With $116 million in new funding on tap, BrainChild Bio said Tuesday that it has initiated a pivotal study of the B7-H3-targeted autologous CAR-T cell therapy BCB-276, in the hopes of delivering a much-needed new treatment option for patients with diffuse intrinsic pontine glioma (DIPG). With the current standard-of-care treatment for DIPG limited to palliative focal radiation therapy, median overall survival for this rare and aggressive paediatric brainstem tumour is about 11 months from diagn

Having recently secured FDA approval for the first in a new class of immunomodulatory drugs for multiple myeloma, Bristol Myers Squibb continues to make advances in the field, announcing Tuesday positive data from a registrational trial of its GPRC5D-directed CAR-T cell therapy arlocabtagene autoleucel (arlo-cel). The results come from the Phase II QUINTESSENTIAL study, which investigated arlo-cel in patients with quadruple-class exposed relapsed and refractory multiple myeloma (RRMM). BMS note

Phase 3 study will evaluate safety and efficacy of an investigational once-daily formulation of trientine tetrahydrochloride compared to D-penicillamine as first line treatment in patients with Wilson disease. Sept. 8, 2026 -- Orphalan Inc. today announced the launch of the TRADITiONAL Study, a global, multicenter Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first line treatment of individuals with Wilson disease. The study

HARBOR did not meet its primary endpoint of vHOT in DM1, a progressive neuromuscular disease with high unmet need and no approved treatment options Del-desiran showed evidence of clinical activity in secondary endpoints and exploratory analyses; full HARBOR dataset to be analyzed and appropriate development path to be determined AOC pipeline continues to advance with FDA priority review granted for delpacibart zotadirsen in DMD44 and planned FDA meeting for delpacibart braxlosiran in FSHD, based

Inhibrx CEO Mark Lappe was particularly keen on INBRX-106’s performance in patients with HPV-positive disease and sees a nearly $4 billion near-term opportunity thanks to the Phase 2 results. Patients with head and neck cancer who took Inhibrx Biosciences’ OX40 receptor agonist in combination with Merck’s Keytruda responded at a higher rate than those who took Keytruda alone in a Phase 2 trial, clearing the way for the biotech to access what executives estimate is a $50 billion-plus market. IN

- This trial marks the fourth Phase I peptide study for Ascletis this year. - ASC36 oral tablets achieved absolute oral bioavailability of 6% to 8% at steady state in non-human primate (NHP) studies utilizing Ascletis' proprietary Peptide Oral Transport ENhancement Technology (POTENT). - ASC36 oral tablets reduced mean body weight up to 13.2% from baseline after once-daily dosing for seven days in NHPs. - ASC36 subcutaneous (SQ) injection demonstrated approximately 32% and 91% greater relative b

- Oral Presentation in Session VI: Hereditary Angioedema Sept. 4, 2026 -- Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a clinical-stage small interfering RNA (siRNA) therapeutics company, today announced that updated Phase II data from its ongoing BW-20805 study for hereditary angioedema (HAE) was selected for oral presentation at the Bradykinin Symposium 2026, being held on September 3-4, 2026 in Berlin, Germany. BW-20805 has shown impressive efficacy throughout the Phase II trial and da

The single global Phase 3 trial, ArMaDa3 (NCT07770828), is the first pivotal gene therapy trial in geographic atrophy (GA) U.S. Food and Drug Administration (FDA) granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) designation, providing enhanced agency engagement throughout development and eligibility for accelerated approval and priority review Phase 3 design fully aligned with FDA; Biologics License Application (BLA) filing anticipated in 2028 OCU410 is designed as a one-time subreti

Signatera demonstrates 100% positive predictive value for treatment response assessment, detects recurrence months earlier than imaging, and supports precise, individualized lymphoma management through serial ctDNA monitoring Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, today announced the publication of new data in Blood Neoplasia, validating the prognostic and predictive value of Signatera in patients with both newly diagnosed and relapsed/refractory (

Positive Toxicology Results From 7-Day Rat and Dog Studies Characterize Tolerability and Inform Dose Selection for Next Phase of IND-Enabling Program, Targeting Q1 2027 IND Submission Sept. 01, 2026 -- MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA) ("MIRA" or the "Company"), a clinical-stage pharmaceutical company developing novel oral small-molecule therapeutics, today announced positive results from completed 7-day dose-range finding toxicology and toxicokinetic studies of MIRA-55 in rats and dogs