The US FDA has granted accelerated approval to Kresladi (marnetegragene autotemcel; RP-L201), an autologous hematopoietic stem cell-based gene therapy developed by Rocket Pharmaceuticals for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic variants in the ITGB2 gene. The approval makes Kresladi the first gene therapy cleared by the FDA for this ultra-rare primary immunodeficiency. The agency also granted Rocket a Rare Pediatric Disease Priority Review Vo

-- Financing led by Access Biotechnology with participation from new and existing investors -- Financing to support advancement of two next-generation PI3Kα pan-mutant inhibitors for breast cancer and vascular anomalies – expect to for each program in 1H 2027 -- Liam Ratcliffe Joins OnKure’s Board of Directors March 27, 2026 -- OnKure Therapeutics, Inc. (Nasdaq: OKUR), a clinical-stage biopharmaceutical company focused on developing novel precision medicines, today announced that it has entere

March 27, 2026 -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, announced today that it has entered into a securities purchase agreement to sell 5,338,078 shares of its common stock at a price of $14.05 per share in a private placement. Vor Bio anticipates the gross proceeds from the private placement to be approximately $75 million, before deducting any offering-related expenses. Vor Bio did not engage a placement agent in conn

Simulations Plus has announced strategic partnership programmes with three pharmaceutical businesses to advance AI-enabled modelling in drug development. The partnerships aim to apply AI within scientifically validated modelling workflows and define scalable, next-generation approaches throughout the drug development life cycle. The programmes will use Simulations Plus software platforms, including ADMET Predictor, GastroPlus, Thales, and MonolixSuite. By working closely with these pharmaceut

FDA grants Orphan Drug Designation to SignaBlok's first-in-class, new mechanism-based TREM-1 peptide inhibitor to treat retinopathy of prematurity March 26, 2026 -- SignaBlok, Inc., a preclinical stage biotechnology company pioneering novel, first-in-class peptide therapies for multiple inflammation-associated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s TREM-1 peptide inhibitor for the treatment of retinopathy of

Data highlights efficacy and safety of risankizumab in psoriatic disease, including high impact areas in psoriasis and long-term treatment of psoriatic arthritis Data includes real-world evidence of minimal disease activity and clinical long-term safety outcomes of upadacitinib in atopic dermatitis, as well as investigational Phase 3 data for vitiligo and alopecia areata March 27, 2026 -- AbbVie (NYSE: ABBV) today announced new research across its dermatology portfolio to be presented at the 20

While not reaching statistical significance, there was a slowing of disease progression relative to placebo, as measured by IBMFRS, in the overall patient population at the lower dose of ulviprubart, with a 50% slowing of disease progression relative to placebo across both doses in a pre-specified subgroup of patients with less severe disease Ulviprubart demonstrated a favorable safety and tolerability pro with placebo in the study Study validates ulviprubart’s mechanism of targeted depletion

Beam Therapeutics reported updated clinical data from its ongoing Phase I/II trial of BEAM-302, a lipid-nanoparticle-delivered base editing therapy under development for alpha-1 antitrypsin deficiency. According to the firm’s press release, a single 60 mg dose raised levels of the protective protein alpha-1 antitrypsin (AAT) above the threshold associated with lung protection in all patients. Mean levels reached 16.1 µM and were sustained across five to twelve months of follow-up, supporting sel

– Expands Otsuka portfolio in psychiatric and neurological fields and aims to accelerate development of Transcend's portfolio, including the TSND-201 program for post-traumatic stress disorder (PTSD) – March 27, 2026 -- Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Transcend Therapeutics, Inc. (Transcend) announce that the two companies entered into an agreement today, under which Otsuka, through its wholly owned subsidiary Otsuka America, Inc., will fully acquire Transcend. The acquisition is e

The US FDA has approved Opdivo in combination with doxorubicin, vinblastine and dacarbazine (AVD) to treat patients 12 years and older with previously untreated stage 3 or 4 cHL. In the EU, approval has been granted by the European Commission (EC) for Opdivo in combination with brentuximab vedotin to treat patients between five and 30 years of age with relapsed or refractory cHL who have already received one prior line of therapy. SWOG 1826, a phase 3 study on which the US FDA based its approv