Sanofi announces promising late-stage results for its immunology candidate amlitelimab and expands into genetic therapies via an investment in Sensorion, signaling a strategic push into high-value therapeutic areas.
Cogent Biosciences secures a key FDA Breakthrough Therapy Designation for its bezuclastinib-sunitinib combination in advanced GIST, following promising Phase 3 data that showed a halving of disease progression risk.
New long-term results from the pivotal EMBARK trial indicate ELEVIDYS, Sarepta's gene therapy, continues to alter the disease trajectory for boys with Duchenne muscular dystrophy, with no new safety concerns emerging over three years.
Takeda Pharmaceutical raises its FY2025 revenue and profit guidance following resilient nine-month results and announces U.S. availability of a new ready-to-use immunoglobulin therapy for primary immunodeficiency, fueling discussions on its growth strategy amid high debt and margin pressures.
A Florida couple is suing their fertility clinic after discovering the baby girl born via IVF is not genetically related to them, sparking a complex legal and ethical battle over parentage and clinic accountability.
H.C. Wainwright maintains a Buy rating on Lexeo Therapeutics but slashes its price target, citing persistent market doubts over the company's PKP2 cardiomyopathy program despite encouraging early clinical results.
Following the FDA's decision to remove the clinical hold on Intellia's Nex-Z hATTR-PN trial, analysts at Baird and H.C. Wainwright have significantly raised their price targets for the CRISPR gene-editing pioneer, citing reduced regulatory risk and a clearer path forward.
Quince Therapeutics' lead candidate, eDSP, has failed to meet primary and secondary endpoints in its pivotal Phase III NEAT trial for the rare neurological disorder Ataxia-Telangiectasia (A-T), triggering a catastrophic drop in the company's share price.
Pharmaceutical giant Eli Lilly announces a major $3.5 billion manufacturing expansion for its weight-loss drugs and enters two strategic research collaborations in gene editing and immunology, signaling aggressive growth plans amid a recent stock valuation dip.
Sanofi's experimental oral drug venglustat demonstrated significant neurological benefits for patients with Type 3 Gaucher disease in a late-stage trial, but failed to meet its primary endpoint in a separate study for Fabry disease, casting uncertainty on its regulatory path.
Eli Lilly bolsters its long-term growth strategy through two new R&D partnerships and a key regulatory milestone for an ovarian cancer drug, signaling a strategic push beyond its blockbuster diabetes and obesity treatments.
At its Analyst Day, Korro Bio detailed its lead candidate KRRO-121, an RNA-editing therapeutic designed to tackle hyperammonemia in urea cycle disorders and hepatic encephalopathy by stabilizing a key liver enzyme, positioning it as a potential paradigm shift from current burdensome treatments.
Novo Nordisk charts a bold new course, launching a pivotal oral obesity drug while restructuring its workforce and deepening its investment in next-generation cell therapies for metabolic diseases.
ResMed Inc. reports an 11% revenue surge for its second fiscal quarter, driven by robust mask sales and strategic acquisitions, while navigating competitive pressures and capitalizing on emerging healthcare trends.
While investors chase AI and big tech, a niche biotech firm focused on rare genetic obesity disorders has quietly delivered staggering returns, with Wall Street seeing further 40% upside. Rhythm Pharmaceuticals' targeted approach and upcoming catalysts have analysts bullish, despite its relative anonymity.
Barclays initiates coverage of 4D Molecular Therapeutics with an Overweight rating and a $33 price target, citing a favorable industry backdrop with potential M&A tailwinds and shifting focus away from drug pricing pressures. The gene therapy developer also outlined key clinical milestones expected through 2026.
While Legend Biotech's CAR-T therapy Carvykti missed Q4 sales estimates, prompting mixed analyst reactions, some firms emphasize its curative potential and view the stock's recent decline as a strategic entry point for investors.
Wells Fargo boosts its price target for Taysha Gene Therapies (TSHA), citing strong progress in its pivotal Rett syndrome gene therapy program. The company has dosed the first patient in a key trial phase and received FDA alignment on its regulatory pathway.
The Apis Flagship Fund reported robust quarterly and annual gains for 2025, significantly beating global benchmarks. While highlighting its performance drivers, the fund's investor letter also discussed its position on gene therapy firm uniQure (QURE).
Sanofi reports a pivotal Phase 3 win for its investigational therapy venglustat in a rare neurological disorder, alongside positive EU regulatory news for Rezurock and progress for amlitelimab, signaling a strategic shift toward high-value specialty medicines.
