Treating fetuses with stem cells proves safe in milestone spina bifida trial The study is now scaling up to do more in utero procedures and evaluate effects on disability as children grow

Honored to be part of this trial, led by my amazing colleague Dr. Farmer, with much teamwork. Thank you for funding from @cirmnews.bsky.social (CIRM). 🫶
Treating fetuses with stem cells proves safe in milestone spina bifida trial | Science | AAAS www.science.org/content/arti...

She Was Born Without an Immune System. Gene Therapy Saved Her Life.

She Was Born Without an Immune System. Gene Therapy Saved Her Life. www.nytimes.com/2025/11/27/w...

Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy Pivotal Phase 3 randomized, double-blind, placebo-controlled study (n=106) met the primary endpoint (PUL v2.0) and the key secondary cardiac…...

Congratulations to Capricor, PI Dr. Craig McDonald of
@ucdavishealth.bsky.social & the patients for these exciting results. Thank you to our @cirmnews.bsky.social
- funded Alpha Clinic for assisting with the trial logistics & product formulation and delivery. 🙏

www.capricor.com/investors/ne...

Judge Orders Trump Not to Threaten University of California’s Funding

Thank you Judge Lin!

“As a part of Friday’s ruling, Judge Lin said the defendants in the case, including Mr. Trump and an array of government agencies, could not link federal funding “to any measures” that would violate the First Amendment.”

www.nytimes.com/2025/11/14/u...

Baby KJ's doctors will ask FDA this winter to OK a clinical trial of pioneering gene editing approach Baby KJ's medical team intend to ask the FDA to start a clinical trial that would be open to patients with seven different urea cycle disorders.

Baby KJ; doctors will ask FDA this winter to OK a clinical trial of pioneering gene editing approach.
#genetherapy
#baseediting
#crispr
#geneediting

www.statnews.com/2025/10/16/b... via @statnews.com

#NORDSummit: Lee Greenwood, whose 5-year-old daughter Noa was born with #CanavanDisease, a severe #GeneticDisorder found mainly among Ashkenazi Jews. In June 2022, the girl received lifesaving #GeneTherapy and is now developing normally.
@nordrare.bsky.social

#RareDisease #RareSky #MedSky

A new review highlights the advances in DNA nucleases and delivery vectors for in vivo #GeneTherapy, outlining key developments, challenges, and their future potential to treat diverse #Diseases. #medsky

#OpenAccess: www.sciencedirect.com/science/arti...

This Month on HemeHub: SCD and Gene Therapy

Join us as we explore advances in the treatment of #sicklecelldisease through #genetherapy, featuring pivotal trials of Lyfgenia and Casgevy.

Stay tuned for our upcoming session featuring Dr. John Tisdale, Senior Investigator at the NHLBI.

basicscience.ucdmc.ucdavis.edu/beleford-lab...

Press Releases | Investors & Media Reimagining the future of healthcare through the power of gene therapy.

Congratulations to ⁦‪UniQure and to the #huntingtonsdisease community! 💙💜

www.uniqure.com/investors-me...

AABB’s Cell and Gene Therapy Standards for Pharmacy, First Edition, Takes Effect October 1 | BioInformant Sept 8, 2025, Bethesda, MD – The Association for the Advancement of Blood & Biotherapies (AABB) is pleased to announce the release of the first edition of Cell and Gene Therapy Standards for Pharmacy,...

AABB’s Cell and Gene Therapy Standards for Pharmacy, First Edition, Takes Effect October 1 - bioinformant.com/aabbs-cell-a...
@aabbupdates.bsky.social

ScaleReady Awards G-Rex® Grants to Support Cell and Gene Therapy Manufacturing in California /PRNewswire/ -- ScaleReady, in collaboration with Wilson Wolf Manufacturing, Bio-Techne Corporation (NASDAQ: TECH) and CellReady, today announced that eight...

Congratulations to Brian Fury, our GMP Facility Director at UC Davis Health @ucdavis.bsky.social, and to all of the other recipients of these G-Rex grant awards from ScaleReady.

www.prnewswire.com/news-release...

A calico cat looks out from a box with text below stating a new cancer drug could help both cats and people, credited to UC Davis.

UC Davis & @ucsanfrancisco.bsky.social scientists completed a clinical trial on a new cancer drug for pet cats with squamous cell carcinoma. Researchers believe it will also be effective for humans with similar head and neck cancers. Read more about the clinical trial: ucdav.is/46dxcfM

Enjoy the conference! ☺️

Momentary break for a much-needed palate cleanser. 🫠
Never knew these adorable creatures existed until this moment. (and nope, it is not AI- they are real! 🥰)

Dream team to develop in utero gene editing therapy for a rare neurological genetic disorder Professor Aijun Wang won a $3.2 million NIH grant to develop a cure to Dup15q syndrome using an in-utero gene editing formula.

I am so honored to be a part of this team. Thank you to the NIH for starting up their funding again. Please hang in there, fellow scientists!
health.ucdavis.edu/medical-scho...

Human brain vascular multi-omics elucidates disease-risk associations Reid et al. develop MultiVINE-seq to map thousands of non-coding disease variants to genes in human brain vascular cells. Cerebrovascular disease variants compromise vessel integrity, whereas Alzheime...

🚨 NEW PUBLICATION 🚨 "Human brain vascular multi-omics elucidates disease-risk associations"

www.cell.com/neuron/abstr...

@ucsanfrancisco.bsky.social
@madiganreid.bsky.social
@cellpress.bsky.social

Want to collaborate with the International Brain Laboratory on your own project? We have funding to work with groups anywhere in the world to do new large-scale projects and we are looking for new partners! Learn more and apply: www.internationalbrainlab.com/ibl-core-apply

House appropriators snub Kennedy, include mRNA vaccine funding in spending bill Lawmakers have snubbed health secretary RFK Jr. by including an amendment in a spending bill mandating that an agency under his purview continue to research messenger RNA vaccines.

House appropriators snub Kennedy, include mRNA vaccine funding in spending bill www.statnews.com/2025/09/10/m... via @statnews.com

'An attack on knowledge': UCLA researchers say lifesaving research is in jeopardy - Daily Bruin Dr. Jerzy Kupiec-Weglinski has led organ transplant research at UCLA for nearly three decades. Kupiec-Weglinski, the vice chairman of basic science research in the David Geffen School of Medicine’s su...

The regime halted all federal grants then told UC administration if they pay $1 billion they could have them back.
It’s like ransomware.
UC is a public university system and we cannot do this.
So, no grants = no research and labs will shut down.
Are we great yet?

dailybruin.com/2025/08/09/a...

'An attack on knowledge': UCLA researchers say lifesaving research is in jeopardy - Daily Bruin Dr. Jerzy Kupiec-Weglinski has led organ transplant research at UCLA for nearly three decades. Kupiec-Weglinski, the vice chairman of basic science research in the David Geffen School of Medicine’s su...

The regime halted all federal grants then told UC administration if they pay $1 billion they could have them back.
It’s like ransomware.
UC is a public university system and we cannot do this.
So, no grants = no research and labs will shut down.
Are we great yet?

dailybruin.com/2025/08/09/a...

Trump administration violated impoundment law by canceling NIH grants, slowing new awards, GAO finds The Government Accountability Office found that the Trump administration, by abruptly canceling NIH grants, had violated a 1974 federal law

Trump administration violated impoundment law by canceling NIH grants, slowing new awards, GAO finds www.statnews.com/2025/08/05/g... via @statnews.com

Good morning and happy Saturday. Take a deep breath and keep riding those waves. 🙏

Stimulating immune cells in vitro is a common experimental lab model. We profiled 150K blood immune cells treated with 11 different stimuli to compare the effects. The data are freely available for researchers. See the preprint for our findings and to access the data 🧪 doi.org/10.1101/2025...

Bioengineered immunocompetent preclinical trial-on-chip tool enables screening of CAR T cell therapy for leukaemia - Nature Biomedical Engineering An ex vivo organotypic and immunocompetent human leukaemia chip system is used for CAR T cell therapy modelling and screening of patient response.

Bioengineered immunocompetent preclinical trial-on-chip tool enables screening of CAR T cell therapy for leukaemia. #Tcell #immunotherapy #organonachip 🧪https://www.nature.com/articles/s41551-025-01428-2

World’s Premier Cancer Institute Faces Crippling Cuts and Chaos - KFF Health News After spearheading a 34% cut in cancer mortality, the National Cancer Institute at the NIH is bleeding resources and staff and could see its budget cut by nearly 40%.

After spearheading a 34% cut in cancer mortality, the National Cancer Institute at the NIH is bleeding resources and staff and could see its budget cut by nearly 40%. kffhealthnews.org/news/article...

Federal judge indefinitely blocks Trump administration from cutting off Harvard’s ability to host foreign students | CNN Politics A federal judge on Friday indefinitely blocked the Trump administration from revoking Harvard University��s ability to host international students and scholars while legal challenges continue.

www.cnn.com/2025/06/20/p...

Kennedy Says ‘Charlatans’ Are No Reason to Block Unproven Stem Cell Treatments

Explore this gift article from The New York Times. You can read it for free without a subscription.

www.nytimes.com/2025/06/05/h...

And this is your new improved FDA. Into everyone’s medical life, a few charlatans must fall.

Specific targeting of brain endothelial cells using enhancer AAV vectors Velazquez-Rivera et al. have developed and validated a set of new BEC-enhancer AAV vectors that allow for selective genetic labeling of, and access to, brain endothelial cells (BECs). These useful too...

"Specific targeting of brain endothelial cells using enhancer AAV vectors"
www.cell.com/neuron/fullt...

Xiangmin Xu & colleagues
@cp-neuron.bsky.social

😢😢😢😢