Around the Helix: Cell and Gene Therapy Company Updates – May 22, 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. Liso-Cel/Breyanzi Gets Accelerated Approval for Relapsed/Refractory Follicular Lymphoma

The FDA has granted accelerated approval to Bristol Myers Squibb’s lisocabtagene maraleucel chimeric antigen receptor (CAR) T-cell therapy for treating adults with relapsed or refractory follicular lymphoma who have received 2 or more prior lines of systemic therapy.

2. AADC Deficiency Gene Therapy up For FDA Priority Review

The FDA has accepted PTC Therapeutics’ biologics license application (BLA) for eladocagene exuparvovec gene therapy with priority review for treating patients with aromatic L–amino acid decarboxylase (AADC) deficiency with a PDUFA date of November 13, 2024.

3. Atara Submits BLA for Tab-Cel EBV+ PTLD T-Cell Therapy

Atara Biotherapeutics has submitted a BLA for tabelecleucel (tab-cel) monotherapy for treating adult and pediatric patients 2 years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least 1 prior therapy.

4. Ocugen Gene Therapies Progress in Stargardt, Retinitis Pigmentosa, and Geographic Atrophy

The company has announced that it completed dosing in cohort 2 of patients with Stargardt disease in the phase 1/2 GARDian trial (NCT05956626) of OCU410ST, received investigational new drug clearance for a phase 3 trial of OCU400 in patients with retinitis pigmentosa, and is enrolling the next cohort of patients with geographic atrophy in the phase 1/2 ArMaDa trial (NCT06018558) of OCU410.

5. Cartesian Therapeutics Garners RMAT Designation for Myasthenia Gravis CAR-T

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to Cartesian's lead product Descartes-08, an investigational autologous mRNA-engineered CAR-T therapy for the treatment of autoimmune diseases including myasthenia gravis (MG). It is currently being evaluated in the phase 2a portion of a clinical trial (NCT04146051) for MG.

6. Evotec Pulls Out of the Gene Therapy Business

The company has shut down its Evotec Gene Therapy site, which was located in Orth an der Donau, Austria, and dropped its pursuit of gene therapy development, as part of its "priority reset" strategy, with an eye for profitable growth and a focus on development of "core modalities". The company noted that approximately 40 of its employees may be affected by this change, but that it is working to minimize the impact for them.

7. Captain T Cell Snags €8.5 Million Via Seed Financing

Captain T Cell, a biotech focused on the development of T-cell receptor (TCR) T-cell therapies for solid tumor indications, has closed out a round of seed financing that has put a total of €8.5 million in its lap. "This successful financing round allows us to accelerate our high-potential therapies and brings us closer to providing life-saving options for patients underserved by current treatments," Felix Lorenz, PhD, the CEO of Captain T Cell, said in a statement. "We are steadfast in our mission to progress our lead candidate towards the clinic and to establish our TCR-ALLO platform as a leader in off-the-shelf solid tumor therapeutics.”

8. AmplifyBio and Xcell Biosciences Team Up With an Eye for Enhancing TCR T-Cell Manufacturing

Under a new partnership agreement, the 2 companies will work to streamline and otherwise improve manufacturing for TCR T-cell products aimed at solid tumor indications, with Xcell's AVATAR Foundry platform set to play a key role. "We’re excited to work together to demonstrate that T-cells grown in the AVATAR system can achieve higher potency and greater tumor-killing activity compared to T cells grown in traditional incubators," Brian Feth, MBA, the cofounder and CEO of Xcellbio, said in a statement. "This has tremendous potential to one day make a difference for patient care.”