When we evaluate biotech startups for inclusion in our annual Endpoints 11 list, we look for companies that are swinging for the fences with cutting-edge science, where success or failure will make a big dent. Nominations open until July 15: endpoints.news/nominations-...

I'll be in London covering the #Alzheimers conference #AAIC26 for @endpts.com in less than two weeks. Send me a note if you'll be there and are presenting new data!

Spot Bio has raised $40M to deliver genetic instructions for full-length dystrophin (the big muscle protein that's broken or missing in muscular dystrophy) with natural nanoparticles known as extracellular vesicles. My latest for @endpts.com has the details: endpoints.news/spot-bio-lau...

Kopra Bio is planning a trial in China to test a genetically engineered virus that helps expose brain tumors by releasing an inflammatory molecule that recruits immune cells. Read more about the approach and the company in my latest for @endpts.com endpoints.news/ucsf-spinout...

Lilly is partnering with Ascidian to develop RNA "exon editing" therapies for genetic kidney diseases (an interesting drug delivery challenge!) It's at least the 6th deal Lilly has struck in just over a week, reaching a total deal value of $10B. More in @endpts.com : endpoints.news/lilly-strike...

The @alleninstitute.org, long known for its focus on simply trying to understand how the brain works, is launching a $200 million initiative to translate its knowledge into drug development for neurodegenerative diseases. My story for @endpts.com has the details: endpoints.news/allen-instit...

Tune Tx's CRISPR-based epigenetic editing shows promise against chronic hepatitis B infections, and the company says its on track for a cure. I explain the novel technology and break down the company's biomarker data in my latest story for @endpts.com : endpoints.news/epigenetic-e...

At a time when most US gene editing companies have trimmed their pipelines, China-based YolTech Tx has brought six CRISPR therapies into the clinic. Now they've raised $70M to ramp up trials in China and the US. Read more in my exclusive for @endpts.com: endpoints.news/china-gene-e...

New York City-based Waypoint Bio has raised a $20 million series A to run three CAR-T trials (investigator initiated trials) in China, with a focus on solid tumors (including some in vivo approaches). My latest for @endpts.com has the details: endpoints.news/waypoint-bio...

It's been a busy week at #ASGCT2026. In my "Dispatch," I wrote about how an vivo CAR-T overload, about two startups working on CRISPR therapies for Alzheimer's, and about a missed opportunity to ask hard questions of the FDA. Read more in @endpts.com: endpoints.news/asgct-dispat...

First news from #ASGCT2026 this morning. Flagship startup Serif Biomedicines has presented its first preclinical data in rodents and monkeys suggesting that it has solved two key technical challenges in non-viral gene therapy. Read more in @endpts.com: endpoints.news/flagship-sta...

Greetings from #ASGCT2026 in Boston. The cell and gene therapy space may still be struggling for funding, but the science remains exciting. I'll be on the ground all week bringing you the latest news for @endpts.com. Lots to come, stay tuned!

European regulators greenlight Fractyl Health's clinical test of the first ever GLP-1 gene therapy. Read more in my exclusive interview in @endpts: endpoints.news/european-reg...

Two former Orbital Therapeutics scientists have launched a new #mRNA therapy startup to bring the durability of circular RNA into the old-fashioned and easier-to-make linear mRNA. Read more in my exclusive for @endpts.com: endpoints.news/former-orbit...

Scientists trying to understand why cancer immunotherapy works so well for some patients and not others have found a potential answer in one cancer survivor’s poop. Read more about the microbiome startup Kanvas Bio in my latest for @endpts.com : endpoints.news/microbiome-s...

#Recombinase based gene editing was barely on anyone's radar a year ago. Now several startups are working on it, and @elilillyandcompany.bsky.social has partnerships with 2 of them (Profluent and Seamless) and has invested in a third (Stylus). More in @endpts.com: endpoints.news/lilly-strike...

UC Berkeley startup Addition Therapeutics is betting that a new gene editing tech based on jumping genes (retrotransposons) will be key to making a safe, redoseable and reversible GLP-1 gene therapy. I explore the idea in my latest exclusive for @endpts.com: endpoints.news/exclusive-uc...

Big day for #CRISPR. Almost 14 years after the gene editing technology was first published, @intelliatx.bsky.social's CRISPR therapy has succeeded in the first Phase 3 trial of in vivo editing (inside the body), putting them on path to approval in 2027. @endpts.com - endpoints.news/intellias-in...

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...

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...

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

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

"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...

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...

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...

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...

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...

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...

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-...

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...

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...

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...

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...

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...

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...

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 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...

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-...

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 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...

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...

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...

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...

@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...

My latest for @endpts.com: Chinese #CRISPR startup AccurEdit has raised $75 million for gene editing therapies. It has data in ATTR comparable to Intellia, data in PCSK9 comparable or slightly better than Verve, and is working on a novel therapy for MASH/NASH. endpoints.news/china-startu...

Lilly is partnering with Seamless Therapeutics on a gene editing therapy based not on CRISPR but enzymes called recombinases. It's big news for the post-CRISPR wave of startups making tools for precise gene insertion. My story in @endpts.com has the details: endpoints.news/lilly-partne...

Harvard biologist @davidsinclairphd.bsky.social's startup @lifebiosciences.bsky.social is about to begin a clinical study of its gene therapy for "partial epigenetic reprogramming." It's a big first in the longevity field. I've got the details for @endpts.com - endpoints.news/exclusive-fd...

Scribe Therapeutics, a #CRISPR gene editing company cofounded by Jennifer Doudna in 2017, plans to start its first clinical trial this summer. The startup's lead program is an epigenetic editor targeting PCSK9 to lower cholesterol. More details in @endpts.com: endpoints.news/scribe-thera...

Former Tome Bio leader John Finn is continuing his search for the ideal gene insertion tool at Basecamp Research. Today, the startup revealed its first AI-generated enzymes (large serine recombinases) that can insert genes without CRISPR. More in @endpts.com: endpoints.news/nvidia-partn...

Mirador Therapeutics has raised a $250M series B, bringing its total funding to more than $650M. The startup plans to have at least 10 clinical trial readouts of immunology drugs by the end of 2027. More details in my exclusive for @endpts.com: - endpoints.news/mirador-rais...