NATIONAL INSTITUTES OF HEALTH

Sinopia Biosciences awarded $2.2M Phase II SBIR grant from NIDCR to advance oral mucositis program using LEADS® platform, combining AI/ML and high-throughput screening for therapeutic target identification.

Aura Biosciences' new therapy, bel-sar, for early-stage choroidal melanoma shows promise in Phase II trials, potentially offering a vision-preserving alternative to radiation therapy. The treatment uses virus-drug conjugates activated by light to target tumors, with results indicating high efficacy and safety. Aura plans to advance to Phase III trials and explore broader cancer applications.

Ractigen Therapeutics receives FDA orphan drug designation for RAG-18, a saRNA for DMD/Becker muscular dystrophy. Biogen's nusinersen shows higher dose efficacy in SMA. Cartesian Therapeutics initiates Descartes-15 CAR-T phase 1 trial for r/r multiple myeloma. Imugene's azer-cel CAR-T produces complete responses in r/r diffuse large B cell lymphoma. Novartis licenses Voyager Therapeutics' capsid for neurological target. BridgeBio Pharma's BBP-812 receives FDA RMAT designation for Canavan disease. Genprex plans to spin off its diabetes program into NewCo. NCI awards TransCode $2 million for TTX-MC138 RNA therapy in advanced solid tumors.

Kintara Therapeutics provides corporate and REM-001 clinical study updates, including a merger agreement with TuHURA Biosciences and a special stockholder meeting on September 20, 2024. The REM-001 study for cutaneous metastatic breast cancer has enrolled four of ten needed patients, with no safety issues identified.

UC Davis' external research awards in FY 2023-24 exceeded $1 billion, with the School of Medicine receiving $400 million. Federal funding was $441 million, state funding $237 million, and industry funding $95 million. NIH remains the top federal funder, supporting projects like Alzheimer's research and public health emergency preparedness. Industry partnerships fund clinical trials, and UC Davis research produced 149 inventions, 208 patent applications, and 109 patents.

Researchers led by Marija Drndić and Dimitri Monos have developed a dual-layer nanopore system (GURU) that enhances DNA sequencing and protein identification by providing precise control and detection of molecules. This platform, eliminating the need for proteins, offers improved accuracy and longer operational lifespans, potentially revolutionizing sequencing technologies for complex genomic regions like HLA genes.

Technological advances have led to the development of ultra-sensitive blood-based biomarkers for Alzheimer’s disease, offering potential for early, inexpensive diagnosis and follow-up, though challenges remain before deployment in routine clinical care.

The Duffy-null phenotype causes lower blood neutrophil levels due to their presence in other tissues. Clinical trials often exclude Duffy-null patients, even with normal neutrophil counts, leading to potential health inequity. Researchers recommend testing for the phenotype and adjusting trial criteria to include these patients.

Columbia University-led IndiPHARM project, funded by $39.5M from ARPA-H, aims to develop a platform measuring drugs, metabolites, and environmental factors to optimize therapeutic efficacy, reducing adverse drug events and improving patient care.