Beam Therapeutics sued YolTech's Yuxuan Wu & Zi Jun Wang and Serapha Bio for allegedly stealing trade secrets related to CRISPR base editing & lipid nanoparticle tech.

Drugs like Trikafta, first approved in 2019, have changed cystic fibrosis from a death sentence to a manageable chronic condition for the vast majority of patients. But the drug is only approved for c...

Vivodyne, backed by about $80M, grows vascularized human tissues to screen drugs as the FDA and NIH push alternatives to animal testing.

Endpoints 11: SonoThera is using ultrasound and microbubbles to push full-length dystrophin into muscle, with a $125M Series B and a 2027 trial.

Endpoints 11: ARCH-backed Medici Therapeutics is pairing personalized mRNA cancer vaccines with TIL cell therapy, with $50M+ more coming in a Series A.

Endpoints Executive Editor Drew Armstrong on why picking the 2026 Endpoints 11 was the hardest yet, and what to expect at the Sept. 24 Boston gala.

Stanford's Sergiu Pasca bred mice lacking cortex/hippocampus for the first time, replacing cells with human neurons, aiding neuropsychiatric research including frontotemporal dementia and autism.

Two pediatric deaths in Chinese CRISPR trials reignite debate over AAV delivery risks, as many of the 15 genetic-medicine experts Endpoints spoke with point to high viral doses rather than gene editin...

Typewriter Therapeutics raises $56M Series A to build RNA-based jumping-gene therapies for in vivo CAR-T and genetic liver disease.

ARPA-H awards up to $125M across 5 teams, including Waterfall Scientific, to build automated systems that can make personalized RNA medicines in under a week.

FDA approves Revolution Medicines' daraxonrasib (Rasonque) for second-line pancreatic cancer, after it nearly doubled survival in a Phase 3 to 13.2 months vs. 6.7 months on chemo.

Aurora Therapeutics, the first bespoke CRISPR startup, lays off staff and drops its PKU program as CEO Ed Kaye exits, months after a $16M seed round.

GOP Reps. Moolenaar & Cline urge FDA's Diamantas to reject Chinese clinical trial data unless sites are audited annually, citing patient safety risks & 3 deaths.

China’s cheap, fast and quiet system for testing cutting-edge medicines is facing scrutiny after the deaths of three people in investigator-initiated trials.

RiboX confirms a patient died in its unregulated China trial for RXIM002, an in vivo CAR-T therapy — the third such disclosed death in recent weeks.

GSK has launched a 33,600-person Finland trial of Shingrix to test if shingles vaccine reduces dementia risk in those 76+.

We interview Betty Tijms of Alzheimer Center Amsterdam, who has identified 5 Alzheimer's subtypes and urges drugmakers to treat the condition as multiple diseases for better trials.

We interview Betty Tijms of Alzheimer Center Amsterdam, who has identified 5 Alzheimer's subtypes and urges drugmakers to treat the condition as multiple diseases for better trials.

Nava Therapeutics discloses it has raised $89M as it targets in vivo CAR-T and kidney delivery with novel lipid nanoparticles.

Biogen's tau-lowering diranersen slows Alzheimer's decline 26% at the lowest dose in Phase 2, but an inverse dose response and a missed endpoint cloud the data.

ARPA-H taps 7 teams for up to $160M to build bespoke gene editing drugs, betting on Baby KJ's playbook — but some scientists say funding falls short.

Endpoints opens nominations for its 2026 Endpoints 11 awards; submit standout private biotech startups by July 14 ahead of the Sept. 24 gala dinner.

Spot Biosystems emerges from stealth with $40M raised, reporting early human data showing dystrophin restoration in two children with Duchenne muscular dystrophy.

UCSF spinout Kopra Bio has raised $9.1M to inject a genetically engineered virus into glioblastoma patients in a China trial.

Last month, Ascidian Therapeutics presented the first data suggesting a new way to fix broken genes may be safe in people. Now Eli Lilly has struck a deal with the startup.

The Allen Institute is launching a $200 million initiative to explore specific neurodegenerative diseases and even develop drugs for those conditions.

Tune Therapeutics' new method of CRISPR-based gene editing shows promise in curtailing chronic hepatitis B infections.

Yoltech Therapeutics, one of China’s most prolific gene editing companies has raised its biggest round of funding ahead of plans to go public in Hong Kong.

New York City-based startup Waypoint Bio has raised $20 million in series A financing to test multiple cell therapies in China.

It’s a crucial moment for the cell and gene therapy field. At the American Society of Gene & Cell Therapy’s annual meeting, several trends emerged: In vivo CAR-T is everywhere, CRISPR comes for Alzhei...

Serif Biomedicines, a startup launched last month by Flagship Pioneering, believes it has solved two technical challenges that have long prevented gene therapy from being delivered without troublesome...

Drug developers are racing to develop gene therapies that could replace chronic GLP-1 injections and pills with a one-and-done treatment. Now a company based just outside of Boston is poised to become...

Two scientists think they've solved a technical problem that has kept the mRNA revolution from reaching its full potential.

A biotech startup has studied a cancer survivor’s stool and identified about 50 strains of bacteria that collectively seemed to create the right conditions to help immunotherapy cure her advanced colo...

A new generation of scientists is trying to revitalize recombinase gene editing by using AI to create bespoke enzymes that can target specific spots in the human genome. And Eli Lilly is buying into t...

Addition Therapeutics is working on a genetic medicine that would turn a patient’s liver cells into little factories to permanently pump out their own weight loss drugs, the startup told Endpoints New...

An experimental treatment that uses CRISPR to edit genes directly inside the body has just succeeded in a Phase 3 trial, a first for the technology that puts it on track for a potential FDA approval i...

A trio of scientists behind the first gene therapy approved in the United States, Jean Bennett, Albert Maguire and Katherine High, have been awarded a Breakthrough Prize in Life Sciences. It’s arguabl...

A new report trying to settle a decades-old debate about Alzheimer’s disease has reached a dramatic conclusion: antibody drugs that target sticky amyloid beta proteins in the brain simply don’t work. ...

Every blockbuster new drug starts as hundreds of hours in the lab and behind the computer screen. Join us to get a window into that painstaking, sometimes painful process. What mechanisms are getting ...

Every blockbuster new drug starts as hundreds of hours in the lab and behind the computer screen. Join us to get a window into that painstaking, sometimes painful process. What mechanisms are getting ...

At a moment when many drug developers are shying away from politically-charged mRNA vaccines, the Parker Institute for Cancer Immunotherapy is doubling down on the technology’s promise to treat and pr...

Five people with beta thalassemia who had their blood stem cells genetically altered no longer require regular blood transfusions to stay healthy. Their Chinese-made treatment is likely to be cheaper,...

Life Biosciences, a startup co-founded by Harvard biologist David Sinclair, has raised an $80 million Series D financing. The money will support a Phase 1 trial of a gene therapy in two forms of visio...

Ambitious Vancouver startup Aspect Biosystems is trying to ensure the next big diabetes breakthrough retains its ties to Canada, unlike the insulin developers of a century ago.

Alltrna receives clearance for the first clinical trial of a tRNA-based therapy that could one day be used to treat multiple genetic diseases, starting with tests in Australia. The company raised $109...

Beam Therapeutics reports positive results from CRISPR base editing therapy for AATD, saying it is ready to seek accelerated approval for a moderate two-dose regimen.

Vaxess Technologies rebrands as Terrestrial Bio, raises $50M to pivot from vaccines to GLP-1 patches for semaglutide delivery, after AstraZeneca partnership ends.