Following 100% transfusion independence in Chinese TDT patients, new study confirms tBE is equally safe and effective for African SCD patients and TDT patients from South/Southeast Asia. Sept. 7, 2026 -- On September 7, 2026, Shanghai, Cell Stem Cell published online clinical research from CorrectSequence Therapeutics (Correctseq) in collaboration with multiple institutions in a paper titled "Clinical base editing for β-hemoglobinopathies across different genetic backgrounds", demonstrating tha

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

Pfizer has found a new home for an early-stage antibody-drug conjugate (ADC) it gained through its $43-billion takeout of Seagen — then subsequently binned — according to a US securities filing from Medicus Pharma on Wednesday. In March, the pharma terminated a Phase I trial of PF-08046031 (SGN-CD228A) in patients with melanoma and other solid tumours, citing "strategic reasons." The melanotransferrin (CD228)–targeting candidate is one of more than a half-dozen Seagen-sourced ADCs that Pfizer h

US-based biotechnology company Superluminal Medicines has completed a $60m Series B financing round to support its research into therapies for rare genetic forms of obesity and hypothalamic obesity. The funding was led by BVF Partners, with participation from new investors Deep Track Capital and Perceptive Advisors. Existing backers included Catalio Capital Management, Cooley, Eli Lilly and Company, Gaingels, Insight Partners, Nvidia, and RA Capital Management. The company plans to use the pr

GSK is spending up to $1.3 billion on a first-of-its-kind cancer therapy slated to enter the clinic this year. In a deal announced Thursday, GSK agreed to pay Hutchmed $110 million up front, and potentially roughly $1.19 billion more in milestone payments and royalties, for a majority of the rights to a treatment known as HMPL-A830. That treatment is what Hutchmed refers to as an “antibody-targeted therapeutic conjugate,” and could potentially address multiple tumor types. Typical antibody-dru

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 (