NEW YORK – Charlestown, Massachusetts-based Solid Biosciences this week said it has received rare pediatric disease designation from the US Food and Drug Administration for SGT-003, its gene therapy candidate for Duchenne muscular dystrophy. That adds to orphan drug and fast-track designations that the firm has already received from the FDA for the gene therapy, for which Solid expects to begin dosing patients in a Phase I/II trial in Q2.
2seventy Bio said this week that it has completed a deal to sell its oncology and autoimmune research and development programs to Regeneron Pharmaceuticals. 2seventy first announced the deal in January, sharing that it would become a single-product company focused exclusively on its autologous CAR T-cell therapy Abecma (idecabtagene vicleucel), which it comarkets with Bristol Myers Squibb for certain multiple myeloma patients.
SimBioSys said this week that it has partnered with biotech incubator General Inception to make its PhenoScope tumor phenotyping platform available to General Inception’s portfolio of over 40 early-stage oncology drug development companies.
Jazz Pharmaceuticals said this week that it has completed the rolling submission of a biologics license application (BLA) to the US Food and Drug Administration seeking accelerated approval for its HER2-targeted bispecific antibody zanidatamab as a second-line treatment for advanced or metastatic HER2-positive biliary tract cancer. The BLA includes data from the Phase IIb HERIZON-BTC-01 trial, in which zanidatamab demonstrated an overall response rate of 41.3 percent in HER2-positive biliary tract cancer.
New York City-based Myrtelle this week said it has received regenerative medicine advanced therapy designation from the US Food and Drug Administration for its lead gene therapy candidate, rAAV-Olig001-ASPA, for the treatment of Canavan disease, a childhood genetic brain disease caused by mutations in the ASPA gene. Myrtelle has also been granted orphan drug, rare pediatric disease, and fast-track designations from the FDA and orphan drug designation and advanced therapy medicinal product classification from the European Medicines Agency and is part of the UK Medicines and Healthcare Products Regulatory Agency's innovative licensing and access pathway.
Novigenix said this week it has partnered with the IMMUcan consortium to support the development of RNA sequencing biomarkers that can advance the understanding of how the immune system responds to checkpoint inhibitors in cancer patients. The IMMUcan consortium joins academic, patient, and industry partners that aim to generate real-world evidence to better understand interactions between the immune system and tumors and the effect of therapeutic interventions on tumors. Novigenix has an artificial intelligence-driven RNA platform that can map systemic immune responses, generate individual immune response profiles, and tailor treatments to those profiles.
Context Therapeutics said this week that, on March 28, it submitted an investigational new drug application to the US Food and Drug Application seeking clearance to begin its first-in-human study of its bispecific antibody, CTIM-76. The Phase I dose escalation and expansion trial will involve patients with Claudin 6 (CLDN6)-positive gynecologic and testicular cancers.
Standard BioTools said this week that it has inked a deal through 2026 with Bristol Myers Squibb for use of its SomaScan Platform in clinical trials of therapies in multiple disease areas. BMS has previously used SomaScan to support research in immunology, cardiovascular disease, and pulmonary fibrosis. The SomaScan platform uses proprietary aptamers called Somamers to measure up to 11,000 proteins in biofluid samples.
Viome said this week that it inked a distribution deal with Henry Schein for its Oral Health Pro With CancerDetect laboratory-developed test for early biomarkers of oral and throat cancers. Dental professionals can now purchase Viome’s collection kits directly from Henry Schein, which will ship them to the dental practice, where saliva samples will be collected and registered with Viome online, then sent to Viome’s CLIA lab for testing.
Sophia Genetics and Bangalore, India-based Strand Life Sciences said this week they have entered into a global strategic partnership to provide technologies and services to companies in the precision medicine space. Under the partnership, Sophia will provide support via its decentralized Sophia DDM multimodal data-analysis platform and Strand via its curated variant databases and bioinformatics solutions expertise. The companies also said that they will codevelop tests, among other initiatives.
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.