Around the Helix: Cell and Gene Therapy Company Updates – December 6, 2023

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. Adaptimmune Submits Afami-Cel First Engineered TCR-T Therapy BLA for Synovial Sarcoma

Adaptimmune Therapeutics has submitted its Biologics License Application (BLA) for afami-cel (afamitresgene autoleucel) T-cell receptor (TCR) T-cell therapy for treating synovial sarcoma.

2. uniQure Expanding Clinical Pipeline to Fabry Disease

The FDA has cleared uniQure’s investigational new drug application to evaluate AMT-191 gene therapy in patients with Fabry disease.

3. Patient Eligibility Expanded for Phase 1/2 REVEAL Trial of TSHA-102 in Rett Syndrome

Health Canada has granted authorization of a protocol amendment to the ongoing phase 1/2 REVEAL trial (NCT05606614) of Taysha Gene Therapies’ investigational treatment TSHA-102, according to a recent announcement.

4. Direct Biologics’ Phase 1 Clinical Trial Seeks to Treat Crohn Disease With Extracellular Vesicles

Direct Biologics’ ExoFlo, an investigational therapy composed of extracellular vesicles derived from bone marrow mesenchymal stem cells, is currently being evaluated in a phase 1 clinical trial for the treatment of medically refractory Crohn disease. In observance of Crohn and Colitis Awareness Week, observed from December 1 to 7, 2023, by the patient and clinician communities, CGTLive decided to take a closer look at this ongoing study.

5. After Elevidys: Duchenne Muscular Dystrophy Advocacy Past First Gene Therapy Approval

CGTLive looked back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

6. CRISPR Therapeutics Seeks Greener Pastures in Autoimmune Disease CAR-T

CRISPR noted that it is seeking to expand its assessment of CTX112, its next-generation CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy currently being evaluated for the treatment of B-cell malignancies, into autoimmune disease indications like systemic lupus erythematosus. The company also announced that it plans to discontinue development of its first-generation oncology CAR-T therapies CTX110 and CTX130.

7. Phenomic AI Seeks to Give Astella’s Cell Therapies a Boost With Antibodies

Under a new collaboration agreement, Phenomic will leverage its scTx single cell transcriptomics platform for the development of antibodies directed at a target in the solid tumor stroma with the aim of improving the efficacy of Astellas Pharma's cell therapies. “scTx’s massive database and machine learning-engine provides insights into the tumor stroma, a major barrier to cancer therapies, at a resolution not previously possible and ultimately enables Phenomic to develop therapies for the hardest to treat tumors,” Girish Aakalu, PhD, the CEO of Phenomic, said in a statement.

8. Brain Tumor CAR-T to be the Focus of New Seattle Children’s Spinout

The newly launched BrainChild Bio, which received initial equity funding and an exclusive license to CAR-T technology from Seattle Children’s but will conduct itself as an independently managed company, will direct its initial efforts towards developing a CAR-T therapy for the pediatric cancer diffuse intrinsic pontine glioma.

9. FUJIFILM Infuses Cash Into Its Cell Therapy Manufacturing Subsidiaries

The investment of $200 million in total will be put toward completion of a new headquarters for manufacturing and research for FUJIFILM Cellular Dynamics and expansion of an existing site for FUJIFILM Diosynth Biotechnologies. “This investment is an important step for Fujifilm to lead as an innovative global manufacturing partner for the growing cell therapy market,” Teiichi Goto, the president, CEO, and representative director of the FUJIFILM Corporation, said in a statement. “The combined resources of the Fujifilm Group will enable us to meet the diverse and expanding manufacturing needs of our customers.”

10. Chinese Biotech Insider Reaffirms the Utility of CAR-T Therapy Following FDA Announcement of Risk Investigation

According to Yicai Global, following the FDA’s announcement that it will be investigating the risk of T-cell malignancies in patients that have received B-cell maturation antigen (BCMA)-directed or CD19-directed autologous CAR-T therapies, an unnamed insider from a Chinese biopharmaceutical company expressed the view that although further investigation is warranted, the benefits of CAR-T therapy outweigh the risks for patients with cancer.