Around the Helix: Cell and Gene Therapy Company Updates – August 17, 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. FDA Approves Beti-Cel Gene Therapy for β-Thalassemia

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).

2. Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports

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.

3. NK Cell Therapy Trial Doses First Patient With Lymphoma

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.

4. CRISPR Therapeutic Ready to Dose in Duchenne Muscular Dystrophy

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).

5. Frontotemporal Dementia Gene Therapy Trial Initiates Dosing

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).

6. ALS Cell Therapy Receives US Patent

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).

7. Pompe Disease mRNA Therapy Receives FDA Orphan Drug Designation

Aro Biotherapeutics’ ABX1100, an investigational Centyrin-siRNA conjugate for the treatment of Pompe disease, has received orphan drug designation from the FDA.

8. ProQR Pivots From Ophthalmic to CNS, Liver RNA Therapies

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).

9. Regen Files for Patent on CAR T-Cell Exhaustion Prevention Technology

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.

10. WuXi ATU to License TESSA AAV Manufacturing Technology to Janssen

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.

11. Timothy D. Hunt Appointed CEO of the Allience for Regenerative Medicine

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.

12. Prescient Therapeutics, Q-Gen Cell Enter 5-Year Production Agreement

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.

13. AGC Biologics Announces Partnership With RoosterBio Focused on Accelerating Manufacture of Cell and Exosome Therapies

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.

14. Pedro Huertas, MD, PhD, Appointed CMO of Kriya Therapeutics' Rare Disease Division

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.