Around the Helix: Cell and Gene Therapy Company Updates – October 18, 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. Hold Lifted on ASO for Myotonic Dystrophy Type 1, Trial to Initiate in the US

The FDA has lifted a clinical hold on PepGen’s Investigational New Drug (IND) application for its FREEDOM-DM1 phase 1 study of PGN-EDODM1, an antisense oligonucleotide (ASO) for the potential treatment of Myotonic Dystrophy Type 1, which the company will initiate presently.

2. Kyverna’s CAR-T KYV-101 Gets FDA IND Clearance for Trial in Scleroderma

Kyverna Therapeutics’ KYV-101, an investigational chimeric antigen receptor T-cell (CAR-T) therapy, has received clearance of its IND application by the FDA for a clinical trial in patients with diffuse cutaneous systemic sclerosis (scleroderma).

3. BioCardia Targets Smaller Population of Heart Failure With Proposed Modified Study Protocol

BioCardia has completed enrollment in its phase 3 trial (NCT02438306) of CardiAMP in patients with heart failure with reduced ejection fraction and is initiating discussions with the FDA to create a second study protocol with modified endpoints of the trial after it was previously announced that the primary endpoint was unlikely to be reached.

4. Intellia’s CRISPR Therapy for ATTR-CM First to Enter Late-Stage Trials

The FDA has cleared Intellia Therapeutics’ IND application to initiate a phase 3 trial of NTLA-2001 in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy (CM). The trial, which is set to start by the end of 2023, will represent the first late-stage clinical trial to evaluate an in vivo CRISPR-based gene editing therapy.

5. Bill & Melinda Gates Foundation Extends Grant Supporting STRM.BIO’s Work on Cell-Specific Gene Therapy Delivery

STRM.BIO is conducting preclinical research on methods of delivering the foundation’s gene therapy products with the use of extracellular vesicles. The newly extended grant funding will be put towards research intended to allow for cell type specificity during in vivo delivery.

6. NIH/NINDS HEAL Program Recognizes Potential Gene Therapy Alternative to Opioids for Pain Relief

Roy C. Levitt, MD, a clinical professor in the Department of Anesthesiology at the University of Miami Miller School of Medicine, and the founder and executive chairman of Adolore, has received the UH3 Award from the NIH/NINDS HEAL Program for ADB-102, a replication defective herpes simplex virus vector-based carbonic anhydrase-8 analgesic peptide gene therapy intended to treat moderate-to-severe chronic osteoarthritis knee pain. "Chronic pain continues to be a major health problem worldwide that represents an annual cost of $650 billion in the US and there remains a significant unmet need for safe and effective non-opioid pain therapies,” Levitt said in a statement.

7. Be The Match BioTherapies Bolsters Cryopreservation Capacity Via New Collaboration With Cryoport

As part of the new agreement, the 2 partners will also aim to improve and apheresis collection and bioprocessesing with the ultimate goal of providing optimized cell therapy starting material to research institutions, cell therapy companies, and manufacturers in the United States and Europe.

8. SpliceBio and Spark Therapeutics Team Up to Tackle Retinal Disease

The agreement, which is focused on addressing a specific, yet undisclosed, inherited retinal disease, involves Spark making use of SpliceBio’s Protein Splicing platform to develop a novel gene therapy product aimed at addressing mutations in a gene that would be too large to deliver via an adeno-associated virus vector.

9. Arovella Therapeutics Licenses Monoclonal Antibody from SparX Group for use in Cell Therapy

As part of a new collaboration, Arovella Therapeutics will incorporate SPX-101, SparX’s monoclonal antibody (mAb) directed at Claudin 18.2, an antigen found on some solid tumor types, into its Invariant Natural Killer T (iNKT) cell therapy. "Our partnership with Arovella represents a transformative phase in advancing mAb-based therapies,” Michael Baker, PhD, the CEO of Arovella, said in a statement. “iNKTcells, distinguished by their recognition of lipid antigens via the CD1d molecule, have emerged as potent therapeutic vectors.”