Addition Therapeutics raises $106.5M for all-RNA (no DNA required) gene insertion therapies based on retrotransposons, aka 'jumping genes' Its part of a growing trend of gene editing companies looking beyond CRISPR. My story for @endpts.com has the details: endpoints.news/addition-the...

Western drugmakers are increasingly turning to Chinese investigator-initiated trials for a cheaper and faster way to test new drugs. “If they’re not doing it, they’re going to lose,” one US CEO told me. @leileiwu.bsky.social and I have the story for @endpts.com: endpoints.news/western-gene...

Flagship's gene writing startup Tessera Therapeutics strikes $150M partnership with Regeneron for its AATD therapy, which will start clinical trials soon. CRISPR Tx and Prime Medicine are also working on similar therapies for AATD. More details in @endpts.com: endpoints.news/gene-writing...

China biotech Zhongmou Therapeutics has developed an optogenetic gene therapy that uses an engineered alga protein to restore sight, and it hopes the approach could become a ‘one-size-fits-all’ rival to Roche's Luxturna. Read more in @endpts.com: endpoints.news/chinas-zhong...

There's been a death in a #CRISPR study, something the field has long feared. A man who was recently hospitalized with liver injury a few weeks after receiving Intellia's experimental gene editing therapy died last night. More details in my story for @endpts.com endpoints.news/patient-in-i...

Arena BioWorks shuts down after investors pull plug on big bold bet to reinvent biotech, founder Stuart Schreiber confirmed to me. He pointed to biotech macro conditions and also investors seeking bigger returns on AI and tech. Details in my story for @endpts.com: endpoints.news/arena-biowor...

Jennifer Doudna's latest startup, Azalea Therapeutics, launched with $82M. It's based on virus like particles developed by CEO Jenny Hamilton and a new CAR-T trick developed by UCSF's Justin Eyquem. Their goal? The ultimate CRISPR-based in vivo CAR-T. @endpts.com - endpoints.news/jennifer-dou...

The scientists behind Baby KJ’s life-saving CRISPR therapy have a plan to help more patients. Join me and @drewsnews.bsky.social at 9:45 for a conversation with UPenn's Kiran Musunuru and CHOP's Rebecca Ahrens-Nicklas to learn more! @endpts.com endpoints.news/post-hoc-liv...

DNA-chopping enzyme that breaks up NETs (neutrophil extracellular traps) may offer new way to fight autoimmune disease, early data from #Neutrolis suggest. The company has raised $50M from Morningside. Read more in my latest exclusive for @endpts.com. #ACR2025 endpoints.news/dna-chopping...

Causeway Therapeutics had developed a synthetic #microRNA therapy that it hopes will heal injured tendons. The company shared its Phase 2 results with me and its plans to raise a large series A for bigger trials. Read more in my latest story for @endpts.com - endpoints.news/microrna-sta...

Halda Therapeutics has made small molecule drugs called RIPTACs that inhibit an essential protein cells need to live, but only in the presence of a second protein that is primarily found in cancer cells. My story for @endpts.com on Halda's first clinical data: endpoints.news/halda-reveal...

Remix Therapeutics has revealed the first clinical data of its "mRNA degrader," a small molecule drug that forces the inclusion of a 'kill switch' into mRNA, leading to its destruction. Its first drug targets the undruggable oncogene MYB. Read more in @endpts.com: endpoints.news/remix-reveal...

Arcturus announces mixed results of its inhaled #mRNA therapy designed to produce CFTR protein in cystic fibrosis patients. And the FDA is asking for more data before reviewing its #Covid-19 vaccine for approval. My story for @endpts.com has the details: endpoints.news/arcturus-ann...

Starna Therapeutics, a Chinese biotech startup founded by mRNA and LNP scientists, has raised a $44M series B to advance its in vivo CAR-T programs after promising, albeit early, results in lupus and cancer patients. Read more in my exclusive story for @endpts.com: endpoints.news/starna-thera...

#CRISPR Therapeutics recently debuted a new gene editing tool called SyNTase editing that it described as 'a significant advance' over prime editing. Prime Medicine disagrees. My latest story for @endpts.com digs into the details: endpoints.news/crispr-thera...

British startup Genomics has spent over a decade amassing a human genotype-phenotype database from over 25,000 GWAS studies. Now it is launching a new product to get that data, and software to analyze it, into the hands of drug developers. My story for @endpts.com: endpoints.news/british-star...

Exclusive for @endpts.com: Lipid nanoparticle startup Liberate Bio has emerged with $31M in seed funding from Khosla Ventures and plans to test in vivo cell therapies targeting macrophages and monocytes in the clinic in 2026. endpoints.news/exclusive-li...

The Yale spinout Bexorg has raised a $23M series A for its cutting-edge and unconventional approch to preclinical research: testing drugs on ‘rebooted’ human brains from organ donors. My story for @endpts.com has the details: endpoints.news/bexorg-raise...

#ESGCT2025: A woman who was previously ineligible for @genethonfr.bsky.social's Crigler–Najjar syndrome gene therapy due to pre-existing immunity to AAV8 got the treatment after Hansa Bio's antibody-chopping drug imlifidase cleared the way. Read more in @endpts.com : endpoints.news/new-study-su...

At #ESGCT2025, Mammoth Bio presented the first monkey data of its lead program, a gene editing therapy that uses its small Cas-Phi enzyme to target APOC3 for triglyceride reduction. A clinical trial is planed for the second half 2026. More in my story for @endpts.com: endpoints.news/mammoth-bios...

At #ESGCT2025, Seppo Ylä-Herttuala from the University of Eastern Finland reported success in Phase 2b study of a gene therapy that uses a surgical technique to inject adenoviruses containing the VEGF-D gene into ischemic heart tissue. My story for @endpts.com: endpoints.news/finnish-rese...

At #ESGCT2025, the scientists behind Baby KJ’s custom #CRISPR gene editing therapy said they are planning a clinical trial to help more children and turn the one-off drug into an approvable, repeatable procedure. My story for @endpts.com has more details. - endpoints.news/qa-scientist...

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...