All articles tagged with #gene editing
Eli Lilly’s high-dose, one-time gene-editing therapy VERVE-102 lowered LDL cholesterol by 62% in a Phase 1 trial, with no treatment-related serious adverse events, signaling potential for a single-shot approach to prevent heart disease after Lilly’s $1 billion acquisition of Verve Therapeutics.
Intellia Therapeutics said its one-time, in vivo CRISPR-based treatment for hereditary angioedema met its Phase 3 primary endpoint, reducing attacks by 87% vs placebo and achieving 62% attack-free patients at six months after a liver-directed infusion, with a favorable safety profile. The company has started a rolling FDA submission and targets a U.S. launch in 2027 if approved.
Four Boston-area researchers were honored at the 2026 Breakthrough Prize ceremonies: Stuart Orkin and Lee Roberts received Breakthrough Prizes of $3 million each for gene editing and muon physics, while Dillon Brout (Boston University) and Shu-Heng Shao (MIT) were New Horizon Prize recipients, each $100,000, for cosmology and generalized symmetries in quantum field theory. The awards, called the 'Oscars of Science' and cofounded by Zuckerberg and Chan, celebrate long-term breakthroughs—from sickle cell gene-editing therapies to precision cosmology data and new physics ideas.
NIH-backed researchers identified the small Al3Cas12f CRISPR enzyme and engineered the Al3Cas12f RKK variant to dramatically improve editing in human cells, raising efficiency from under 10% to over 80% (up to 90% in a key region). This enzyme's size allows packaging into AAV delivery vectors, a major step toward in-body gene therapies for diseases like cancer and ALS; next, researchers will test AAV-packaged delivery to move toward clinical use.
Dallas-based Colossal Biosciences is pursuing revival of extinct species—including dire wolves and the dodo—using ancient DNA and CRISPR. The effort has drawn praise for pushing conservation tech and public engagement, but also sharp criticism from scientists who question feasibility and ecological risks, warn against conflating revived animals with extinct ones, and worry about undermining habitat protection; supporters see potential conservation benefits and genetic diversity applications, while critics urge caution and rigorous science.
A gene-edited fungus used to make mycoprotein grows protein nearly twice as fast with far less sugar after deleting two genes with CRISPR to thin its cell wall; lifecycle analyses across six countries show substantial reductions in climate emissions, land use, and freshwater pollution, plus improved essential amino-acid quality. Regulators and large-scale production hurdles remain before it reaches diners.
The FDA unveiled a new plausible mechanism pathway to approve medicines crafted for patients’ individual mutations, moving the idea from preview into formal policy and potentially enabling gene-editing–based and other targeted therapies for rare diseases.
Silicon Valley-backed startups are marketing embryo screening and editing services that claim to predict or enhance traits such as IQ and height for around $50,000. Backers include OpenAI’s Sam Altman, Peter Thiel and Brian Armstrong, while critics warn these efforts could create a privileged class of genetically enhanced humans and raise profound ethical and safety concerns. The piece cites experts who question the science, legal bans on human embryo editing in the US, and pioneers like He Jiankui as cautionary precedents, highlighting the tension between autonomy and potential inequality as the race to ‘accelerate evolution’ faces moral scrutiny.
Jennifer Doudna's startup Aurora Therapeutics aims to scale personalized gene-editing treatments for rare diseases like PKU using CRISPR technology and a new FDA pathway that expedites approval for bespoke therapies based on limited patient data.
A prominent gene editing researcher from UC Berkeley, Fyodor Urnov, and venture capitalist Johnny Hu are launching a startup to scale personalized CRISPR medicines, aiming to treat ultra-rare genetic mutations and overcome regulatory and commercial challenges.
Scientists at the University of Kentucky developed a mouse model that can switch the APOE gene from a high-risk to a protective form in adults, leading to improvements in Alzheimer’s disease markers and cognitive function, highlighting potential gene-based therapies for the future.
The article discusses upcoming scientific developments in 2026, including advancements in AI, human gene editing, space missions, and the impact of US policy changes on science, highlighting key events to watch for this year.
Despite political challenges, 2025 saw significant medical advances including a groundbreaking gene edit for a baby, approval of 44 new drugs, progress in cancer treatments, improved vaccines, and innovative biotech research, highlighting biology's pivotal role in this century of medicine.
2025 has seen major medical breakthroughs including remote robotic surgery, a blood test for Alzheimer's, brain implants for ALS, advances in organ transplants, and gene editing treatments, transforming healthcare and patient outcomes.
The article discusses the ongoing development of gene editing technologies, highlighting the excitement and competition among scientists to discover the most effective and final form of gene editing tools.