Around the Helix: Cell and Gene Therapy Company Updates – April 17, 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. Mustang Bio Seeks to Bring CD20-Targeted CAR-T MB-106 From Cancer to Autoimmune Disease

Mustang Bio has announced its intention to pursue a phase 1 investigator-sponsored clinical trial for the evaluation of MB-106, an investigational autologous CD20-directed chimeric antigen receptor T-cell (CAR-T) therapy originally developed for the treatment of hematological malignancies, in autoimmune disease.

2. Tr1X’s Allogeneic Treg Therapy TRX103 Cleared for US Trial for Preventing GvHD

Tr1X’s TRX103, an investigational allogeneic regulatory T-cell (allo-Treg) therapy, has received clearance of an investigational new drug (IND) application from the FDA for a clinical trial for the prevention of graft versus host disease (GvHD) in patients undergoing HLA-mismatched hematopoietic stem cell transplantation.

3. 4DMT Aligns on Path Forward for Cystic Fibrosis Gene Therapy

4D Molecular Therapeutics (4DMT) has aligned on a pivotal phase 3 trial and registration path for developing 4D-710 gene therapy for the treatment of cystic fibrosis (CF) lung disease for patients whose disease is ineligible for or cannot tolerate approved CF modulator therapies. “We are encouraged by our productive interactions with the FDA and EMA on pivotal development plans for 4D-710, which we believe has the potential to be a transformative therapy for people with the highest unmet medical need for CF lung disease,” David Kirn, MD, cofounder and Chief Executive Officer, 4DMT, said in a statement.

4. New Clinical Data Presented at AACR 2024

Catch up on CGTLive's spot coverage of the American Association for Cancer Research (AACR) Annual Meeting 2024, held on April 5-10, 2024, in San Diego, California.

5. Adaptimmune and Genentech Axe Multiyear Cell Therapy Collaboration

The 2 companies, which formed a collaboration agreement in 2021, had sought to develop allogeneic T-cell therapies for cancer. In light of the termination, Adaptimmune noted it will continue to focus on its own late-stage cell therapy product, afami-cel, which is nearing a potential FDA approval in sarcoma.

6. Precision Biosciences Closes Out Gene Editing Collaboration With Prevail

Precision Biosciences has closed out a collaboration with Prevail Therapeutics that the 2 companies originally entered in 2021, with Precision now having exercised its option to regain control of the 3 preclinical gene editing programs that Prevail had been working on advancing towards clinical trials.

7. Century Therapeutics Brings Clade Therapeutics Into the Fold

Century Therapeutics has acquired Clade Therapeutics, thus expanding its pipeline of induced pluripotent stem cell-derived cell therapy programs for autoimmune disease and oncology indications. Alongside this announcement, Century also reported that it plans to submit IND applications for additional autoimmune disease indications for its lead cell therapy program CNTY-101 and that it secured a $60 million private placement of its common stock.

8. CMS Proposes Raising the Reimbursement Rate for Lovo-cel and Exa-cel

The Centers for Medicare & Medicaid Services (CMS) has issued a proposed rule for the 2025 fiscal year that includes a proposal to raise the new technology add-on payment percentage for the 2 recently approved sickle cell disease gene therapies to 75%, up from the typical 65%.