Around the Helix: Cell and Gene Therapy Company Updates – October 25, 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. BrainStorm Cell Therapeutics Pulls BLA for ALS Cell Therapy NurOwn

BrainStorm Cell Therapeutics has withdrawn its biologics license application (BLA) for debamestrocel (NurOwn), an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product intended to treat amyotrophic lateral sclerosis (ALS), prior to its Prescription Drug User Fee Act action date, which was slated for December 8, 2023.

2. Nkarta Enters the Lupus Nephritis CAR Cell Therapy Race With NKX019 IND Clearance

Nkarta’s NKX019, an investigational allogeneic chimeric antigen receptor (CAR) natural killer (NK) cell therapy, has received clearance of an investigational new drug (IND) application enabling its evaluation in a clinical trial for patients with lupus nephritis (LN). Notably, Lupus Therapeutics, the clinical research affiliate of the Lupus Research Alliance, has thrown their support behind Nkarta’s program.

3. Verve’s Hypercholesterolemia Gene-Editing Therapy VERVE-101 Cleared for US Trial After Rocky Start

Verve Therapeutics’ IND application for VERVE-101, an investigational gene-editing therapy intended to treat heterozygous familial hypercholesterolemia, has been cleared by the FDA. Verve originally submitted the IND application to the FDA in October 2022, but it was placed on hold by the agency in November 2022.

4. Trial for CytoAgents’ Drug for Management of CAR-T-Associated CRS Begins Enrolling Participants

CytoAgents has begun enrolling patients in its phase 1b/2a clinical trial (NCT05905328; CTA-2101) evaluating CTO1681, its small molecule drug intended to treat cytokine release syndrome (CRS, also known as cytokine storm) associated with treatment with CAR T-cell (CAR-T) therapy.

5. Epidermolysis Bullosa Awareness Week Begins

To kick off Epidermolysis Bullosa (EB) Awareness Week, observed annually from October 25 to 31 by the patient and clinician community, CGTLive interviewed Brett Kopelan, MA, the executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, and Heather Fullmer, the community engagement director of debra of America, about the current state of care in EB and how the advent of gene therapy could potentially change the treatment landscape for this patient population.

6. Newly Launched Company VedaBio Seeks to Disrupt Molecular Detection Field 

VedaBio, which recently launched with more than $40 million from investors, announced that its CRISPR Cascade Platform can provide analytical turnaround in less than 1 minute. “With the CRISPR Cascade reaction, we have unlocked the true power of CRISPR with a platform that delivers near-instant molecular detection of highly multiplexed analytes with best-in-class accuracy, all without the need for target amplification,” Anurup Ganguli, PhD, the co-founder and chief executive officer of VedaBio, said in a statement.

7. Ultragenyx to Launch Cash Neutral Spinout Focused on Alzheimer Disease

Almogenyx, which will receive relevant intellectual property and know-how from majority shareholder Ultragenyx, will carry out activities related to the development of a therapeutic consisting of a lysosomal protease delivered by an AAV vector for the treatment of Alzheimer disease. “I couldn’t sit by and watch something this exciting and not actually put it into play,” Emil Kakkis, MD, PhD, the president and CEO of Ultragenyx, said to investors, as reported by Biopharma Dive. “I can see this just being a leap ahead of how you might treat Alzheimer disease.”

8. Beam Therapeutics to Let Go of 20% of Employees

The downsizing, which will affect over 100 members of the company’s workforce, is expected to be carried out before the end of the year. The move is part of a series of measures related to its strategic restructuring; the company is seeking to prioritize its programs for sickle cell disease and alpha-1 antitrypsin deficiency.

9. OBiO Technology to Provide Refreshgene With CDMO/CMO Services

OBiO will provide contract development and manufacturing organization (CDMO) and contract manufacturing organization (CMO) services related to Refreshgene Therapeutics’ RRG-001 gene therapy program. "In the chemical, manufacturing, and controls development of RRG001 pipeline's IND enabling, we chose to cooperate with OBiO, which is proved to be an experienced and collaborative CDMO,” Liu Xinxing, the CEO of Refreshgene, said in a statement. “As a result, we further strengthen and expand the scope of strategic cooperation with OBiO.”

10. GenomeFrontier Enlists BioCina’s CDMO Services

As part of a newly announced agreement, BioCina will provide CDMO services related to GenomeFrontier’s program aimed at addressing cancer with a CAR-T that is manufactured with the use of Minicircle DNA and Plasmid DNA rather than DNA delivered via a viral vector.