Around the Helix: Cell and Gene Therapy Company Updates – April 3, 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. Verve Moves on to Second Gen Cardiovascular CRISPR Therapy After Adverse Events

Verve Therapeutics is pausing enrollment in its phase 1b heart-1 clinical trial (NCT05398029) of VERVE-101 CRISPR-editing therapy in consultation with the study’s independent data and safety monitoring board after the sixth participant treated experienced asymptomatic grade 3 adverse events.

2. First US Patient With Hemophilia B Receives Gene Therapy

The Center for Inherited Blood Disorders has administered the first successful gene therapy infusion to a patient with hemophilia A in the clinical setting.

3. Zevorcabtagene Autoleucel Approved in China for R/R Multiple Myeloma, Phase 2 Study in US Ongoing

In March, China’s National Medical Products Administration approved CARsgen's autologous chimeric angtigen receptor (CAR) T-cell therapy zevorcabtagene autoleucel (zevor-cel; previously known as CT053) for the treatment of adult patients with relapsed/refractory multiple myeloma (r/r) whose disease progressed after at least 3 lines of therapy, including treatment with a proteasome inhibitor and an immunomodulatory drug.

4. Atamyo’s Limb-Girdle Muscular Dystrophy Type 2C/R5 Gene Therapy Cleared for Clinical Trial in Italy and France

Atamyo Therapeutics’ clinical trial application for ATA-200, an investigational adeno-associated virus vector-based gene therapy for the treatment of γ-sarcoglycan-related limb-girdle muscular dystrophy Type 2C/R5, was subsequently cleared by the Italian Medicines Agency and then by the French Medicines Agency.

5. bluebird bio’s Shares Drop Following Accounting Error Reveal

bluebird bio, which is currently in the process of launching its recently FDA-approved sickle cell disease gene therapy lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), experienced a 12% drop in the value of its shares after it disclosed an accounting error that will necessitate a restating of financial statements from all of 2022 and much of 2023.

6. Investors React Negatively to Nkarta’s Shift to Autoimmune Disease Focus

Nkarta’s shares plummeted in value by 31% after the company made the choice to deprioritize 2 of its oncologyCAR natural killer cell therapy programs, NKX101 and the cancer-specific program for NKX019, in favor of prioritizing NKX019’s development in lupus nephritis.

7. Michael J. Fox Foundation Will Fund bit.bio’s Cell-based Research in Parkinson Disease

Under a new collaboration agreement, Michael J. Fox Foundation (MJFF) will provide financing for certain bit.bio research efforts in Parkinson disease (PD). The collaborators announced that for the first project under this agreement, bit.bio will seek to develop a specific cell type relevant to PD into a cell-based product for use in PD research. “This important collaboration with MJFF gives us the opportunity to accelerate the development and delivery of a series of human cell types and associated disease models to the PD research community,” Mark Kotter, MD, PhD, the founder and CEO of bit.bio, said in a statement.

8. Carisma Therapeutics Announces Layoffs in Relation to New Priorities

Carisma Therapeutics announced that it is prioritizing the development of CT-0525, an investigational antiHER2 CAR macrophage (CAR-M) product, over CT-508, another of its investigational antiHER2 CAR-M products. Carisma stated that in relation to this change, other pipeline reprioritizations, and a corporate restructuring, it will be letting go about 37% of its employees during Q2.