Around the Helix: Cell and Gene Therapy Company Updates – April 24, 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. FDA Issues CRL for Abeona's Epidermolysis Bullosa Gene Therapy Pz-Cel

The FDA has issued a complete response letter (CRL) for the biologics license application (BLA) for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy for the treatment of patients with recessive dystrophic epidermolysis bullosa, according to an announcement from Abeona Therapeutics, the therapy’s developer.

2. European Commission Approves Janssen and Legend Biotech’s Carvykti for Expanded Indication in Earlier Line Multiple Myeloma

The European Commission has approved Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti) for an expanded indication in adult patients with relapsed/refractory multiple myeloma who have been treated with at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, showed disease progression on their last therapy, and have disease that is lenalidomide-refractory.

3. One Big Step Away From BLA Submission for Sangamo’s Fabry Gene Therapy

The FDA has aligned with Sangamo Therapeutics on an abbreviated pathway to approval of its gene therapy ST-920 (isaralgagene civaparvovec) for treating Fabry disease following the presentation of data demonstrating disease improvements after treatment from the phase 1/2 STAAR trial (NCT04046224). Sangamo is seeking a collaboration partner to advance the therapy through potential registration and commercialization.

4. FDA Requires Boxed Warnings for Secondary Cancer for Approved CAR-T Therapies

The FDA is requiring that boxed warnings for T-cell malignancies after treatment with chimeric antigen receptor (CAR) T-cell therapy be added to all approved CAR-T therapies.

5. Bristol Myers Squibb Enlists Cellares to Streamline its CAR-T Manufacturing

Under a new capacity reservation and supply agreement, valued at up to $380 million in total, Cellares will furnish the use of several of its Cell Shuttle and Cell Q systems for Bristol Myers Squibb's exclusive use in the manufacture of its own CAR-T products.

6. GeneVentiv Therapeutics Handed $2.5 Million Grant for Preclinical Hemophilia Gene Therapy

GeneVentiv has secured a Direct to Phase II Small Business Innovation Research grant from the National Heart Lung Blood Institute to support investigational new drug (IND) application-enabling studies for GENV-HEM, an investigational AAV gene therapy product intended to treat both hemophilia A and hemophilia B, with or without inhibitors.

7. GSK Chooses 2 of Wave Life Sciences' siRNA Programs for Further Development

Under an established collaboration agreement, GSK has elected to pay Wave $12 million for the option to further develop 2 preclinical small interfering RNA (siRNA) programs for liver indications. GSK's role will include taking the programs through IND-enabling studies, clinical trials, and commercialization. “We’ve reached an exciting point in our research collaboration, as selection of GSK’s first 2 programs signals the next phase of our work together to bring transformative RNA medicines to patients,” Paul Bolno, MD, MBA, president and chief executive officer of Wave Life Sciences, said in a statement.

8. CSafe Launches Cryogenic Storage Dewar for Transport of Cell and Gene Therapy Products

CSafe, a company focused on temperature-controlled shipping, has launched a line of cryogenic dewars intended for use in shipping cell and gene therapy products. The dewars, which are part of the company's CGT Cryo Series, are reusable, capable of maintaining temperatures below -150 °C, and are high-impact-resistant.