Gene therapy, CRISPR, synthetic biology, and emerging platforms—what's real, what's hype, and what translates.
Two Nature Biotechnology papers describe distinct prime editing approaches for inserting or replacing larger DNA fragments without generating double-strand breaks. Together, they point to a maturing effort to expand precise genome engineering beyond point edits and short indels.
Two Nature Biotechnology papers describe distinct prime editing approaches for inserting or replacing larger DNA fragments without generating double-strand breaks. Together, they point to a maturing effort to expand precise genome engineering beyond point edits and short indels.
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A Nature paper describes Bonsai, a Bayesian method that reconstructs tree structures from high-dimensional data with heterogeneous measurement noise. The authors position it as an alternative to widely used visualization approaches that can distort structure in single-cell omics datasets.
A review arguing that dietary protein restriction may promote longevity is drawing pushback from nutrition experts who say the evidence leans heavily on animal research and could be misapplied to older adults. Their critique centers on a practical point: preserving strength and function in humans is not the same as extending lifespan in model systems.
EyePoint’s duravyu failed to beat aflibercept on vision improvement in the Phase 3 Lugano trial, sending the company’s shares down 70% at market open. Secondary data on treatment burden and safety kept the readout from being a clean collapse, but the primary miss now hangs over the matching Lucia study.
Definium Therapeutics reported that its single-dose oral LSD candidate DT120 met primary and key secondary endpoints in a Phase 3 generalized anxiety disorder study. The result adds a second late-stage win this summer and shifts attention to a follow-on trial that could shape a filing path.
A Stanford-led study found that tau may drive neurodegeneration by disrupting mitochondrial electron flow, creating reactive oxygen species, cellular stress and inflammation. The work suggests a new way to intervene beyond simply targeting tau aggregates.
After two previous rejections, Replimune’s advanced melanoma immunotherapy secures FDA approval, capping years of regulatory twists and strong oncologist support.
The FDA has approved a ground-breaking narcolepsy drug from Takeda, signaling a huge step forward for patients and the broader drug class.
Pathos AI has made headlines with two major cancer deals involving AstraZeneca and China’s Alphamab. While specific financial details remain under wraps for the AstraZeneca pact, Pathos could pay over $2 billion for a late-stage breast cancer asset developed by Alphamab.
Apnimed’s successful $192 million IPO propels the company toward the launch of Oxnimbi, which could become the first FDA-approved oral medication for obstructive sleep apnea if regulatory review proceeds as planned.
Early clinical findings from ProMIS Neurosciences reveal potential progress toward a safer amyloid-clearing drug for Alzheimer’s disease, addressing a long-standing challenge of managing adverse effects.
