Around the Helix: Cell and Gene Therapy Company Updates – November 27, 2024

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. FDA Approves StemCyte’s Cord Blood Cell Therapy Regenecyte for Unrelated Donor Hematopoietic Progenitor Cell Transplantation Procedures

The FDA has approved StemCyte’s Regenecyte, an allogeneic hematopoietic stem cell therapy derived from human umbilical cord blood, for use in unrelated donor hematopoietic progenitor cell transplantation procedures alongside an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with inherited or acquired disorders affecting the hematopoietic system, including those resulting from myeloablative treatment.

2. Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy

Genethon’s GNT0004, an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy intended to treated Duchenne muscular dystrophy (DMD), has demonstrated the ability to stabilize or improve motor function in patients treated in the phase 1/2 portion of a phase 1/2/3 clinical trial.

3. First Patient Dosed in RIDGE-1 Trial for Tenaya’s ARV Cardiomyopathy Gene Therapy TN-401

The first patient has been dosed in the phase 1b RIDGE-1 clinical trial (NCT06228924) evaluating Tenaya Therapeutics’ TN-401, an investigational AAV-based gene therapy, for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).

4. Improving Patient Access to Cell Therapy Through Decentralized Manufacturing

Although the cell and gene therapies (CGTs) industry continues to advance the approvability, accessibility, and affordability of this new generation of treatments, it remains a constant challenge to create not only safe and effective products but also commercially viable ones. Escalating development costs and complex supply chains have manufacturers increasingly exploring new production models to streamline logistics, increase throughput, and enhance patient access.

5. Ocugen's OCU410ST Garners EMA Orphan Drug Designation

Ocugen's OCU410ST, an investigational AAV vector-based gene therapy based on the company's modifier gene therapy platform that delivers the RORA (RAR Related Orphan Receptor A) gene, has received orphan drug designation from the European Medicines Agency (EMA) for the treatment of Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

6. Vyriad and Novartis Team Up to Tackle In Vivo CAR-T Race

Vyriad and Novartis have entered a strategic collaboration agreement aimed at developing an in vivo approach to generating chimeric antigen receptor T-cell (CAR-T) therapy. Vyriad will provide its active targeting lentiviral vector platform to the effort. “Taking an in vivo approach to CAR-T cell therapies has the potential to transform the field,” Stephen J. Russell, MD PhD, the CEO of Vyriad said in a statement. “We believe our targeted delivery platform is poised to enable this vision for the industry and our collaboration with Novartis represents an exciting step forward in advancing the next generation of CAR-T cell treatments.”