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

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

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

Key Highlights: Excellent Safety Profile: As of mid-March 2026, 113 heavily pretreated patients with solid tumors have been enrolled in the Phase I trial of CS2009, with a median follow-up of approximately 6 months. The more mature data continue to show a favorable safety profile, with 23% incidence of Grade ≥3 Treatment-Related Adverse Events (TRAEs). No excessive toxicities that typically occurred in combination therapies containing CTLA-4 and PD-(L)1 were observed, and the incidence of Grade

78-Week Study Evaluating Subcutaneous VK2735 in Adults with Obesity and Type 2 Diabetes March 26, 2026 -- Viking Therapeutics, Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced the completion of patient enrollment in its Phase 3 VANQUISH-2 clinical trial of subcutaneous VK2735, the company's dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulino

First patient dosed in Opti-GAIN, a first-in-human Phase I/II clinical trial of CTx001 in Geographic Atrophy secondary to AMD CTx001 is an investigational AAV-based gene therapy designed to deliver mini-CR1, a potent modulator of multiple pathways of the complement system Opti-GAIN is supported by Pre-GAIN, an ongoing natural history study designed to provide short-term GA progression insights and support the development of novel endpoints March 25, 2026 -- Complement Therapeutics GmbH (CTx), a

BGM0504 shown to be generally well tolerated Overall safety, pk, and efficacy profile supports once-daily oral dosing March 25, 2026 -- BrightGene Bio-Medical Technology Co., Ltd. (SSE: 688166, "BrightGene" or "the Company") today announced positive topline results from Phase 1 clinical studies of BGM0504, an investigational oral GLP-1/GIP dual receptor agonist, in adult participants in China and the United States, including healthy volunteers and individuals with overweight or obesity. Across

GLIX1, a TET2 activator, is a first-in-class, oral, small molecule targeting DNA damage response in glioblastoma and other cancers GLIX1 has demonstrated potent anti-tumor activity in multiple GBM models, excellent blood-brain barrier penetration and a favorable safety profile in preclinical toxicology studies Study is being conducted by world-leading glioblastoma investigators, led by Dr. Roger Stupp March 26, 2026 -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical development stage bio

Application submitted for manufacturing and marketing approval of ripretinib in Japan for the indication of “gastrointestinal stromal tumor that has progressed following cancer chemotherapy” Ripretinib has significantly prolonged the primary endpoint of progression-free survival compared to placebo in the INVICTUS study Already approved in the United States and Europe; recognized as a highly necessary medicine by the government review committee addressing drug loss in Japan Ono Pharmaceutical C