Arbor Biotechnologies has partnered with Chiesi Group in #CRISPR gene editing deal worth up to $2 billion, just 10 weeks after announcing it had dosed the first patient in its PH1 trial. Next up for Arbor: a trio of ALS therapies. Read more in @endpts.com - endpoints.news/arbor-partne...

I am heading to Spain next week to cover the #ESGCT2025 meeting for @endpts.com. If you're presenting new cell therapy, gene therapy, gene editing or RNA therapy research at the meeting, or just want to meet, reach out!

The French biotech EG 427 has made a gene therpay based on a herpesvirus vector that delivers a botulinum toxin to sensory neurons to reduce urinary incontinence by 88% in spinal cord injury patients. More details on the data and science in my exclusive for @endpts - endpoints.news/exclusive-fr...

In 2023, Biogen said it would deprioritize its early stage gene therapy research, but a small team of scientists continued working on next-gen AAV capsids. Now Biogen has cut its remaining AAV work. Read more in my exclusive for @endpts.com - endpoints.news/biogen-calls...

Feng Zhang's startup Aera Therapeutics has announced an in vivo CAR-T therapy for autoimmune disease as its lead program, and plans to be in clinical trials in mid-2026. Read more about that program, and two more at Aera, in my exclusive for @endpts.com - endpoints.news/aera-therape...

One of my favorite rituals at @endpts is picking our annual list of the most promising startups pushing science and medicine forward. Hundreds are nominated, we seriously debate 40-50 of them, and we whittle it down to the Endpoints 11. The list is out! endpoints.news/the-2025-win...

I spoke with #LaskerAward winner Michael Welsh from the University of Iowa about how he helped unravel the molecular mechanisms of #CysticFibrosis that laid the foundation for Vertex's life-saving drugs for the once fatal disease. Read the interview on @endpts.com: endpoints.news/a-conversati...

Chinese #CRISPR company YolTech Therapeutics has raised a $45M series B as it plans for its first Phase 3 study in ATTR, the same genetic disease that US-based Intellia recently began a pair of Phase 3 studies in. Read more in my exclusive for @endpts.com: endpoints.news/chinese-cris...

China biotech Epigenic Therapeutics raises $60M to test #CRISPR #epigenetic therapies for HBV in Phase 1/2 trial, competing with US startups nChroma and Tune. Read more from my exclusive interview with Epigenic CEO Bob Zhang in my story for @endpts.com endpoints.news/china-biotec...

Neuropsych startup Sensorium, founded by Mass General and Harvard scientists, has raised $25M for a Phase 1 study of a new plant-derived drug for anxiety. CEO Jacob Hooker told me about the drug's mechanism for the first time in my exclusive story for @endpts.com: endpoints.news/sensorium-ra...

Gameto, a startup looking to disrupt the in vitro fertilization industry with stem cell technology from George Church's lab, has raised $44 million in Series C financing for its Phase 3 study. My story for @endpts.com has the details: endpoints.news/fertility-st...

After promising proof of concept data from a Phase 1 study earlier this year, Strand Therapeutics has raised $153 million series B funding to test its #mRNA cancer therapies encoding the potent immune-stimulating protein IL-12. Read more in Endpoints News: endpoints.news/strand-thera...

The session at #AAIC25 on Roche's next-gen #Alzheimers drug #trontinemab was jam packed and despite one patient's death, the excitement for this drug was palpable. Read more about the dramatic amyloid reduction and surprisingly low ARIA in @endpts.com - endpoints.news/roche-double...

Orbital Therapeutics, a circular RNA startup that raised $270M for nex-gen RNA drugs in 2023, revealed its first program and first preclinical data for an in vivo CAR-T cell therapy for autoimmune disease. More details in my story for @endpts.com - endpoints.news/circular-rna...

David Baker’s lab @uwproteindesign.bsky.social uses AI to catch nature’s squirmiest proteins, the notoriously undruggable intrinsically disordered proteins. I covered the new methods, which could have implications in cancer, diabetes, pain and more for @endpts.com - endpoints.news/david-bakers...

Sail Bio, the Flagship company that combined the circular RNA startup Laronde and nanoparticle startup Senda, just cut its staff by 30%. The CEO and CMO left earlier this year. Now the CSO and heads of platform, legal and HR are gone too. Read more in @endpts.com endpoints.news/sail-bio-cut...

The FDA approved a new dosing of Lilly’s #Alzheimers drug #Kisunla to lower brain swelling risks known as ARIA-E, based on a new Phase 3 study where modified dosing caused ARIA-E in 14% of patients compared to 24% on original dosing. More in my story for @endpts.com endpoints.news/fda-approves...

A new CRISPR Cures center, fuded by $20M from @chanzuckerberg.bsky.social and led by @urnov.bsky.social, will create 8 custom therapies for patients in the next 3 years. “Our goal is to aggressively walk the walk of CRISPR platformization,” Urnov told me. @endpts.com endpoints.news/crispr-cures...

It's a tough time to be a vaccine startup, but Centivax CEO Jacob Glanville has raised $45M for a universal flu shot made with 22 different mRNA strands encoding viral strains back to 1918. A clinical trial starts next year. My story for @endpts.com has all the details endpoints.news/startup-rais...

Exclusive: Khosla Ventures leads $12M seed for Circulate Health, a startup offering $8K-$10K plasma exchange sessions at longevity clinics to boost healthspan.

If you are at #BIO2025 this morning, stop by room 251 at 9:00 am for my discussion with leaders from Aera, Alnylam, Intellia and Regeneron on "Platform Power" in genetic medicines.

In another interesting alternative to animal models, the organoid startup Parallel Bio has raised $21 million to test drugs and vaccines in its lymph node-in-a-dish technology, sourced from 100 human tissue donors. Read more in my exclusive story for @endpts.com - endpoints.news/organoid-sta...

Most biotech labs look the same, but I recently saw something incredible during my visit to Bexorg: a human brain floating in a bucket, hooked up to a complex machine to keep its constituent cells alive. Read more in my exclusive deep dive for @endpts.com: endpoints.news/rebooting-de...

Casma Therapeutics raised more than $150 million since it launched in 2018 to develop drugs that target autophagy. It's presenting its first preclinical data in Gaucher's disease this week, but recently trimmed its staff to 5 employees. Read more in @endpts.com endpoints.news/after-layoff...

DNA giant Illumina spawned an ecosystem. Now its former employees are both its customers and fiercest competitors. My latest is an in-depth look at a different kind of corporate lineage story, via @endpts.com endpts.com/how-illumina...

AI bio could learn a few things from overhyped history of CRISPR. A smart tale from @andrewedunn.bsky.social in his latest Post-Hoc for @endpts.com: endpts.com/post-hoc-ai-...

Here's a wild idea: attach a cocaine-like molecule to the surface of a torn-up adenovirus and inject it in chronic drug users to help them develop antibodies that prevent cocaine from reaching the brain. I have the details of the first clinical results in @endpts.com endpts.com/researchers-...

Crispr Therapeutics surprised me yesterday when it partnered with an siRNA company. After all, isn't CRISPR supposed to do anything that siRNA can do, only permanently? I talked to CEO Sam Kulkarni about why even he doesn't think gene editing is a panacea. @endpts.com endpts.com/crispr-thera...

In a much anticipated update for the gene insertion field, David Liu and Samuel Sternberg have figured out how to get #CRISPR associated transposases (CASTs) to insert full genes efficiently in human cells. @leileiwu.bsky.social and I have the story for @endpts.com endpts.com/david-liu-sa...

BREAKING NEWS: Last August, scientists began creating a custom #CRISPR therapy to fix a mutation responsible for one baby's debilitating disease. He was treated in February and doing better. My deep dive for @endpts.com had the details: endpts.com/philadelphia...

Patrick Hsu's startup Stylus Medicine has launched with $85 million to develop in vivo CAR-T cell therapies with a recombinase-based gene insertion technology that promises to be simpler than CRISPR-based approaches. My exclusive story for @endpts.com has the details: endpts.com/exclusive-pa...

#CRISPR isn't living up to its potential. Thousands of rare diseases that could be treated with gene editing remain untouched by drug companies. Two scientists, @urnov.bsky.social and @kiranmusunuru.bsky.social, have a plan to fix that. Read more in my story @endpts.com: endpts.com/crispr-isnt-...

The first results are in from CRISPR Therapeutics' gene editing therapy targeting ANGPTL3 to prevent heart disease. The treatment reduced LDL cholesterol and triglycerides by as much as 80%. Read more in my story for @endpts.com endpts.com/crispr-thera...

Topas Therapeutics claims success in early test of "immune tolerance" therapy for celiac disease, but many questions remain. Read more about the data from the Phase 1/2 trial in my exclusive story for @endpts.com endpts.com/topas-claims...

Vertex has dropped its #AAV gene therapy research, impacting partnerships with Affinia Therapeutics and tRNA therapy startup Tevard Biosciences, and raising questions about its #CRISPR therapies for muscular dystrophy. Read more in my scoop for @endpts.com endpts.com/vertex-drops...

CRISPR pioneer Feng Zhang's protein nanoparticle startup Aera Therapeutics is turning to lipid nanoparticles to jumpstart its pipeline as it enters the in vivo CAR-T cell therapy race. Read more in my story for @endpts.com endpts.com/feng-zhangs-...

I talked to a dozen former employees from #SalioGen Therapeutics to learn why the gene editing startup suddenly shut down last year. It's a story of how a great idea turns out to be much harder than expected. Read more in my feature for @endpts.com - endpts.com/how-saliogen...

Artios Pharma presented data on its ATR inhibitor (blocks a key DNA damage response enzyme) at #AACR25 today. 50% response (tumor shrinkage) in patients whose tumors lacked a counterpart DNA repair enzyme called ATM. Read more in my story for @endpts.com endpts.com/artios-cance...

There's been a bit of revolutionary spirit in Boston, and Donald Ingber, founding director of the @wyssinstitute.bsky.social at Harvard, doesn't mince his words when comparing the Trump Administration's attack on science to King George's blockade of Boston Harbor. wyss.harvard.edu/news/common-...

Deals with Chinese biotech companies are booming. @jaredwhitlock.bsky.social has a smart story that explains the trend, and potential political headwinds, for @endpts.com endpts.com/chinas-biote...

George Church startup Matter Bio will study long-lived animals and humans ('SuperAgers' and centenarians) to find the genetic secrets of longevity and use those discoveries as a starting point for gene therapies. Read more in my exclusive for @endpts.com endpts.com/george-churc...

A Chinese startup has achieved another first with #CRISPR. HuidaGene has injected a Cas13 (RNA editing) therapy into two kids with a severe neurodevelopmental disorder. They've also treated two kids with muscular dystrophy. Read more in my story for @endpts.com endpts.com/china-biotec...

An important story from @kylelahucik.bsky.social: The biotech slump is hurting the earliest stage companies trying to raise seed rounds, which have sunk to their lowest in years. The ripple effects could be profound. Read more in @endpts.com endpts.com/in-a-rocky-y...

Siddhartha Mukherjee's cell therapy startup Myeloid Therapeutics narrows its focus to its 'in vivo' cell therapy programs as its staff shrinks. The future of its retrotransposon-based gene editing system is unclear. Read more in my story for @endpts.com endpts.com/cell-therapy...

Some good news for the CRISPR field. Verve Tx unveils positive (but early) data from its 2nd attempt at heart disease, with strong cholesterol reduction and no safety concerns that hindered its 1st attempt. @leileiwu.bsky.social has the story for @endpts.com: endpts.com/verve-unveil...

The FDA unveiled plans to replace animal testing with alternatives like organoids. But many questions remain about how that will work and what it will mean for the safety and efficacy of first-in-human drugs. @maxgelman.bsendpts.com and I have the story for @endpts.com: endpts.com/fda-unveils-...

Tome Bio founders Jonathan Gootenberg and Omar Abudayyeh have wrangled retrotransposons into a programmable tool for targeted gene insertion. Still a lot of work to be done, but they hope it will overcome some of the complexity issues with prior tech. More in @endpts.com endpts.com/tome-bio-fou...

Alzheon's amyloid-targeting pill has failed another Phase 3 study, this time in Alzheimer's patients with two copies of the APOE4 gene that dramatically enhances the risk of dementia. Read more in my story for @endpts.com endpts.com/alzheons-amy...

I talked to base and prime editing inventor David Liu about academic science under pressure, Peter Marks' departure from the FDA, and Liu's upcoming work on "disease-agnostic" gene editing. Read more in my Q&A for @endpts.com: endpts.com/qa-david-liu...

"This was never about Covid. This is about being prepared for the next viral pandemic, which is going to come, we all know it." I heard from more than a dozen scientists whose NIH funding for antivirals and vaccines was cut in a Covid research cull. More in @endpts.com endpts.com/covid-resear...