Phase 1 Trial

Nanobiotix (NASDAQ: NBTX) has announced the publication of a peer-reviewed case study in Clinical and Translational Radiation Oncology.  The case study reports on the first patient treatment experience with potential first-in-class radioenhancer NBTXR3 in pancreatic ductal...

Regenxbio Inc (NASDAQ: RGNX) announced additional interim data from Cohorts 1-3 of Phase 1/2 trial of RGX-121 for patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II),  The data were shared at the 18th Annual WORLDSymposium.   RGX-121 is an...

IGM Biosciences Inc (NASDAQ: IGMS) has progressed two Phase 1 trials evaluating IGM-6268, an anti-SARS-CoV-2 IgM monoclonal antibody, to treat and prevent infection COVID-19.  The first Phase 1 trial is a single (SAD) and multiple (MAD) ascending dose study to assess IGM-6268...

4D Molecular Therapeutics Inc (NASDAQ: FDMT) has posted updated interim data from the Phase 1/2 trial of 4D-310 in Fabry disease at the 18th Annual WORLDSymposium. Following 4D-310 infusion, mean serum AGA enzyme activity was within, or significantly above, the normal range in all three...

Avrobio Inc's (NASDAQ: AVRO) gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion. The data comes from a Phase 1/2 trial testing the safety and efficacy of AVR-RD-04 for cystinosis who had...

CAR-T company Arcellx Inc (NASDAQ: ACLX) priced its IPO at $15 per share, raising $123.8 million at the low end of the Company’s proposed range of $15 - $17. The Company offered 8.3 million shares during the initial public offering. Arcellx's lead ddCAR candidate,...

The FDA has signed off Pardes Biosciences Inc's (NASDAQ: PRDS) Investigational New Drug (IND) application for PBI-0451 for COVID-19. Pending further engagement with FDA and other regulators, the Company anticipates initiating global Phase 2/3 studies of PBI-0451 in SARS-CoV-2...

The FDA has signed off Viridian Therapeutics Inc's (NASDAQ: VRDN) investigational new drug (IND) application of VRDN-002.  The Company's next-generation IGF-1R antibody, VRDN-002, is a humanized monoclonal antibody that incorporates half-life extension technology and is...

Taysha Gene Therapies Inc (NASDAQ: TSHA) reported initial serum β-hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, two forms of GM2 gangliosidosis.  According to Taysha, TSHA-101 is the first bicistronic vector in...

4D pharma plc (NASDAQ: LBPS) has announced additional clinical data from Part A of its Phase 1/2 trial of MRx-4DP0004, an orally-delivered single strain Live Biotherapeutic for asthma.  As previously announced, Part A met the primary endpoint, and the safety profile of MRx...

China's National Medical Products Administration (NMPA) has signed off SELLAS Life Sciences Group Inc's (NASDAQ: SLS) IND application to initiate the first clinical trial for 3D189 in patients with hematological malignancies. 3D189, also known as SELLAS' lead product...

Vaccinex Inc (NASDAQ: VCNX) shares are gaining after the Company reported interim response data in the Phase Ib segment of the KEYNOTE-B84 study. The trial is evaluating Vaccinex's pepinemab combined with Merck & Co Inc's (NYSE: MRK) Keytruda (pembrolizumab) in...

The Alzheimer's Drug Discovery Foundation (ADDF) and The Association for Frontotemporal Degeneration (AFTD) will support Wave Life Sciences Ltd's (NASDAQ: WVE) amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD) program. The organizations will...

89bio Inc (NASDAQ: ETNB) announced topline results from a cohort of 20 patients (Cohort 7) in the Phase 1b/2a proof-of-concept study evaluating pegozafermin (formerly BIO89-100) for nonalcoholic steatohepatitis (NASH). The results show that 63% of patients achieved a two-point or...

The FDA has cleared Tango Therapeutics Inc's (NASDAQ: TNGX) Investigational New Drug (IND) application for its lead program, TNG908. TNG908 is a synthetic lethal small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5) designed to kill cancer cells with a...