Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
The FDA has approved bluebird bio’s gene therapy betibeglogene autotemcel (beti-cel), now marketed as ZYNTEGLO, for adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions (transfusion dependent thalassemia; TDT).
Two children with spinal muscular atrophy (SMA) have died after being treated with onasemnogene abeparvovec (Zolgensma; Novartis) from acute liver failure, a known safety risk of the therapy. New information revealed by Novartis and reported by Bloomberg indicates that 1 patient was outside of the FDA-approved max age for treatment that is indicated in the US; however, the patient's age and weight were in alignment with the approved indication in the country of treatment.
Gamida Cell has dosed the first patient in their company-sponsored phase 1/2 trial of the natural killer (NK) cell therapy GDA-201 for the potential treatment of follicular and diffuse large B cell lymphomas.
The FDA has given investigational new drug application (IND) clearance to Cure Rare Disease’s CRISPR therapeutic, CRD-TMH-001, for treating Duchenne muscular dystrophy (DMD).
Passage Bio has dosed the first patient with frontotemporal dementia with granulin mutations (FTD-GRN) with their gene therapy PBFT02 in the phase 1/2 upliFT-D study (NCT04747431).
Israeli cell therapy company Kadimastem has been granted a patent from the United States Patent and Trademark Office for AstroRx, a cell therapy consisting of astrocytes derived from human embryonic stem cells intended for the treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS).
Aro Biotherapeutics’ ABX1100, an investigational Centyrin-siRNA conjugate for the treatment of Pompe disease, has received orphan drug designation from the FDA.
ProQR has announced their intention to focus exclusively on their Axiomer RNA-editing technology platform after negative results and feedback on their phase 2/3 ILLUMINATE trial (NCT03913143) of sepofarsen for treating Leber congenital amaurosis 10 (LCA10).
Regen BioPharma is filing for a patent for its technique of preventing T-cell exhaustion to create more durable and efficacious chimeric antigen receptor (CAR) T-cell therapies against solid tumors.
The TESSA system is capable of producing 10 times more adeno-associated viral (AAV) vectors compared to traditional AAV manufacturing techniques. The licensing agreement will also allow Janssenn to work on WuXi's proprietary clonal suspension HEK293 cell line.
Hunt's previous experience includes being chief culture and corporate affairs officer at Xilio Therapeutics, chief corporate affairs officer at Editas Medicine, and holding executive public affairs roles at Cubist Pharmaceuticals and Biogen. He also served as an Advisory Group member for the Value-Based Payments for Medical Products consortium at the Duke-Margolis Center for Health Policy and as a member of the Board of Directors of Life Science Cares.
Q-Gen Cell will manufacture Prescient's OmniCAR cell lines, which are designed to express a universal immune receptor called SpyCatcher which binds with targeting ligands, an approach which could potentially allow for the targeting of any cancer antigen.
The partnership will utilize AGC Biologics' global manufacturing capabilities and RoosterBio's process development services to manufacture human mesenchymal stem/stromal cells (hMSCs) and exosome therapeutics for clients.
Huertas' prior experience includes serving as the chief medical officer for several companies, including Inozyme Pharma, Sentien Biotechnologies, and Eloxx Pharmaceuticals. He also previously worked in clinical and development roles for Pfizer, Shire, Amicus, and Genzyme Corp.