Around the Helix: Cell and Gene Therapy Company Updates – June 19, 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. New Clinical Data Presented at EHA's 2024 Meeting

Catch up on CGTLive's coverage of the European Hematology Association (EHA) 2024 Congress, held June 13 to 16, both virtually and in Madrid, Spain.

2. FDA Recognizes BlueRock’s Parkinson Disease Cell Therapy With RMAT Designation

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BlueRock Therapeutics’ bemdaneprocel cell therapy for the potential treatment of Parkinson disease (PD).

3. Pfizer’s Phase 3 Trial for DMD Gene Therapy Fordadistrogene Movaparvovec Misses the Mark on Primary End Point

Pfizer’s phase 3 CIFFREO study (NCT04281485) evaluating fordadistrogene movaparvovec (PF-06939926), an investigational adeno-associated virus (AAV) vector-based gene therapy expressing what Pfizer calls “minidystrophin”, failed to achieve its primary end point, according to an announcement from the company.

4. Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Reduces Hereditary Angioedema Attack Rate in Long-Term Follow-Up Data

Intellia Therapeutics’ NTLA-2002, an investigational CRISPR/Cas9-based gene-editing therapy that is delivered systemically as a single-dose and is being evaluated in a phase 1/2 clinical trial (NCT05120830) for the treatment of hereditary angioedema (HAE), significantly reduced monthly HAE attacks, according to long-term follow-up data presented at the European Academy of Allergy and Clinical Immunology Congress 2024, held May 31-June 3 in Valencia, Spain.

5. Ultragenyx Intends to Seek Accelerated Approval for MPSIII Gene Therapy BLA

Following a meeting with the FDA, the company stated that it will seek an accelerated approval pathway, with the use of cerebral spinal fluid heparan sulfate as a surrogate end point, for a future biologics license application (BLA) submission for UX111, its AAV vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome).

6. New Company Spur Therapeutics Emerges Out of Freeline Therapeutics’ Acquisition of Swan Bio

Freeline Therapeutics, whose lead program constitutes Gaucher disease gene therapy candidate FLT201, has acquired Swan Bio, which is developing SBT101, an investigational gene therapy intended to treat adrenomyeloneuropathy. Notably, Syncona, the founding shareholder for both original companies, contributed $50 million to the newly combined company.

7. Charles River to Provide Manufacturing Support to Captain T Cell

Under a new production agreement, Charles River Laboratories will manufacture plasmid DNA and viral vectors for use in the production of a T-cell receptor (TCR) T-cell therapy that Captain T Cell is developing for the treatment of solid tumors. Notably, Captain T Cell recently snagged €8.5 million in a round of seed financing.

8. Roche and Ascidian Therapeutics Join Forces to Tackle Neurological Targets

In the new collaboration, through which Ascidian will receive an initial payment of $42 million and potential later payments of up to $1.8 billion, Roche and Ascidian will share preclinical responsibilities for the development of therapeutics based on Ascidian’s RNA exon editing platform. Following preclinical development, Roche will then be responsible for potential clinical development, manufacturing, and commercialization.