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Regulatory approvals – gene therapy for ultra-rare immune deficiency
The FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), the first gene therapy option...
Regulatory – EMA moves to withdraw mpox label
The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended withdrawing the mpox indication for Siga Technologies’ tecovirimat, while keeping approval for smallpox, cowpox, and...
Drug approvals – Novo Nordisk’s first once-weekly basal insulin in the US
The FDA approved Novo Nordisk’s Awiqli (insulin icodec) injection 700 units/mL, making it the first and only once-weekly, long-acting basal insulin in the US. The approval gives Type 2 diabetes...
M&A – Otsuka to acquire Transcend for a PTSD-focused neuroplasticity program
Otsuka is moving to buy Transcend Therapeutics for about $700 million upfront (roughly $1.2 billion total deal value), positioning the Japanese drugmaker to enter or expand psychiatric pipelines...
M&A – Novartis pays up to $2B for allergy biotech Excellergy
Novartis agreed to acquire Excellergy in a deal valued at up to $2 billion, bringing a next-generation anti-IgE program into the Swiss pharma’s allergy portfolio. Excellergy’s lead, Exl-111, is an...
Clinical and pipeline signal – AstraZeneca’s tozorakimab posts Phase 3 COPD wins
AstraZeneca reported positive Phase 3 results for tozorakimab in chronic obstructive pulmonary disease (COPD), saying the antibody met primary goals in two trials (Oberon and Titania). The company...
Oncology – Wave Life Sciences sinks on obesity data linked to INHBE targeting
Wave Life Sciences took a sharp market hit after obesity trial readouts suggested limited incremental efficacy at higher doses of WVE-007, an INHBE-targeted therapy. The reported mRNA expression...
Precision medicine – UCLA ATLAS biobank ties genetics to ancestry-specific disease risk and semaglutide response
Investigators at UCLA used the UCLA ATLAS Community Health Initiative biobank to map genotype–phenotype associations across ancestries and identify pharmacogenomic signals tied to semaglutide...
Biomedical innovation – implantable “living pharmacy” devices produce multiple drugs in vivo
A multi-institutional team led by Northwestern University and collaborating groups reported progress toward implantable “living pharmacies” that manufacture multiple medicines inside the body. In...
Diagnostics – Maryland awards $520K to expand multi-cancer early detection screening with 20/20 BioLabs assay
Maryland’s Department of Health awarded more than $520,000 through a firefighter grant program to expand multi-cancer early detection (MCED) screening using 20/20 BioLabs’ OneTest assay. The...
Big Pharma acquisitions reshape near-term pipeline bets
Novartis moved to shore up its allergy franchise with an acquisition of Excellergy in a deal worth up to $2 billion, bringing the startup’s next-generation anti-IgE program under the Swiss...
FDA approvals—rare disease gene therapy and next-gen diabetes care
The FDA cleared Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel) as the first gene therapy for severe leukocyte adhesion deficiency type I (LAD-I), granting accelerated approval for...
EMA CHMP backs new oncology and label expansions
The EMA’s CHMP recommended four new medicines in March, including opinions covering oncology therapies and label updates. CHMP also recommended two FDA-approved therapies for extended-stage small...
CAR-T and advanced cell therapy—risk, safety, and platform direction
AstraZeneca’s in vivo CAR-T program continues to generate early clinical signals as new data from a China trial show cancer responses in a subset of patients, but the dataset includes a death that...
Oncology diagnostics—molecular testing improves bile duct cancer detection
A University of Pittsburgh-led team reported that BiliSeq, a 28-gene next-generation sequencing panel for bile duct tissue, meaningfully improves bile duct cancer detection compared with pathology...
Novel therapeutics biology—gut-immune mechanisms in multiple sclerosis
Keio University researchers linked intestinal epithelial cells to neuroinflammation in multiple sclerosis, reporting a mechanism in which IECs promote pathogenic TH17 T cell development that...
Implantable drug delivery—multi-drug “living pharmacy” platform advances in rodents
Northwestern University and collaborators reported progress toward implantable “living pharmacies,” engineering cells encapsulated in a wireless, fully implantable device designed to continuously...
U.S. real-world screening expansion—multi-cancer early detection for firefighters in Maryland
Maryland’s Department of Health awarded more than $520,000 to 18 fire departments to run multi-cancer early detection screening using 20/20 BioLabs’ OneTest blood assay. The funding supports...
Robotic and AI-assisted medicine—stroke telesurgery infrastructure
Xcath Robotics reported ongoing momentum for its Iris robotic platform, positioning telesurgery as a near-term pathway for expanding stroke treatment beyond specialized centers. CEO Eduardo...
Biotech R&D finance—Pinnacle’s funding and Wave’s obesity setback
Pinnacle Medicines raised $89 million in a Series B to advance its oral peptide programs, according to deal terms that bring total financing to $134 million. The round was co-led by LAV and...