Around the Helix: Cell and Gene Therapy Company Updates – July 16, 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. Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel Slammed With CRL

Capricor Therapeutics has received a complete response letter (CRL) from the FDA regarding its biologics license application (BLA) for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy that was under review for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy.

2. Nanoscope Therapeutics Puts Rolling BLA for Retinitis Pigmentosa Gene Therapy MCO-010 in Front of FDA

Nanoscope Therapeutics has submitted a rolling BLA to the FDA for MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, for the treatment of retinitis pigmentosa.

3. Ultragenyx’s MPSIIIA Gene Therapy UX111 Met With CRL

Ultragenyx has received a CRL from the FDA regarding its BLA for UX111 (ABO-102), an adeno-associated virus (AAV) vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome).

4. Fostering Collaboration in Cell and Gene Therapy Research

CGTLive spoke with Nathan Yozwiak, PhD, the head of research at Mass General Brigham’s Gene and Cell Therapy Institute (GCTI), to learn more about how GCTI accomplishes its mission. Yozwiak emphasized the unique aspects of GCTI and highlighted the role of its scale in bringing researchers and other experts together.

5. Klotho Neurosciences Snags Orphan Drug Designation for ALS Gene Therapy

Klotho's KLTO-202 is a secreted-Klotho (s-KL) promoter, gene, and delivery system (KLTO-202, or s-KL-AAV.myo) intended to treat amyotrophic lateral sclerosis (ALS) by promoting expression of s-KL protein. "We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein," Joseph Sinkule, the CEO of Klotho, said in a statement.

6. Genascence Garners RMAT Designation for Knee Osteoarthritis Gene Therapy

Genascence has received regenerative medicine advanced therapy (RMAT) designation for GNSC-001, an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy intended to block expression of interleukin 1 (IL-1) that has been evaluated in 2 clinical trials (NCT02790723 and NCT05835895) for the treatment of knee osteoarthritis (OA). “GNSC-001 is the first IL-1 inhibitor for OA that has been shown to generate IL-1Ra expression levels that maintain therapeutic thresholds long-term following a single administration to inhibit pathogenic IL-1," Thomas Chalberg, PhD, the founder and CEO of Genascence, said in a statement. "The FDA RMAT designation for GNSC-001 underscores the strength of the clinical data to date, recognizing its potential to transform the treatment paradigm for OA."