Around the Helix: Cell and Gene Therapy Company Updates – December 20, 2023

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. Expert Insights at ASH’s 65th Annual Meeting

Catch up on CGTLive’s interviews with experts at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California. New videos will continue to be posted over the coming weeks.

2. FDA Blocks CARsgen’s 3 Clinical Stage CAR-Ts Over CMC Concerns

The FDA has placed clinical holds on 3 of CARsgen’s chimeric antigen receptor (CAR) T-cell therapies: CT053, also known as zevorcabtagene autoleucel (zevor-cel); CT071; and CT041; because of chemistry, manufacturing, and controls-related concerns that arose after an FDA inspection of the company’s manufacturing facility in Durham, North Carolina.

3. Excision Seeks to Suppress HIV Viral Replication With EBT-101 CRISPR Gene Therapy

In observance of HIV/AIDS Awareness Month, held annually in December, and following up on World AIDS Day, held annually on December 1, CGTLive took a closer look at the open-label, multicenter, phase 1/2 clinical trial (NCT05144386) for Excision BioTherapeutics’ EBT-101.

4. MSC Therapy Improves Pain, Stiffness, Function in Osteoarthritis Compared to Placebo

Stempeucel allogeneic mesenchymal stem cell (MSC) therapy (Stempeutics) improved symptoms of grade 2/3 osteoarthritis, according to new findings from a phase 3 trial conducted in India that were published in The American Journal of Sports Medicine.

5. Ocugen’s IRD Gene Therapy Garners FDA RMAT Designation

Ocugen’s OCU400, an investigational modifier gene therapy intended to treat inherited retinal diseases (IRD), has been granted regenerative medicines advanced therapies (RMAT) designation by the FDA for the treatment of retinitis pigmentosa associated with mutations in the RHO gene. This is the latest of several designations granted to OCU400, which utilizes the company's Modifier Gene Therapy Platform. The platform may allow the product to treat multiple retinal diseases.

6. Lexeo Snags Orphan Drug and Fast Track Designations for Arrhythmogenic Cardiomyopathy Gene Therapy

Lexeo Therapeutics’ LX2020 is an AAV vector-based gene therapy intended to provide a functional copy of plakophilin-2, a gene associated with arrhythmogenic cardiomyopathy. Several months prior to receiving these new designations from the FDA, LX2020 also received clearance from the agency for a first-in-human clinical trial.

7. Editas Medicines Extends Cash Runway Via New Deal With Vertex

Under a new nonexclusive license agreement, Vertex Pharmaceuticals, which codeveloped the recently FDA-approved CRISPR-based gene therapy exagamglogene autotemcel for the treatment of sickle cell disease with CRISPR Therapeutics, will gain the right to use Editas’ Cas9 editing technology in gene-editing medicines directed at the BCL11A gene. The move is expected to pull Editas’ cash runway into 2026.

8. Andelyn Biosciences Steps Up to the Plate for PPQ Manufacturing of Ultragenyx’s Sanfilippo Syndrome Gene Therapy

Under a new agreement, contract development and manufacturing organization Andelyn Biosciences will conduct late-stage process performance qualification (PPQ) manufacturing for Ultragenyx’s UX111, which is an intravenously administered self-complementary AAV9 vector-based gene therapy intended to treat mucopolysaccharidosis IIIA (also known as Sanfilippo syndrome type A).

9. FDA Approves AutoGenomics’ AvertD Genetic Test

AvertD, a DNA laboratory test exclusively for use under prescription, is intended to help determine the risk of opioid use disorder in patients making the decision of whether to go forward with a 4 to 30 day prescription course of oral opioid pain medications for acute pain. The test involves a cheek swab to collect DNA and may only be used for patients who are 18 years of age or older, have consented to the test, and have not previously received oral opioid analgesics.