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The U.S. Congress greenlit a historic $315 million in federal ALS research funding for 2026 amid Rare Disease Month, spotlighting biotech progress like VectorY Therapeutics’ first patient dosing in its TDP-43-targeting PIONEER-ALS trial and EverythingALS’ pharma consortia driving biomarker innovations and trial alignment.
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Many scientists-turned-CEOs paradoxically abandon scientific principles when it comes to commercializing their innovations. But applying the scientific method to business decisions can help life science entrepreneurs avoid common pitfalls, attract investment and ultimately bring transformative technologies to market.
FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
PitchBook’s 2025 biopharma VC analysis clocked $33.8 billion in capital dispatched in 2025, mainly to companies with later-stage programs ready to roll into the clinic.
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The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
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Following a lawsuit filed last week, Sage has officially rejected Biogen’s unsolicited buyout offer, which valued the embattled biotech at just $469 million.
Patients taking Novo Nordisk’s Wegovy plus Veru’s enobosarm saw 71% lower lean mass loss than those who were taking Wegovy alone but investors await more data.
The monthly maintenance regimen, which offers a more convenient frequency than the initially approved treatment schedule for patients with Alzheimer’s, could help with Leqembi’s thus far disappointing uptake and sales.
From March 2020 through September 2022, Biohaven wielded meals at high-end restaurants and paid speaking opportunities to induce healthcare providers to prescribe its migraine therapy Nurtec ODT, according to the Department of Justice.
Bristol Myers Squibb’s Opdivo plus Yervoy, as well as Pfizer’s Braftovi, have each shown strong Phase III performances that could position them as new standards of care in certain subtypes of metastatic colorectal cancer.
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.
The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this month the company has secured an approval for AstraZeneca-partnered Dato-DXd, to be marketed as Datroway.
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
Less than a day into his second term, President Donald Trump ordered a freeze on communications at major public health agencies, among other moves that have sent waves through the biopharma industry.