Ryan Cross
@scienceboss.bsky.social
Senior Science Correspondent at Endpoints News. Reach out privately on Signal: RyanCross.25
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...
Patient in Intellia CRISPR trial dies after being hospitalized with liver injury
Patient in 80s dies after receiving Intellia Therapeutics' CRISPR therapy nex-z for ATTR. Death follows liver injury; exact cause unclear. CEO John Leonard announces trial pause.
endpoints.news
November 7, 2025 at 3:46 AM
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...
Arena BioWorks shuts down after investors pull plug on bold bid to reinvent biotech model
Arena BioWorks, backed by $500M from billionaires, shuts down after less than 2 years. Founder Stuart Schreiber cites biotech market conditions and investors seeking AI opportunities.
endpoints.news
November 4, 2025 at 11:31 PM
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...
Jennifer Doudna startup launches with $82M to apply new CRISPR delivery technology to in vivo CAR-T
Nobel laureate Jennifer Doudna's Azalea Therapeutics raises $82M to develop new CRISPR delivery system for in vivo CAR-T cell therapies targeting blood cancers.
endpoints.news
November 4, 2025 at 1:37 PM
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...
Watch at 9:45 a.m.: Meet the scientists behind Baby KJ’s life-saving CRISPR therapy
U Penn's Musunuru and CHOP's Ahrens-Nicklas discuss plans to expand Baby KJ's custom CRISPR therapy for rare genetic diseases into an FDA-approved treatment.
endpoints.news
October 31, 2025 at 12:59 PM
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...
Exclusive: Breaking up immune webs called NETs may offer new way to treat autoimmune disease, early data suggest
Neutrolis tests enzyme therapy targeting neutrophil extracellular traps (NETs) in Phase 1 trial, showing promise for autoimmune diseases. Drug cleared symptoms in teen with DNASE1L3 deficiency.
endpoints.news
October 29, 2025 at 1:41 PM
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...
Exclusive: Scottish microRNA startup claims signs of success in Phase 2 tendinopathy study
Causeway Therapeutics' microRNA drug for tennis elbow missed primary endpoint in Phase 2, but showed promise in properly injected patients. Plans Phase 3 amid $150M+ fundraising.
endpoints.news
October 27, 2025 at 4:34 PM
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...
Halda's first clinical test of RIPTAC drugs suggests a safer way to kill cancer
Halda Therapeutics reports promising data for HLD-0915 in prostate cancer trial, with 59% of patients showing PSA reduction of 50%+ after two treatment cycles.
endpoints.news
October 24, 2025 at 6:35 PM
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...
Remix Therapeutics reveals first signs of tumor shrinkage with its small molecule mRNA degrader
Remix Therapeutics, which is developing a new way to shut down the expression of cancer-causing genes, has seen the first signs of success in a rare salivary gland cancer.
endpoints.news
October 24, 2025 at 2:15 PM
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...
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...
Arcturus’ stock halved after mixed results of mRNA cystic fibrosis therapy, Covid-19 vaccine delay
The biotech's ambitious attempt to restore the critical protein that is broken or missing in people with cystic fibrosis has yielded mixed results in an ongoing Phase 2 study.
endpoints.news
October 22, 2025 at 7:51 PM
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...
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...
Exclusive: China biotech raises $44M to advance in vivo CAR-T programs after early clinical data
Chinese biotech Starna Therapeutics reports early success in clinical tests of in vivo CAR-T therapy, eliminating B cells in lupus and lymphoma patients, raising $44M Series B from LYFE Capital and ot...
endpoints.news
October 22, 2025 at 2:25 PM
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...
CRISPR Therapeutics says its new gene editing tool is 'a significant advance' over prime editing. Prime Medicine disagrees
CRISPR Therapeutics unveils SyNTase editor tool, plans AATD trial for 2026. Prime Medicine disputes novelty, suggesting similarities to their prime editing tech. Licensing talks reported.
endpoints.news
October 21, 2025 at 1:55 PM
#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...
British startup Genomics launches business to help drug developers
Oxford spinout Genomics launches Mystra, an AI tool for drug development, using vast genetic database. CEO Peter Donnelly courts pharma leaders from Eli Lilly, Novartis, Roche & Takeda.
endpoints.news
October 16, 2025 at 7:55 PM
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...
Exclusive: Lipid nanoparticle startup emerges with $31M from Khosla for in vivo cell therapies
Liberate Bio raises $31M seed funding from Khosla Ventures, developing in vivo cell therapy using monocytes and macrophages to fight cancer and other diseases.
endpoints.news
October 15, 2025 at 1:41 PM
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...
Yale spinout raises $23M to test drugs on ‘rebooted’ human brains from organ donors
Bexorg has collected $23 million in a Series A raise to scale up its human brain experiments, bringing its total funding to $42.5 million, the startup announced Wednesday.
endpoints.news
October 15, 2025 at 1:25 PM
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...
endpoints.news/new-study-su...
Person previously ineligible for gene therapy granted treatment after antibody-chopping drug clears the way
Study shows Hansa's Idefirix helps deliver Genethon's AAV8 gene therapy GNT003 in previously ineligible Crigler-Najjar patient by clearing antibodies, reports Do Cao at ESGCT.
endpoints.news
October 10, 2025 at 7:16 PM
#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...
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...
endpoints.news/mammoth-bios...
Mammoth Biosciences is getting closer to finding out if its big bet on tiny CRISPR enzymes will pay off
Mammoth Biosciences reveals monkey data for CRISPR therapy targeting APOC3 gene to lower triglycerides, with CEO Trevor Martin planning clinical trials in 2026.
endpoints.news
October 9, 2025 at 2:31 PM
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...
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...
endpoints.news/finnish-rese...
Finnish researchers report success in mid-stage study of heart disease gene therapy
Researchers from the University of Eastern Finland said a gene therapy for people with refractory angina, a form of chronic chest pain that persists despite treatment, was successful in a Phase 2b stu...
endpoints.news
October 9, 2025 at 12:49 PM
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...
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...
Q&A: The scientists behind Baby KJ’s custom CRISPR drug are planning a trial to help more children
Scientists led by Kiran Musunuru plan clinical trial for custom gene editing therapies, following success with
endpoints.news
October 9, 2025 at 10:17 AM
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...
Arbor partners with Chiesi Group in gene editing deal with potential $2B future payments
Chiesi Group partners with Arbor Biotechnologies in CRISPR deal worth up to $2B for PH1 therapy and rare liver diseases, following first patient dosing in clinical trial.
endpoints.news
October 6, 2025 at 6:52 PM
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!
October 2, 2025 at 12:45 PM
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...
Exclusive: French startup's gene therapy dramatically reduces urinary incontinence in spinal cord injury patients
EG 427's gene therapy shows 88% reduction in urinary incontinence for spinal cord injury patients at 12 weeks, using herpesvirus vector to deliver botulinum toxin F to bladder.
endpoints.news
October 1, 2025 at 3:31 PM
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...
Exclusive: Biogen calls it quits on AAV gene therapy, again
Biogen is ending its gene therapy work that relies on adeno-associated viruses, or AAVs, the company confirmed to Endpoints News, making it the latest pharma to walk away from the viral-based delivery...
endpoints.news
September 25, 2025 at 6:42 PM
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...
Exclusive: Aera Therapeutics jumps into in vivo CAR-T race with trial planned for 2026
Aera Therapeutics is the latest company hoping that a simple infusion can reprogram the immune system to wipe out errant cells responsible for autoimmune disease. It's planning on testing the in vivo ...
endpoints.news
September 23, 2025 at 7:07 PM
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...
The Endpoints 11 list: Meet the most exciting biotech startups of 2025
Announcing the 2025 Endpoints 11 winners — the most exciting, high-risk, high-reward biotech startups of the year.
endpoints.news
September 19, 2025 at 6:04 PM
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...
Q&A: Lasker Award winner Michael Welsh on the science that led to life-saving cystic fibrosis drugs
Scientists Welsh, González, and Negulescu win Lasker Award for cystic fibrosis research, including Welsh's molecular insights and Vertex's Trikafta drug development.
endpoints.news
September 11, 2025 at 1:30 PM
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...