After 25 years of brave & brilliant work by hundreds of scientists in my lab to understand then safely reverse aging for the first time, it was moving to witness the first human dose being delivered 🥹 https://t.co/veQsyUEORz
Cheers, chills, and a standing ovation when RASolute 302 showed unprecedented survival on daraxonrasib for patients with progressive pancreatic cancer
Seldom do you sense you’re witnessing a historic moment in cancer care but this feels like ras targeting has arrived
#ASCO26
$LLY
ASCO 2026: Lilly exec points to ‘nutty’ 100% response rate for in vivo CAR-T as justifying Kelonia buyout
- Kelonia shared the largest slice of data yet for its in vivo CAR-T, helping to justify the $3.2 billion price tag Lilly recently paid for the biotech.
https://t.co/USromttwns
From clinical progress to community perspectives, our latest Congressional briefing explored next-generation approaches for genetic hearing loss. Thanks to @RepKevinMullin for your leadership on biomedical innovation. Read more: https://t.co/EI2dPu151g
#ASGCTAdvocacy
Our medicine for certain children aged 2-11 years living with uncontrolled chronic spontaneous urticaria (CSU) has been approved by the FDA. Read more: https://t.co/NrAppCp2jM
CC: @sanofi
Before a gene therapy reaches clinical trials, it has to pass the "pre-IND" milestone. How can patient groups influence this critical FDA meeting?
Join Jocelyn Duff (@CureCMT4J) and Claire Marasco (@ForgeBio) for a quick 10-minute dive into early regulatory strategy: https://t.co/kuuO4etiho
Aging can now be decoded 🧬
Scientists are identifying #biomarkers across cells, tissues, and organs
to track how the body ages—step by step.
aging is now measurable at every level
We’re not guessing anymore.
We’re mapping it.
Read:
https://t.co/XkA7HPkf4r
#Aging#Longevity
Today, President Trump announced plans to impose a 100% tariff on certain patented pharmaceuticals and their active ingredients. Under the recent Executive Order, cell and gene therapies will be exempt.
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https://t.co/jN1fqUlWyI
The cell and gene therapy (CGT) community welcomes the FDA’s accelerated approval of KRESLADI, Rocket Pharma’s gene therapy to treat severe Leukocyte Adhesion Deficiency-I. 1/4
https://t.co/ysgrLlRe8i
Announcing a new event! ARM is co-hosting a hybrid workshop with the FDA's Center for Biologics Evaluation and Research, Office of Therapeutic Products, on Advancing Pediatric Cell and Gene Therapy Clinical Trials on April 9, 2026.
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The world’s largest strategic biopharma companies see the promise of CGT. Of the 30 largest biopharma companies by market cap, 20 are invested in the development or commercialization of CGT.
Read ARM's full ‘Reasons to Believe’ analysis here: https://t.co/Aw2QbDA5hQ
CGT’s 2025 #YearInReview | Leading cell and gene therapy organizations call for a 10-year moratorium on human heritable genome editing
In March, ARM, ISCT, and the ASGCT released a declaration calling for a 10-year global moratorium on heritable human genome editing.
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At last weeks PSC-Derived Cell Therapies Symposium, SereNeuro Therapeutics, a preclinical biotechnology company developing non-opioid pain therapies, unveiled new data on a novel approach to chronic pain management & joint tissue preservation. Learn more: https://t.co/AOYco2qJ0i
🚨 BIG WIN FOR THE #RareDisease COMMUNITY! 🚨
Yesterday, the House passed the #GiveKidsAChanceAct – a major step forward for the Rare #Pediatric Disease Priority Review Voucher (#PRV) program, which has helped bring 63 innovative treatments to children.
Our work isn’t done. Contact your Senators and urge them to pass this bill: https://t.co/rwmWCw6upK
That @US_FDA is recognizing here the urgent need for regulatory strategies to evolve to match the pace of scientific advances in ways that streamline bringing the science safely to patients is incredibly important. I hope these new policies provide a foundation that will eventually allow certain kinds of genetic surgeries to become as routine as life-saving conventional surgeries, in which the process is robust and standardized enough to be rapidly adapted to each patient, without the need for a separate multi-year regulatory process for each surgery.
Today, FDA Commissioner Marty Makary and CBER Director Vinay Prasad released the agency's new plan for a “plausible mechanism pathway” to help patients more quickly access personalized gene editing therapies.
https://t.co/BBGU9AluGD
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