@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...

Gene editing pioneers Jennifer Doudna and @urnov.bsky.social have launched the first startup, Aurora Therapeutics (led by Ed Kaye), focused on making custom #CRISPR therapies that fix a patient's unique genetic mutation. My story for @endpts.com has the details: endpoints.news/gene-editing...

Scoop: Rampart Bioscience, which launched with $125 million in Oct. 2023, has shut down. It's at least the fourth company to stop work on the very challenging task of creating non-viral gene therapies in recent years. More details in my story for @endpts.com: endpoints.news/secretive-ra...

It’s been a tumultuous couple of years for Vizgen and the broader spatial biology field. But with its 10X Genomics and Harvard litigation settled, Vizgen is looking to ramp up production and sales of its tools. Read more in my story for @endpts.com - endpoints.news/vizgen-raise...

Scoop: The well funded gene editing startup Tessera Therapeutics is cutting its headcount by 90 employees (about 35% of its workforce) just a month after landing a $150M partnership with Regeneron for its AATD therapy. Read more in my story for @endpts.com: endpoints.news/tessera-ther...

InduPro, a startup founded by former Merck scientists who invented MicroMapping—a way to identify protein interactions—has struck a partnership with Eli Lilly worth up to $950M to develop 3 bispecific or trispecific cancer drugs. More in my exclusive for @endpts.com: endpoints.news/lilly-partne...

The gene editing toolbox has exploded since CRISPR was invented, with each new tool promising to be better than the last. Read my third deep and final deep dive of 2025 on the #CRISPR gene editing field's growing pains in @endpts.com endpoints.news/as-gene-edit...

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...