Around the Helix: Gene and Cell Therapy Company Updates - January 19, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in gene and cell 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. GeneTherapyLive’s Around the Helix is your chance to catch up with the latest news in gene and cell therapies, including partnerships, pipeline updates, and more.
1. FDA Extends BLA Review for bluebird bio’s β-Thalassemia and CALD Gene Therapies
The FDA has extended the review dates for bluebird bio’s biologics license applications for its investigative gene therapies: betibeglogene autotemcelfor β-thalassemia and elivaldogene autotemcel for cerebral adrenoleukodystrophy to August 19, 2022, and September 16, 2022, respectively.
2. Celularity’s CYNK-101 Wins Fast Track Designation for HER2+ Gastric/GEJ Cancers
The FDA has granted fast track designation to CYNK-101, a genetically modified natural killer cell therapy being developed by Celularity, in combination with standard frontline chemotherapy, trastuzumab (Herceptin), and pembrolizumab (Keytruda) for patients with advanced HER2-positive gastric or gastroesophageal junction adenocarcinoma.
3. Gamida Cell on Track to Submit Omidubicel BLA in First Half of 2022
Gamida Cell is planning to complete their biologics license application for omidubicel, a cell therapy for bone marrow transplant in patients with hematologic malignancies, in the first half of 2022 following positive Type B meeting correspondence from the FDA. The FDA had previously requested additional manufacturing data from a phase 3 study.
4. REGENXBIO Initiates ASCENT Trial of RGX-314 Wet AMD Gene Therapy
ASCENT is the second of 3 pivotal trials of their subretinal gene therapy RGX-314 for the potential treatment of wet age-related macular degeneration. ATMOSPHERE (NCT04704921), the first trial to be initiated by REGENXBIO in collaboration with AbbVie, is currently active and screening patients.
5. Rubius Therapeutics Doses First Patient in Cell Therapy Trial for Solid Tumors
The phase 1/2 trial is assessing RTX-224, an allogeneic, cell therapy candidate expressing 4-1BBL and IL-12, for the potential treatment of relapsed/refractory or locally advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial carcinoma and triple-negative breast cancer.
6. Atara and Imeka Collaborating to Use Advanced Imaging in MS Cell Therapy Trial
Atara Biotherapeutics will use Imeka’s proprietary biomarker imaging technology in the phase 2 EMBOLD trial (NCT03283826) assessing ATA188 as a monotherapy in multiple sclerosis. Atara hopes to use the imaging technology to further analyze the effects of ATA188 on neuroinflammation and remyelination.
7. Mesoblast’s Cell Therapy Reduces Chronic Low Back Pain
Mesoblast’s allogeneic cell therapy rexlemestrocel-L (MPC-06-ID) reduced chronic low back pain associated with degenerative disc disease, according to 36-month data from their 404-patient phase 3 trial (NCT02412735). The cell therapy will be assessed in an additional phase 3 trial.
8. BlueRock Therapeutics Doses First Patient in Phase 1 Cell Therapy Trial in Parkinson Disease
The first patient in Canada has been dosed in a phase 1, open-label trial (NCT04802733) of MSK-DAO1, BlueRock’s pluripotent stem cell-derived dopaminergic neuron cell therapy in patients with advanced Parkinson disease.
9. Longeveron Publishes Trial Design for Frailty in Aging Cell Therapy
The phase 2b trial will assess Lomecel-B, a living cell therapy product, as a regenerative medicine for the potential treatment of aging frailty. The design and rationale of the study was published in the Journal of Frailty and Aging.
10. Tevogen Granted Patent for SARS-CoV-2 T-Cell Therapy Preparation
Tevogen Bio has been granted a patent for their method of preparing and manufacturing TVGN-489, a COVID-19 peptide specific cytotoxic T cell therapy. The cell therapy is designed to target the entire SARS-CoV-2 viral genome rather than only spike proteins.
11. Intellia Doses First Patient With ATTR Amyloidosis and Cardiomyopathy in New Gene Therapy Trial Cohort
The phase 1 trial (NCT04601051) of NTLA-2001, an investigational CRISPR gene editing therapy delivered with Intellia’s lipid nanoparticle technology for the treatment of ATTR amyloidosis, recently added a cardiomyopathy arm which has dosed its first patient. Intellia expects the trial, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy, to complete enrollment in 2022.
