Around the Helix: Cell and Gene Therapy Company Updates – May 7, 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. Clinical Stage Biotech Bloomsbury Genetic Therapies Shuttering its Doors

Bloomsbury Genetic Therapies, a biotech company developing several gene therapy candidates for ultra-orphan rare diseases, is in the process of shutting down its operations. The announcement came via a LinkedIn post made by the company’s cofounder and chief executive officer, Adrien Lemoine, MSc, who noted alongside the news that he will be stepping down from his role as CEO.

2. The Future of Capricor’s Cardiomyopathy Cell Therapy Deramiocel

Capricor Therapeutics is currently seeking to address treatment gaps in the landscape of care for Duchenne muscular dystrophy (DMD) with Deramiocel, its potentially-adjuvant allogeneic cardiosphere-derived cell therapy.
In an interview with CGTLive®, Linda Marbán, PhD, the CEO of Capricor Therapeutics, spoke about the implications of these data and future plans for Deramiocel. Notably, she pointed out that Deramiocel may eventually be evaluated for the treatment of other cardiomyopathies.

3. Neurophth is Tackling LHON-ND4 With Gene Therapy NR082 in Phase 1/2/3 GOLD Trial

Neurophth is currently evaluating NR082 (also known as Opvika and esonadogene mvoparvovec), an investigational gene therapy intended for the treatment of Leber hereditary optic neuropathy (LHON) associated with mtND4 mutations, in the phase 1/2/3 GOLD clinical trial (NCT04912843). For Healthy Vision Month, observed annually in May by the patient and clinician communities, CGTLive® has decided to take a closer look at this ongoing clinical trial.

4. Illuminating Genetic Insights in Parkinson Disease Through PD GENEration

The Parkinson’s Foundation recently initiated a campaign for educating the public on key aspects of Parkinson disease (PD), from symptoms to treatment options. The Foundation’s PD GENEration study, in partnership with Global Parkinson’s Genetics Program, offers alongside the PAM campaign whole genome sequencing (WGS) and genetic counseling to patients with PD across more than 8 countries. In an interview with CGTLive®'s sister site NeurologyLive^®, James Beck, PhD, the chief scientific officer of the Parkinson’s Foundation, highlighted the evolving role of genetics in PD, emphasizing how WGS through PD GENEration could be revealing unexpected findings that challenge clinical assumptions.

5. J&J Gene Therapy Treatment Fails Primary End Point

Data from the LUMEOS phase 3 clinical trial demonstrated a rare disease gene therapy referred to as botaretigene sparoparvovec (bota-vec) has failed to improve the vision-guided mobility of patients with X-linked retinitis pigmentosa (XLRP). Johnson & Johnson (J&J) purchased the full rights to bota-vec, an investigational gene therapy utilizing an adeno-associated virus to move a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene to the retina, in a 2023 deal with MeiraGTx.

6. Sangamo Desrisks Planned Fabry Gene Therapy BLA

Sangamo Therapeutics announced that all patients treated in its phase 1/2 STAAR clinical trial (NCT04046224) evaluating isaralgagene civaparvove (ST-920), an investigational adeno-associated virus vector-based gene therapy product intended to treat Fabry disease, have hit at least 52 weeks of follow-up. Crossing this threshold was a necessary precondition set by the FDA for the company to pursue an accelerated approval pathway for a biologics license application (BLA) submission for the therapy.