Around the Helix: Cell and Gene Therapy Company Updates – January 8, 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 Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA

Capricor Therapeutics has completed submission of a rolling 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, to the FDA.

2. Arbor Biotechnologies’ CRISPR-Based Gene Editing Therapy ABO-101 Cleared for US Trial in Primary Hyperoxaluria Type 1

Arbor Biotechnologies’ ABO-101, a lipid nanoparticle (LNP)-delivered CRISPR-based gene editing therapy, has received clearance from the FDA for an investigational new drug (IND) application, enabling a phase 1/2 study in primary hyperoxaluria type 1 (PH1).

3. Ultragenyx Puts BLA for MPSIII Gene Therapy UX111 in Front of FDA

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

4. RESTEM’s ULSC Therapy Restem-L Fast Tracked by FDA for Idiopathic Inflammatory Myopathy

RESTEM’s Restem-L, an investigational umbilical cord outer lining stem cells (ULSCs) product, has been granted fast track designation by the FDA for idiopathic inflammatory myopathy (IIM, also known as polymyositis and dermatomyositis, PM/DM).

5. ViGeneron Snags FDA Rare Pediatric Disease Designation for CNGA1-Associated Retinitis Pigmentosa Gene Therapy

VG901, which is currently being evaluated in a phase 1b clinical trial, is intended to deliver a copy of the CNGA-1 gene via a proprietary AAV vector referred to as vgAAV. In addition to the new designation, ViGeneron also announced that the data safety monitoring board for the trial has given the green light for dose escalation.

6. uniQure Public Offering Pricing Shares at $17

uniQure has announced that its public offering of 4,411,764 ordinary shares will price each share at $17.00, which the company expects will lead to an aggregate gross proceeds of $75 million before relevant deductions are made. UniQure owns a hemophilia B gene therapy product along with its partner CSL Behring, etranacogene dezaparvovec (marketed as Hemgenix), which was approved by the FDA in November 2022. UniQure is also developing gene therapy products for other diseases including Huntington disease and amyotrophic lateral sclerosis.