily degraded and difficult to transport to target cells. While viral vectors offer one solution, they bring safety and scalability concerns, prompting the search for more stable, non-viral delivery platforms.
Researchers at the University of Victoria have developed a polymeric micelle with a hydrophobic core that securely encapsulates nucleic acids using a two-step block copolymer assembly. This design forms a dense, protective structure around the genetic payload, finished with a hydrophilic PEG shell to aid circulation and uptake. Created using microfluidics, the system offers a tuneable, non-toxic delivery method suitable for gene therapies, vaccines, and other nucleic acid-based treatments.
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