Around the Helix: Cell and Gene Therapy Company Updates – September 17, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. First Patient Treated in Capsida Biotherapeutics’ Trial for Encephalopathy Gene Therapy CAP-002 Passes Away

The first patient treated in the phase 1/2a SYNRGY clinical trial (NCT06983158), which is evaluating CAP-002, an investigational gene therapy intended to treat developmental and epileptic encephalopathy caused by syntaxin-binding protein 1 (STXBP1-DEE) mutations, has died following dosing.

2. Sanofi’s Neovascular Age-Related Macular Degeneration Gene Therapy SAR402663 Garners FDA Fast Track Designation

Sanofi’s SAR402663, an investigational gene therapy, has received fast track designation from the FDA for the treatment of neovascular age-related macular degeneration, also known as wet AMD.

3. ImmunoLogic, Episode 6: "The Future of Personalized Cancer Vaccines”

In this episode, the cohosts speak with Stephen Schoenberger, PhD, a professor at the Center for Cancer Immunotherapy at the La Jolla Institute, about his research into personalized cancer vaccines.

4. Duchenne Action Month 2025: Looking Back at News and Expert Insights

In honor of Duchenne Action Month, observed annually in September by the patient and clinician communities, and following up on World Duchenne Day, held every year on September 7, CGTLive® took a look back at some of our news and interviews on the topic of DMD from the past few months.

5. AskBio Maxes Out Enrollment in Trial for MSA-P Gene Therapy

Asklepios BioPharmaceutical (AskBio) has completed enrollment of patients in the phase 1 clinical trial REGENERATE MSA-101 (NCT04680065), which is evaluating AB-1005 (AAV2-GDNF), an investigational adeno-associated virus vector serotype 2 (AAV2)-based gene therapy for the treatment of multiple system atrophy-parkinsonian type (MSA-P). Notably, AB-1005 is also being evaluated in a separate phase 2 clinical trial (REGENERATE-PD; NCT06285643) for Parkinson disease.

6. Myrtelle’s Canavan Disease Gene Therapy Effects Significant Decrease in CSF NAA and Increases in Brain Myelin Volume

According to new results from a phase 1/2 clinical trial (NCT04833907) that were recently published in Nature Medicine, Myrtelle’s rAAV-Olig001-ASPA (MYR-101), an investigational recombinant AAV vector-based gene therapy intended to treat Canavan disease, showed the ability to significantly decrease levels of N-Acetylaspartate (NAA) in the cerebrospinal fluid (CSF) and the ability to significantly increase brain myelin volume. “The reductions in CSF NAA levels, gains in myelin volume, and functional improvements in treated patients mark a compelling step forward for patients who currently have no approved treatment options,” Adrian Stecyk, MBA, the CEO of Myrtelle, said in a statement.