biotechnology2.1 min read
FDA Rejects Replimune’s Engineered Virus for Melanoma Again, Sparking Regulatory Debate
1 day ago•Source: statnews.com
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5.51 min•21 days ago
Lab-grown esophagus in pigs restores swallowing, signaling pediatric repair prospects
Scientists grew lab-made oesophagi from pig stem cells on scaffolds and implanted them into eight recipient pigs. Over two months the grafts developed into functional tissue with nerves, muscle, and blood vessels, and five pigs survived six months, regaining the ability to swallow. While some initial scar tissue affected swallowing, it diminished over time, suggesting a path toward treating conditions like long-gap oesophageal atresia in children or muscular damage in adults, though human trials are still future work.
1.805 min•1 month ago
Moderna Roivant patent deal caps biotech patent fight at $2.25B
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Lab-grown liver assembloids edge toward miniaturized human livers
Nature•2 months ago
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ImmunityBio Pursues FDA Path to Resubmission for ANKTIVA in Papillary NMIBC
Business Wire•2 months ago
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Biotech Stocks Drop Sharply After Disappointing Bone Disease Drug Trials
Shares of Ultragenyx and Mereo BioPharma plummeted after their Phase 3 trial for the brittle-bone disease treatment setrusumab failed to meet primary endpoints, despite secondary improvements in bone density, leading to significant market value losses and cautious outlooks from analysts.
Rocket Pharmaceuticals' Gene Therapy Trial for Danon Disease Faces FDA Hold After Patient Death
Rocket Pharmaceuticals announced an update on its Phase 2 trial of RP-A501 for Danon disease after a patient experienced a serious adverse event and subsequently passed away, leading to a clinical hold by the FDA. The company is investigating the cause, working with regulators and stakeholders, and has paused dosing to ensure patient safety. The trial aims to evaluate the efficacy of RP-A501, a gene therapy targeting cardiac function in Danon disease, with ongoing efforts to resume the study.
Arrakis CEO Discusses Challenges in Developing RNA-Targeting Cancer Drugs
Arrakis, a biotech startup led by Michael Gilman, is tackling the challenge of targeting the Myc oncogene, which is implicated in 70% of cancers and is difficult to target due to its "intrinsically disordered" protein structure. Instead of targeting the protein directly, Arrakis aims to intercept the mRNA responsible for its production, a novel approach in drug development.
Radiopharmaceutical Growth Stalled by Talent Shortage
The rise of radiopharmaceuticals, a new cancer treatment that targets cancer cells with radioactive isotopes, is facing a significant talent shortage. This innovative approach has attracted numerous biotech startups and substantial investments, with several companies being acquired for over $1 billion. However, the industry's growth is threatened by a lack of skilled professionals needed to develop and implement these therapies.
Summit Therapeutics' Stock Skyrockets After Cancer Drug Outperforms Keytruda in Phase 3 Trial
Summit Therapeutics' stock surged after the company announced that its cancer drug outperformed Keytruda in a Phase 3 trial, just ahead of a major cancer conference in Chicago.
Intellia Withdraws from Hemophilia Collaboration with Regeneron
Intellia has exited a partnership with Regeneron to develop a factor IX gene editing therapy for hemophilia B, opting out of a deal that was signed in 2020. Despite this, Intellia remains eligible for up to $320 million in future milestones and still holds a 35% stake in a factor XIII insertion program for hemophilia A.
"AI's Evolution in Biology: From Black-Box Data to Understanding Life"
The convergence of AI and biology is expected to lead to a new era of "Biology 2.0," with AI breakthroughs transforming biology into a programmable, predictable, and replicable engineering discipline. The transition from traditional "white-box" data to novel "black-box" data modalities developed alongside computational methods is anticipated to drive significant future breakthroughs. The release of ChatGPT by OpenAI has brought AI into the global spotlight, sparking frenetic optimism around AI in the biological sciences and raising hopes for transformative advancements.
"Latigo Secures $135M for Non-Opioid Pain Medicine, Chasing Vertex Success"
Latigo Biotherapeutics, a non-opioid drug developer, has emerged with a $135 million Series A funding after years of development, targeting the same Nav1.8 as Vertex Pharmaceuticals. The company aims to address pain management without the risk of addiction by focusing on the peripheral nervous system.
"Advancements in Bluebird Gene Therapies, Vertex's Pain Drug, and ADC FOMO"
Bluebird Bio's focus on selling gene therapies, such as Lyfgenia for sickle cell disease, Zynteglo for beta thalassemia, and Skysona for cerebral adrenoleukodystrophy, without other marketed products, makes it a unique player in the gene therapy market, raising questions about the profitability of gene therapies as a business. The company's approach and success over the next year may provide insights into the potential for gene therapies to become a profitable venture.
"Doudna's Aclaris CEO Steps Down, Secures $114M Liver Disorder Deal with Genevant"
Jennifer Doudna's lab published a study detailing how antibody fragments can target virus-like particles with gene editing machinery to T cells, co-founding genetic medicine delivery startup Azalea Therapeutics, which has raised $10 million.