Three gene therapy pioneers just won the Breakthrough Prize. This is their story 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...

Three gene therapy pioneers—Jean Bennett, Albert Maguire and Katherine High—just won the Breakthrough Prize. I talked to them about the long, winding road to Luxturna and the struggle to parlay that singular success into more therapies for retinal diseases. @endpts.com endpoints.news/three-gene-t...

Cochrane review dismissing amyloid drugs draws immediate backlash 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. ...

A new Cochrane review dismissing #amyloid drugs has drawn immediate backlash. My story for @endpts.com covers the latest #Alzheimers controversy and examines why past drugs failed and why there's still hope for newer experimental amyloid antibodies. endpoints.news/cochrane-rev...

Later in the day, I’ll be joined by Aurora Therapeutics CEO Ed Kaye for our in-person happy hour. We’ll dig into the company’s ambitious goal of developing a whole series of individualized CRISPR medicines and talk about some of the broader challenges facing gene therapy.

After that, I’ll be back for a panel about a new suite of technologies that sneak antibody drugs across the blood brain barrier to make Alzheimer’s treatments safer and more effective. Featuring Denali CSO Joe Lewcock, Wyss Institute’s James Gorman and Roche’s Azad Bonni.

Then I’ll be handing the mic over to @andrewedunn.bsky.social for an interview with Stanford professor James Zou, who is creating virtual labs and AI "scientists" to accelerate life science research and drug discovery.

I'm kicking the day off by looking at new technologies like recombinases and retrotransposons that could finally crack the challenge of precise gene insertion. Featuring former Intellia CSO Laura Sepp-Lorenzino, Seamless CEO Albert Seymour, and Addition CSO Francine Gregoire.

ENDPOINTS Drug Discovery Day 2026 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 ...

Endpoints Discovery Day is here, starting at 11:00 AM ET. We've got a fantastic lineup spanning gene editing, AI agents, and drug delivery tech for the brain. You can watch online for free by registering here: events.endpoints.news/drugday26 @endpts.com

ENDPOINTS Drug Discovery Day 2026 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 ...

We've got a great lineup for Endpoints Drug Discovery day tomorrow (Wed. April 15). We'll be covering gene editing "beyond CRISPR," AI agents in the lab, drug delivery tech that promises to revolutionize Alzheimer's treatments and more. Sign up here: events.endpoints.news/drugday26

Parker Institute doubles down on cancer vaccines as part of ongoing reboot 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...

"The moment that you don’t do something for fear of becoming political is the moment that you’ve become political yourself," Karen Knudsen told me. "And if mRNA vaccine technology is the way to prevent cancer recurrence, I think it will be warmly embraced." endpoints.news/parker-insti...

A ‘cure’ for five blood disease patients suggests Chinese genetic medicine can compete globally 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,...

Shanghai-based CorrectSequence Tx has developed a #CRISPR therapy that has seemingly cured five patients with beta thalassemia. My latest in @endpts.com explores what that says about the growing cell and gene therapy competition from China. endpoints.news/china-biotec...

David Sinclair startup raises $80M for clinical test of anti-aging gene therapy 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...

Life Biosciences, @davidsinclairphd.bsky.social's startup, has raised $80M for a clinical test of its anti-aging gene therapy using three Yamanaka factors for partial epigenetic reprogramming in the eye to heal damaged retinal cells. More in my story for @endpts.com: endpoints.news/david-sincla...

Diabetes cell therapy startup gets $79M from Canadian government 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.

Aspect Biosystems has quietly become one of the most well funded cell therapy startups. The Vancouver company just raised $79M from the Canadian govt for its diabetes cell therapy, putting its total funding above $500M. Get the details in my latest for @endpts.com: endpoints.news/aspect-biosy...

Exclusive: First clinical trial of tRNA therapy, a new disease-agnostic form of genetic medicine, will start soon 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...

A new form of #RNA medicine where a single therapy could potentially be reused across many different diseases is heading into the clinic.

@alltrna.bsky.social just got clearance to start the first trial of a #tRNA therapy

Read more in my exclusive for @endpts.com: endpoints.news/first-clinic...

Beam looks to accelerated approval for AATD base editing after promising update 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.

Base editing company Beam looks to pull ahead in the race to develop genetic medicines for AATD. After a promising update from its ongoing Phase 1 study, it's enrolling a "pivotal cohort" to support a potential accelerated approval from the FDA. More in @endpts.com: endpoints.news/beam-looks-t...

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

Microneedle vaccine patch startup Vaxess is rebranding as Terrestrial Bio after raising a $50M series C and pivoting from mRNA vaccine work (after AstraZeneca dropped its partnership) to GLP-1 drug delivery. My exclusive for @endpts.com has the details: endpoints.news/microneedle-...

Q&A: Embryo editing entrepreneur Cathy Tie closes one startup and begins another in biology’s most taboo frontier Cathy Tie, former wife of He Jiankui, launches Origin Genomics after Manhattan Genomics shutdown, aiming to develop embryo editing treatments despite controversy and regulations.

Cathy Tie has attracted attention for her relationship with ousted embryo editing scientist He Jiankui and her goal to commercialize embryo editing in the US. I talked to Tie about why her first startup shut down, and her plans for a second company. Q&A @endpts.com: endpoints.news/qa-controver...

Merck partners with Flagship's 'somatic genomics' startup to search for clues to IBD Quotient Therapeutics, a startup studying how tiny genetic typos cause some cells to get sick but allow others to remain healthy, has struck a partnership with Merck for $20 million upfront and future...

Flagship Pioneering startup Quotient Therapeutics has struck its third pharma partnership, this time with Merck, to look for new drug targets based on the growing field of somatic genetics. More details on the approach in my latest for @endpts.com: endpoints.news/merck-partne...

Basecamp debuts an ambitious plan to sequence a trillion genes, but questions remain Basecamp Research aims to sequence 1 trillion proteins in 2 years, aiming to revolutionize AI-driven drug development via Trillion Gene Atlas project.

AI startup @basecamp-research.bsky.social has unveiled an ambitious project to sequence one trillion protein coding genes to create a massive AI model for biology.

@andrewedunn.bsky.social and I examine what it means in our latest story for @endpts.com: endpoints.news/ai-startup-b...

Karen Weintraub and Kevin Miller to join Endpoints News as editors Endpoints News hires Karen Weintraub from USA TODAY and Kevin Miller from Bloomberg News as deputy editors to help oversee biotech coverage and business news.

Excited to see @endpts.com continue to grow with the addition of two outstanding editors, one of whom (@karenweintraub.bsky.social) taught my science news writing class a decade ago when I made the jump from research to journalism. Good things in store, stay tuned! endpoints.news/karen-weintr...

Exclusive: China mRNA therapy startup Innorna to test gout treatment in US Innorna, a Chinese biotech largely based in Shenzhen, won clearance from the FDA to begin a Phase 1 study of an mRNA therapy for refractory gout, the company told Endpoints News.

Another Chinese biotech developing #mRNA therapies has got clearance from the FDA to start a clinical trial in the US. My latest for @endpts.com examines Innorna's experimental gout treatment and what it could mean for the field: endpoints.news/china-mrna-t...

Exclusive: China startup CirCode gets clearance for trial of circular RNA therapy CirCode Biomed, a Chinese startup working on an experimental class of genetic medicines known as circular RNA, has received the FDA's blessing to begin a clinical trial in the US

China startup CirCode is the second biotech to get FDA clearance for a trial of a circular RNA therapy. The company's two lead programs use localized injections of growth factors to spur blood vessel formation in ischemic tissue. More details in @endpts.com: endpoints.news/china-startu...

A simpler, single-strand form of DNA may be key to non-viral gene therapy, study suggests Study shows single-stranded DNA may bypass immune detection for gene therapy, with Mass General Brigham and Full Circles Therapeutics developing circular DNA form for safer delivery.

There’s no more iconic shape in biology than the DNA double helix. But a simpler form of DNA may be key to overcoming immune reactions that have long hindered the development of non-viral gene therapy. My latest for @endpts.com looks at a new study in detail: endpoints.news/a-simpler-fo...

Study suggests non-coding RNA, long considered genomic dark matter, has potential in anti-inflammation Scientists develop synthetic long noncoding RNAs as potential anti-inflammatory drugs, with University of Toronto's Omar Khan showing promising results in human cells and mice.

Study suggests long non-coding RNA (lncRNA) has potential as new class of genetic medicine, including as an anti-inflammatory drug. I talked to the scientists at University of Toronto to learn more for my story in @endpts.com: endpoints.news/study-sugges...

Exclusive: RNA editing startup Tacit Therapeutics launches with $19M for brain diseases Tacit Therapeutics raises $19M to develop RNA editing therapies, targeting Huntington's disease and Dravet syndrome. CEO David Nelles leads effort using RNA splicing to fix genetic mutations.

RNA editing startup Tacit Therapeutics launches with $19M to make "trans-splicing" therapies that replace large swathes of broken genetic code in brain diseases including Dravet syndrome and Huntington's disease. My exclusive for @endpts.com has the details: endpoints.news/rna-editing-...

Exclusive: EveryOne Medicines shuts down, ending custom cure venture that banked on new FDA pathway EveryOne Medicines, a biotech developing individualized genetic therapies, to close after FDA's new guidance falls short of expectations for streamlined drug approvals.

EveryOne Medicines, a startup making individualized therapies for genetically unique diseases, is shutting down a week after the FDA announced a streamlined pathway for those types of bespoke medicines. @jaredwhitlock.bsky.social and I have the scoop for @endpts.com: endpoints.news/everyone-med...

Polymer nanoparticle startup raises $60M for mRNA diabetes therapy BreezeBio, a startup founded a decade ago to develop new delivery technologies for genetic medicines, has raised $60 million to start clinical trials of its first program in type 1 diabetes

Polymer nanoparticle startup BreezeBio (formerly known as GenEdit) has raised $60M to advance an mRNA-based immune tolerance therapy for diabetes into the clinic. More details on the therapy and the delivery system in my story for @endpts.com:
endpoints.news/polymer-nano...

Exclusive: First test of gene therapy for rare form of autism is underway Two years ago, a small biotech startup got permission to test a gene therapy for a rare and disabling form of autism. The plan: Deliver a gene that’s vital for ...

Jaguar Gene Therapy is testing a treatment for a debilitating form of #autism caused by a broken or missing #SHANK3 gene. Early results from six children are expected later this year. My latest for @endpts.com dives into the backstory of the science and the company.
endpoints.news/first-test-o...

Exclusive: Feng Zhang’s epigenetic editing startup shifts focus to siRNA obesity therapies Moonwalk Biosciences, founded by Feng Zhang and Alex Aravanis, shifts from epigenetic editing to siRNA drugs, targeting obesity through adipose tissue manipulation.

Two years ago, #CRISPR pioneer Feng Zhang and former Illumina Alex Aravanis launched Moonwalk Biosciences to develop #epigenetic editing therapies. Now the company is shifting its focus to #siRNA therapies for #obesity. More details in my story for @endpts.com: endpoints.news/epigenetic-e...

Exclusive: Sanofi invests in China startup developing molecular glue for sickle cell disease GluBio Therapeutics plans trials for pills to boost hemoglobin levels. Sanofi invests $30M for first rights to license two experimental drugs targeting sickle cell disease.

The Shanghai and San Diego-based startup GluBio has developed molecular glues that it says can upregulate fetal hemoglobin (much like the CRISPR therapy Casgevy) for sickle cell disease. Now they've got a $30M investment from Sanofi. More in @endpts.com - endpoints.news/sanofi-inves...

Flagship’s Generate files for an IPO in key test for the AI bio field Generate Biomedicines files for IPO 9 days after starting Phase 3 trial of GB-0895 for severe asthma, with Flagship Pioneering owning 56.6% of AI-focused biotech company.

@generatebiomed.bsky.social, one of the biggest companies dedicated to making protein therapies with AI, has filed for an IPO. @andrewedunn.bsky.social and I tackled the S-1 last night and pulled out some interesting details for our story in @endpts.com: endpoints.news/flagships-ge...