Around the Helix: Cell and Gene Therapy Company Updates – July 31, 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. Pfizer Drops Development of DMD Gene Therapy Fordadistrogene Movaparvovec

Pfizer has decided to discontinue development of fordadistrogene movaparvovec (PF-06939926), an investigational adeno-associated virus (AAV) vector-based gene therapy expressing what Pfizer calls “minidystrophin”, which was being evaluated in the phase 3 CIFFREO study (NCT04281485) for the treatment of Duchenne muscular dystrophy, according to news reported by FirstWord Pharma.

2. Actinium’s Antibody Radiation Conjugate Iomab-ACT Cleared for US Trial as Conditioning Therapy for SCD Allo-BMT

Actinium’s Iomab-ACT, an investigational antibody radiation conjugate (ARC) intended to serve as an alternative to chemotherapy conditioning agents, has received clearance of an investigational new drug application from the FDA for trial evaluating it as a conditioning agent for patients planning to receive allogeneic bone marrow transplant (allo-BMT) for the treatment of sickle cell disease (SCD).

3. Fidanacogene Elaparvovec Gets European Approval for Hemophilia B

The European Commission has granted conditional marketing authorization to Pfizer’s fidanacogene elaparvovecgene therapy under the name Durveqtix for the treatment of adults with severe and moderately severe hemophilia B without a history of factor IX (FIX) inhibitors and without detectable antibodies to variant adeno-associated virus (AAV) serotype Rh74.

4. Roche Drops Pompe Gene Therapy Program

Roche has discontinued development of its investigational gene therapy SPK-3006, which was being evaluated in the phase 1/2 RESOLUTE trial (NCT04093349) in participants with late-onset Pompe disease.

5. Nexcella Garners $8 Million CIRM Grant for Amyloidosis CAR-T

The CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) will go towards the advancement of Nexcella's NXC-201 (formerly referred to as HBI0101), an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets B-cell maturation antigen, for the treatment of relapsed/refractory light chain amyloidosis.

6. MilliporeSigma and Gene Therapy Research Institution Team Up to Tackle Parkinson Disease

Under a new memorandum of understanding, MilliporeSigma will provide the use of its Sf-RVN® Insect Cell Line platform to the Japanese biotech Gene Therapy Research Institution to aid the latter's development of an AAV vector-based gene therapy approach intended to treat Parkinson disease.

7. Umoja Biopharma's In Situ CAR-T Therapy Cleared for US Trial in Hematologic Malignancies

The FDA has cleared an investigational new drug application for Umoja Biopharma's UB-VV111, a gene therapy product intended to create CD19-directed CAR T-cells within the body, allowing the company to go forward with a phase 1 dose escalation trial. It will be open to patients with relapsed/refractory large-B-cell lymphoma and chronic lymphocytic leukemia, regardless of whether they have previously received treatment with a CAR-T product.

8. MD Anderson to Study T-cells in Outer Space With Eye for Advancing Cell Therapy

Along with collaborators, MD Anderson has begun a project that will involve research on T-cell biology conducted in the microgravity environment found on the International Space Station. The collaborators hope that the investigations will reveal new information that could inform the development of cell therapy modalities including CAR-T, as endogenous T-cell therapy, T-cell receptor-based therapy, and CAR natural killer cell therapy.