NEW YORK – MD Anderson Cancer Center said this week it launched a new research organization, the Institute for Cell Therapy Discovery and Innovation, to develop cell therapies for cancer, autoimmune diseases, and infections. The Institute will be supported by more than $80 million in philanthropic funding, much of which will be dedicated to clinical trials, and will seek to partner with external researchers as well as biotechnology and pharmaceutical companies to accelerate development of new therapies.
The US Food and Drug Administration has granted Genascence fast-track designation for GNSC-001, an investigational gene therapy it's developing as a treatment for osteoarthritis of the knee, the Palo Alto, California-based company said this week. GNSC-001, which the company said is the first gene therapy being tested in patients for a prevalent musculoskeletal disease, currently is being evaluated in a Phase I trial. A sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Seattle Children's Hospital last week said it started enrolling pediatric patients with systemic lupus erythematosus into a Phase I trial of an autologous CAR T-cell therapy, which it said will be the first clinical trial to investigate a CAR T-cell therapy in this pediatric patient population. Investigators plan to enroll 12 patients who are 2 to 30 years of age into the Phase I trial, known as the Reversing Autoimmunity through Cell Therapy, or REACT-01 study. The CAR T-cell therapy was developed by Seattle Children's Therapeutics, the hospital system's nonprofit therapeutics development arm.
Cleveland-based Abeona Therapeutics this week said that the US Food and Drug Administration has accepted for review a biologics license application (BLA) that the company resubmitted for prademagene zamikeracel (pz-cel), its investigational gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). Abeona initially submitted the BLA last year, but in April, the regulator requested additional chemistry, manufacturing, and controls (CMC) information. The FDA expects to issue a decision on the product by April 29, 2025.
Mount Sinai Health System said this week that it has been awarded nearly $7 million from the National Institutes of Health to build a coalition of academic medical centers and community partners in New York that plans to recruit more than 7,000 participants across the tristate area over the coming year for the NIH's All of Us Research Program. In addition, the coalition will seek to enroll 3,300 new participants with opioid use disorder. Collaborators include Weill Cornell Medicine, New York City Health + Hospitals, the Institute for Family Health, and NYU Langone.
Separately, the All of Us Research Program said this week that the State of California has committed $9.3 million over four years to extend engagement efforts for the project throughout California. Specifically, the California Initiative to Advance Precision Medicine (CIAPM), part of the California Health and Human Services (CalHHS) agency, has signed a memorandum of understanding with All of Us to support the program's engagement and enrollment efforts, with a focus on participation from underrepresented groups in biomedical research.
Oxford, UK-based AlveoGene this week announced that the US Food and Drug Administration has granted it rare pediatric disease designation for AVG-002, a gene therapy it's developing as a treatment for neonatal surfactant protein B (SP-B) deficiency, a rare monogenic disorder that leads to fatal respiratory distress syndrome in newborns. A sponsor with an approved drug with this designation may qualify for a voucher that it can redeem to receive priority review for a different product, or transfer or sell the voucher to another sponsor. AlveoGene, which launched last year to advance inhaled gene therapies for rare respiratory conditions, said that preparations for clinical development of AVG-002 are underway and that it envisions it could file for marketing authorization by 2028.
Solvd Health said this week that it has received PMA supplement approval from the US Food and Drug Administration for use of its Infiniti High Throughput System (HTS) with its AvertD genetic test for assessing risk of developing opioid use disorder. HTS is a semi-automated, scalable, multiplexed microarray analysis system that increases throughput to 960 samples in approximately 8 hours compared to 48 samples on the existing Infiniti Plus.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.