Cell therapy weekly: AstraZeneca to acquire Neogene Therapeutics
This week: AstraZeneca to acquire Neogene Therapeutics, FDA awards Orphan Drug Designation to gene therapy for otoferlin gene-mediated hearing loss and Eikonoklastes Therapeutics partners with Forge Biologics to progress gene therapy for ALS to clinical trial.
The news highlights:
AstraZeneca to acquire Neogene Therapeutics
Neogene Therapeutics (Amsterdam, The Netherlands), a clinical-stage biotechnology company developing T-cell receptor therapies for solid tumors, has entered into a definitive agreement to be acquired by AstraZeneca (Cambridge, UK) for a total consideration of US$320 million.
Carsten Linnemann, CEO of Neogene, stated: “We are excited to work together with AstraZeneca towards our shared mission of transforming the treatment options for patients with solid tumors using next-generation T-cell receptor therapies. Our expertise, clinical portfolio, and platform technologies in this area combined with AstraZeneca’s leadership in oncology and global footprint means we are well-positioned to translate pioneering science into novel treatments for hard-to-treat cancers.”
FDA awards Orphan Drug Designation to gene therapy for otoferlin gene-mediated hearing loss
The FDA has awarded Orphan Drug Designation to OTOF-GT, a gene therapy program for the treatment of patients with otoferlin gene-mediated hearing loss, one of the most common forms of congenital deafness. OTOF-GT is the lead gene therapy program of Sensorion (Montpellier, France) and utilizes a dual adeno-associated viral vector system to deliver transgenes encoding OTOF to the inner hair cells of the cochlea.
Géraldine Honnet, Chief Medical Officer of Sensorion stated: “We are really pleased to have received this significant regulatory feedback from the FDA, following the agency’s recent award of Rare Pediatric Disease designation for OTOF-GT. Orphan Drug Designation will support us in advancing our development program, a gene therapy that offers the potential to help patients with a condition for which there are currently no approved curative therapies. We are excited to have achieved this milestone and remain highly focused on the development of our most promising candidates to produce life-changing therapies to restore, treat and prevent hearing loss disorders.”
Eikonoklastes Therapeutics partners with Forge Biologics to progress gene therapy for ALS to clinical trial
Eikonoklastes Therapeutics and Forge Biologics (both OH, USA) have entered a manufacturing partnership to advance Eikonolastes’ AAV-based gene therapy treatment for sporadic and familial amyotrophic lateral sclerosis (ALS), ET-101, into clinical trials. Under the terms of the partnership, Forge will provide AAV process and development manufacturing services.
CEO, and co-founder of Eikonoklastes Therapeutics, Bruce Halpryn, stated: “We are excited to advance ET-101, our novel treatment for neurodegenerative diseases, towards a first-in-human clinical trial in ALS patients. Partnering our AAV manufacturing with Forge will further our ability to advance into clinical trials. With their end-to-end platform supporting AAV therapies, Forge is accelerating our development to reach patients suffering from ALS.”
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