Around the Helix: Cell and Gene Therapy Company Updates – May 14, 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. FDA States Advisory Committee Meeting Will be Held for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

In a mid-cycle review meeting between the FDA and Capricor Therapeutics regarding the latter’s biologics license application (BLA) for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, the agency stated its intent to hold an advisory committee meeting regarding the BLA.

2. World Ovarian Cancer Day 2025: Looking Back at Progress for Cell Therapy

In honor of World Ovarian Cancer Day, observed annually on May 8 by the patient and clinician communities, CGTLive® took a look back at the progress that was made for cell therapy in ovarian cancer over the past year.

3.Expanding Access to Whole Genome Sequencing in Parkinson Research

CGTLive®'s sister site NeurologyLive^® sat down with James Beck, PhD, chief scientific officer of the Parkinson’s Foundation, to discuss the importance of regional and cultural adaptation and the need to train clinicians in genetic counseling.

4. Aneal Khan MD, MSc, FCCMG, FRCPC, on Evaluating Gene Therapy for Fabry Disease

Following up on Fabry Disease Awareness Month, observed annually in the month of April, CGTLive® got in touch with Aneal Khan MD, MSc, FCCMG, FRCPC, the president of MAGIC Clinic and CEO of Discovery DNA, to discuss gene therapy research in Fabry disease. Khan spoke about the data presented at WORLDSymposium this year and about innovation in the field more generally.

5. Abeona Sells Off Priority Review Voucher Obtained Via Pz-Cel Approval

In April, the FDA granted Abeona Therapeutics a rare pediatric disease priority review voucher in conjunction with the approval of prademagene zamikeracel (pz-cel; EB-101), the company's autologous gene-corrected epidermal sheet therapy, for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). On May 12, Abeona announced that it had agreed to sell the voucher to an undisclosed recipient for $155 million in gross proceeds.

6. BioIVT Brings New Cryopreserved Leukopaks to Market

BioIVT has launched its VivoSTART Cryopreserved GMP Leukopaks, good manufacturing practice (GMP)-compliant cryopreserved leukopaks that are intended to serve as starting material in cell therapy and gene therapy development programs. "Taking into account clients who require GMP-compliant cellular starting materials but may lack the capacity or resources to integrate fresh leukopaks into their development timelines, cryopreserved leukopaks offer the flexibility to allocate and schedule resources for downstream processing of leukapheresis material—ensuring client needs are met through our growing portfolio," Parijat Jain, PhD, the vice president of cell & gene therapy at BioIVT, said in a statement.

7. Spur Therapeutics’ Gene Therapy SBT101 Generally Well-Tolerated in Phase 1 Trial for Adrenoleukodystrophy

Spur Therapeutics’ SBT101, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat X-linked adrenoleukodystrophy, was deemed “generally well-tolerated" in new safety data from the ongoing phase 1/2 PROPEL clinical trial (NCT05394064). The data were presented in a poster at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA.