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Rare disease gene therapy reaches FDA approval
Rocket Pharmaceuticals received FDA approval for Kresladi, its gene therapy for leukocyte-adhesion deficiency type 1 (LAD1), an ultra-rare inherited immunodeficiency that can be fatal in infants...
Large-lung disease readouts strengthen late-stage lung pipeline
United Therapeutics’ Tyvaso cleared another key late-stage milestone in idiopathic pulmonary fibrosis after a second Phase III trial hit its endpoints. The company is positioning for a potential...
Immuno-oncology platform advances into clinical positioning
Memorial Sloan Kettering researchers engineered a new uPAR-directed CAR T-cell therapy intended to expand cell therapy targeting beyond conventional antigens in solid tumors. The work centers on...
Biotech M&A signals continued deal intensity
Novartis moved to expand its allergy and immunology pipeline by acquiring Excellergy. The purchase targets Exl-111, an anti-IgE antibody currently in Phase I testing, with potential applications...
Clinical trial signal in rare thyroid eye disease shifts investor view
Viridian Therapeutics disclosed top-line Phase III results for elegrobart (VRDN-003), an insulin-like growth factor 1 receptor (IGF-1R) monoclonal antibody in active thyroid eye disease. Patients...
Laboratory automation expands with FDA clearance
Roche secured FDA 510(k) clearance for two Cobas Pro Integrated Solutions lab automation units—Cobas c 703 and Cobas ISE Neo—aimed at increasing throughput and reducing workflow friction in...
Clinical diagnostics automation and genomic medicine infrastructure
Canada’s PhenoTips partnered with five clinical institutions to launch the CIPHER network, an initiative funded with C$5.4 million from Genome Canada to accelerate AI genomic medicine. The program...
Clinical outsourcing optimization for trial execution
ICON teamed with Advarra to create an integrated, AI-driven site network model aimed at reducing clinical trial start-up delays. The partnership connects ICON’s trial services operations with...
Obesity GLP-1/GIP pipeline funding via IPO
Kailera Therapeutics moved to the public markets, filing plans for an IPO to fund its obesity portfolio, led by weekly GLP-1/GIP agonist ribupatide (KAI-9531). The Boston-based biotech, backed by...
AI drug discovery deal expands Lilly’s pipeline
Eli Lilly expanded its AI drug discovery collaboration with Insilico Medicine, signing a deal that could total $2.75 billion in development, regulatory, and commercial milestones. Lilly will pay...
FDA accelerates oncology cell therapy pathway with Breakthrough designation
Washington University School of Medicine in St. Louis announced that the U.S. FDA granted Breakthrough Therapy Designation to a CAR-T cell treatment targeting uPAR for solid tumors. The therapy...
Company acquisition expands immune/inflammation and allergy assets
Novartis agreed to acquire Excellergy, adding an anti-IgE antibody program (Exl-111) currently in Phase I testing. The deal broadens Novartis’ immune and inflammation pipeline, with Exl-111...
UK and clinical trial operations: ICON teams with Advarra on integrated site network
ICON partnered with institutional review board and clinical trial management provider Advarra to build an integrated, AI-driven clinical site network model aimed at speeding study startup. The...
Biopharma labor and restructuring pressure: Takeda layoffs in Cambridge
Takeda announced plans to lay off nearly 250 workers at its Cambridge, Massachusetts site, beginning in July, after a board-approved cost-saving plan approved March 25. The workforce reduction is...
Autoimmune eye disease: Viridian’s elegrobart Phase 3 wins but investors demand more
Viridian Therapeutics disclosed topline Phase 3 results for elegrobart (formerly VRDN-003) in active thyroid eye disease, reporting that the subcutaneous, half-life-extended IGF-1R antibody met a...
Idiopathic pulmonary fibrosis: United’s Tyvaso hits endpoints in second Phase 3 trial
United Therapeutics said its second Phase 3 trial of Tyvaso (treprostinil) for idiopathic pulmonary fibrosis met endpoints, strengthening the company’s case for a supplemental NDA and potential...
Cardiovascular med device: Boston Scientific Watchman FLX trial supports stroke reduction profile
Boston Scientific presented data from the Champion-AF trial showing that Watchman FLX provided superior protection from bleeding compared with non-vitamin K oral anticoagulants in atrial...
Private capital: Blackstone closes record $6.3B life sciences fund
Blackstone announced it closed its Blackstone Life Sciences VI fund at $6.3 billion, the largest life sciences private investment vehicle it has raised. The new fund is nearly 40% larger than the...
Gene therapy regulatory milestone
Rocket Pharmaceuticals secured FDA approval for Kresladi, its gene therapy for leukocyte-adhesion deficiency type 1 (LAD-1). The company said the approval makes Kresladi the first gene therapy...
Clinical success in cardiology and device-therapy performance
United Therapeutics’ Tyvaso (treprostinil) hit endpoints in a second phase 3 idiopathic pulmonary fibrosis (IPF) study, strengthening its case for a supplemental NDA review. The trial reportedly...