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...

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...