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J&J eye disease gene therapy bota-vec fails Phase 3

Johnson & Johnson’s gene therapy for a genetic disease that leads to vision loss called X-linked retinitis pigmentosa (XLRP) failed a pivotal trial, according to an update to a J&J healthcare...

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Unity lays off 'all' workers; Novo to get FDA decision on oral Wegovy this year

Plus, news about ImmunityBio and Inventiva: Unity Biotechnology shelves work: The anti-aging drug developer plans to evaluate strategic alternatives and conduct layoffs that affect “all of its...

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AI biotech Recursion cuts pipeline to 'sharpen focus'

Recursion's leaders tried to make the case last year that the company had avoided the fate of some of its AI-focused peers, which had seen their first clinical programs fizzle out. The Salt Lake City ...

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Function buys Ezra, combining preventive testing and scans

Function Health is buying Ezra in a bid to create a haven for people who want copious amounts of data on their bodies. The combined company will be a one-stop shop for preventive lab testing ...

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BioNTech doesn't see ‘near-term’ impact from RFK Jr.'s vaccine policy proposal

A proposal by HHS Secretary Robert F. Kennedy Jr. to change the approval standard of new vaccines shouldn't affect BioNTech's Covid-19 franchise, the company told investors Monday. Biotech and pharma...

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Bristol Myers to lay off over 500 workers in New Jersey

Bristol Myers Squibb is letting go of 516 workers in New Jersey as part of a wide-reaching cost-cutting plan aimed at making the drugmaker more efficient. The company, based in Princeton, NJ, first...

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PTC's Huntington’s study succeeds, but accelerated approval uncertain

PTC Therapeutics announced Monday that its splicing drug for Huntington’s disease met the primary endpoint of a Phase 2 study, but stopped short of spelling out the important data that could ...

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FDA adcomm to review Capricor's Duchenne cell therapy

Capricor Therapeutics' cell therapy for Duchenne muscular dystrophy will face an FDA advisory committee ahead of an approval decision that's expected Aug. 31, the company disclosed on Monday. The San...

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New opioid data may not be generalizable, FDA adcomm says

Several members of an FDA advisory committee warned on Monday that new data from studies on long-term use of opioids might underestimate the harms from their misuse and abuse. The committee reviewed...

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Vertex touts promising launch for non-opioid pain drug Journavx

Vertex said it's gathering momentum with its new product launches, building out new franchises with its sickle cell therapy Casgevy, another cystic fibrosis drug in Alyftrek, and the non-opioid pain...

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Singapore cardio gene therapy biotech collects $45M as it plots Europe expansion

A Singapore-based startup raised a $45 million Series B to start testing a gene therapy in humans next year for a genetic heart muscle disorder, signaling that the AAV-based gene therapy field still...

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Aldeyra plots third FDA filing for dry eye disease drug after latest Phase 3...

Aldeyra Therapeutics isn’t giving up on its drug candidate for dry eye disease despite a challenging path so far. The company is preparing to file reproxalap with the FDA for the third time after a ...

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Degradation startup raises $39M for pivot to PROTACs and clinical entry

PAQ Therapeutics has taken a new direction. The startup shifted its protein degradation approach to the more common PROTAC lane, pivoted to cancer, hired a chief medical officer from Frontier...

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Lilly to pay up to $415M in ALS deal; Actuate’s mid-stage pancreatic cancer data

Plus, news about Atara Biotherapeutics, Immutep, bluebird bio, OSE Immunotherapeutics, Mersana Therapeutics, Innovent, Biomea Fusion and Trailhead Biosystems: Eli Lilly does another ALS deal: The...

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Vertex pauses study of mRNA therapy for cystic fibrosis

Vertex paused an early-stage trial of its inhaled treatment for cystic fibrosis that came from a partnership with Moderna. The biotech said Monday in its first-quarter press release that it's...

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Anti-aging biotech NewLimit raises $130M Series B round

NewLimit, an anti-aging biotech startup backed by Coinbase CEO Brian Armstrong, has raised a $130 million Series B round to get its lead drug candidates closer to human testing. The South San...

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Controversial doctor Vinay Prasad named CBER director at FDA

FDA Commissioner Marty Makary named Vinay Prasad as the next director of the Center for Biologics Evaluation and Research, putting a strident critic of the agency in charge of one of its most important...

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Europe pledges €500M to attract global science talent

The European Union is committing €500 million ($556 million) over the next two years to make Europe a “magnet” for researchers from around the world. The funding was announced Monday by European...

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Trump, FDA zero in on foreign factories to give domestic sites an edge  

With several large pharma companies promising billions of dollars to reshore manufacturing to the US, President Donald Trump and the FDA have started on plans that would give current and future...

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Plans to match US drug prices to those overseas gain steam in Washington

House and Senate lawmakers from both parties are working with the Trump administration on proposals that would use drug prices paid by other developed countries as a reference for the US, an idea that...

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