Around the Helix: Cell and Gene Therapy Company Updates – March 23, 2022

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. 

1. Lineage Cell Therapeutics to Develop Cell Therapy for Hearing Loss

Lineage Cell Therapeutics is expanding its pipeline to develop a cell therapy for the potential treatment of hearing loss, adding another neuronal cell type to their platform. The company will initially focus on auditory neuropathy spectrum disorders.

2. Huntington Disease Gene Therapy AMT-130 Completes Trial Enrollment

uniQure has completed patient enrollment of the first 2 cohorts in its phase 1/2 clinical trial (NCT04120493) of AMT-130, a gene therapy for the potential treatment of early manifest Huntington disease.

3. T-Cell Therapy Gets Orphan Drug Designation for AML

The FDA has granted orphan drug designation to TC Biopharm's T-cell therapy TCB-002 (OmnImmune) for the potential treatment of relapsed/refractory acute myeloid leukemia (AML). The decision was based off ofpositive data from a phase 1a/2b trial (NCT03790072). The company also received MHRA and Research Ethics Committee approvals to initiate a phase 2B/3 clinical trial of the gamma-delta T cell therapy.

4. Val-Rox Gene Therapy Restores Factor VIII Production and Reduces Bleeding in Hemophilia A

Valroctocogene roxaparvovec (val-rox; BioMarin) restored endogenous factor VIII production and significantly reduced bleeding and use of enzyme replacement therapy in patients with hemophilia A, according to complete results from the phase 3 GENEr8-1 study (NCT03370913) published in the New England Journal of Medicine.

5. 2-year Data for Duchenne Muscular Dystrophy Gene Therapy Shows Durable Improvements

SGT-001, Solid Biosciences’ investigational gene therapy for treating Duchenne muscular dystrophy (DMD), improved motor and pulmonary function in treated patients over 2 years, according to new data from the phase 1/2 IGNITE-DMD study (NCT03368742).

6. SQZ Biotech Awarded $2 Million to Develop Parkinson Disease Cell Therapy

The National Institute of General Medical Sciences at National Institutes of Health (NIH) has awarded a $2 million grant to SQZ Biotechnologies to support the development of a novel cell therapy for the potential treatment of Parkinson disease.

7. Ixaka Expands Use of Targeted Nanoparticle Gene Delivery Platform

The company has expanded their intellectual property to develop a variety of therapies delivered with their TNP platform, including mRNA, plasmids and adenovirus associated virus therapies, gene editing technologies as well as lentiviral vector-based therapies.

8. AlloVir Initiates Posoleucel Study for Cell Transplant Viruses

Posoleucel is being evaluated in a phase 3 registrational study. The allogeneic, off-the-shelf, multi-virus-specific T-cell therapy is designed to prevent adenovirus, BK virus, cytomegalovirus, Epstein-Barr virus, human herpesvirus-6, and JC virus in high-risk patients after allogeneic hematopoietic cell transplant.

9. BioCardia’s CardioAMP Cell Therapy Eligible for Medicaid/Medicare Reimbursement

The US Center for Medicare and Medicaid Services has designated a reimbursement code for CardioAMP starting in April 2022. The cell therapy is being investigated in 2 trials for heart failure (NCT02438306) and chronic myocardial ischemia (NCT03455725).

10. iTolerance Advances Development of Immunosuppression-Free Cell Therapy

The company raised $17.1 million in convertible note financing to help develop their iTOL-100 platform, which consists of Strepavidin-FasL fusion protein/biotin-PEG microgel technology. The platform allows for local immune tolerance of allogeneic cell therapies without immunosuppression. iTolerance's lead candidate, iTol-101, targets type 1 diabetes.

11. Affini-T Therapeutics Advances Solid Tumor T-Cell Therapies

Affini-T has completed a $175 million financing co-led by Vida Ventures and Leaps by Bayer. The company will use the funding to advance the development of its KRAS- and p53-targeted T-cell receptor cell therapies.

12. FUJIFILM Acquires Shenandoah Biotechnology

Shenandoah Biotechnology manufactures growth factors, cytokines, and other recombinant protein factors to support the development and manufacture of cell and gene therapies.