Around the Helix: Cell and Gene Therapy Company Updates – October 22, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. Patients Treated With Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Maintain Clinical Benefit for Up to 2 Years

Newly updated data from Spur Therapeutics’ phase 1/2 GALILEO-1 clinical trial (NCT05324943), which is evaluating the adeno-associated virus (AAV) vector-based gene therapy avigbagene parvec (also known as FLT201) for the treatment of Gaucher disease, has shown that patients who received the gene therapy have maintained clinical benefit for up to 2 years post treatment.

2. Sarepta Therapeutics’ Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Produces “Robust Expression” of β-SG in Phase 3 Trial

Sarepta Therapeutics’ SRP-9003 (bidridistrogene xeboparvovec), an investigational AAV vector-based gene therapy intended to treat limb-girdle muscular dystrophy Type 2E (LGMD2E/R4, also known as beta sarcoglycanopathy), has demonstrated the ability to produce “robust expression” of β-sarcoglycan protein (β-SG) in the phase 3 EMERGENE clinical trial (NCT06246513).

3. Patients Treated With AskBio’s Limb-Girdle Muscular Dystrophy Gene Therapy AB-1003 Free of Serious Adverse Events at Up to 52 Weeks

Interim safety results from the randomized, placebo-controlled phase 1/2 LION-CS101 clinical trial (NCT05230459) evaluating Asklepios BioPharmaceutical (AskBio)’s AB-1003 (LION-101), an investigational AAV vector-based gene therapy intended to treat limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), have shown that patients remained free of serious adverse events (SAEs) at up to 52 weeks posttreatment.

4. AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Orphan Drug Designation, Cleared for Trial in UK

AAVantgarde Bio’s AAVB-039, an investigational AAV vector-based gene therapy intended to treat Stargardt disease, has received orphan drug designation from the FDA and clearance of a clinical trial application from the United Kingdom’s Medicines and Healthcare products Regulatory Agency.

5. INOVIO's DNA-Encoded Monoclonal Antibodies Demonstrate Proof of Concept in Phase 1 Trial

Results from a phase 1 clinical trial (NCT05293249) in healthy adult participants has shown that synthetic plasmid DNA administered intramuscular allows in vivo production of encoded monoclonal antibodies intended to prevent COVID-19. The findings were published in Nature Medicine. "We believe the data from all participants, now published in a premiere scientific journal, demonstrates the breakthrough potential of DNA-encoded monoclonal antibodies as a long-acting, scalable and tolerable alternative to traditional delivery of monoclonal antibodies," Laurent Humeau, PhD, INOVIO's chief scientific officer, said in a statement. "With promise in a broad range of diseases, we're excited to advance this novel technology with our current collaborators and through potential future partnerships."

6. Cell-Based Influenza Vaccine Shows Greater Protective Ability Than Egg-Based Vaccine in Real-World Study

Data from a real-world study of the United States 2023/2024 influenza season published in Infectious Diseases and Therapy and shared by CSL Seqirus showed that cell-based influenza vaccines has an estimated relative vaccine effectiveness of 19.8% (95% CI; 15.7 – 23.8%) over standard egg-based influenza vaccines in preventing test-confirmed influenza. “These data from the 2023/24 season show the clinical benefit of cell-based influenza vaccines over standard egg-based vaccines, and they continue to be relevant as we enter this year’s influenza season,” Gregg Sylester, MD, the chief health officer and head of medical affairs at CSL Seqirus, said in a statement. “Last year, we saw the worst flu season in 15 years and the highest number of pediatric deaths since the 2009 pandemic. Behind every statistic is a person, a family’s loved one, and we must continue to consider real-world evidence as an educational tool to encourage safe and effective vaccination for all eligible people 6 months and older.”