Penn Medicine, Children's Hospital of Philadelphia team awarded Breakthrough Prize for developing gene therapy for inherited blindness - United States /PRNewswire/ -- Their discovery started with a group of blind dogs living at a vet school. Now, the work has been awarded the prestigious Breakthrough Prize at...

Penn Medicine, Children’s Hospital of Philadelphia team awarded Breakthrough Prize for developing gene therapy for inherited blindness

https://www.europesays.com/us/737665/

Today, Jean Bennett, MD, PHD, and Albert Maguire, MD, both emeritus professors of Ophthalmology in the …#us #news #usnews

Well this is an instant classic and fabulous demonstration of why implementation details are so important.

If you are an early career NHP researcher (postdoc or asstiant professor) and interested in attending #ACNP2027 please apply.

I’ve also rolled off the E&T Committee that reviews these awards and am now an “ambassador” so if anyone wants some advice about applying for travel awards, reach out.

And an agent

Instead of analyzing the IBL dataset, use your computational skills to analyze this dataset and your results will be 1,000 times more translationally relevant

Nonuniform scaling of cerebellar cortical-nuclear architecture across primates revealed by cross-species atlases www.biorxiv.org/content/10.64898/2026.04...

grants.nih.gov/news-events/...
Please please please fill this out and speak up for animal research, especially primate research. So many diseases of the body and brain will only be cured if we support the models that best reflect human biology.

A visual representation of an adeno-associated virus

An 𝐚𝐝𝐞𝐧𝐨-𝐚𝐬𝐬𝐨𝐜𝐢𝐚𝐭𝐞𝐝 𝐯𝐢𝐫𝐮𝐬 is a naturally occurring, nonpathogenic virus that has been adapted for use as a vector for gene therapy, in which part of the original genome of the virus is replaced with a transgene cassette. Full definition: nej.md/4scg4j1

#ScienceSky

Targeted AAV6 gene therapy restores corneal endothelial function in three hereditary corneal dystrophies Zhang et al. report an AAV6-based intracameral gene delivery strategy that selectively transduces corneal endothelium, achieves long-term transgene expression, and preserves corneal transparency across multiple models of hereditary corneal dystrophy.

Online now: Targeted AAV6 gene therapy restores corneal endothelial function in three hereditary corneal dystrophies

Jill Wood, who runs a company that researches gene therapy for her son Jonah's rare disease, relies on funding from the SBIR and STTR programs. But the programs have been paused as they await congressional reauthorization.

What if gene regulation can treat epilepsy? Early trials of zorevunersen (a gene therapy) show ~85% seizure reduction in some kids with Dravet syndrome. How does this change our view of genetic disease treatment?

Mass General Brigham snags $50M donation for gene therapy facilities, clinical trials - Boston Business Journal Mass General Brigham continues to build its coffers to fund new cancer research as the health care giant braces for its split from Dana-Farber Cancer Institute.

Mass General Brigham snags $50M donation for gene therapy facilities, clinical trials

www.bizjournals.com/boston/news/...

A NeuroD1 AAV‐Based Gene Therapy for Functional Brain Repair in Alzheimer's Disease‐Like Non‐Human Primate Model

Pfizer pushes forward playing God with new venture in permanently altering DNA
After gene therapy exit, Pfizer locks in global license for Beam gene editing candidate

After dumping its sole remaining gene therapy asset last year,
1

Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment For all its blockbuster hopes and high-profile status in the gene therapy field, Roctavian's commercial saga has ended.

Wow. That’s really heartbreaking.

Failing to find a buyer, BioMarin pulls Roctavian in gene therapy's biggest disappointment

The FDA creates a quicker path for gene therapies
->NPR | More on "FDA gene therapy rare diseases" at BigEarthData.ai

Neuroscience has a species problem If neuroscience is serious about building general principles of brain function, cross-species dialogue must become a core organizing principle.

The biggest problem holding neuroscience back right now isn’t data or tools, thanks in large part to the BRAIN Initiative.

It’s fragmentation across species. I wrote this to hopefully spark discussion around an issue that can only be solved as a community👇

www.thetransmitter.org/animal-model...

Lilly stops development of three drugs, including gene therapy - United Kingdom Eli Lilly confirmed it was terminating the development of three candidates. Image credit: Jennie Book / Shutterstock.com

Lilly stops development of three drugs, including gene therapy

https://www.europesays.com/uk/748013/

Eli Lilly confirmed it was terminating the development of three candidates. Image credit: Jennie Book / Shutterstock.com Eli…

UK and Japan strengthen science and technology ties The UK and Japan have announced a sweeping pharmaceutical and technology partnership aimed at accelerating drug discovery.

The UK and Japan strengthen R&D ties with an £11m investment in gene therapy manufacturing and a national rare disease genomics pilot. The partnership leverages AI and quantum computing to accelerate drug discovery.

Learn more on the collaboration: www.emjreviews.com/emj-gold/new...

After NINDS director ouster, 40 neuroscience organizations press U.S. Congress for oversight over hiring process A letter signed by the groups asks Congress to ensure that scientific expertise remains a priority in the search for a new director of the National Institute of Neurological Disorders and Stroke.

A letter signed by 40 neuroscience groups asks Congress to ensure that scientific expertise remains a priority in the search for a new director of the National Institute of Neurological Disorders and Stroke.

By @avaskham.bsky.social

#neuroskyence

www.thetransmitter.org/science-and-...

Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease - Yahoo Finance Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease  Yahoo Finance

Signal or noise? > Belief BioMed Congratulates Partner AskBio on IND Acceptance
by FDA for Investigational Gene Therapy for Late-Onset Pompe
Disease - Yahoo Finance >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #pharmaceutical #healthcare #pharma #biotech

Hope for haploinsufficiency diseases Genetic conditions like Dravet syndrome, which causes severe childhood epilepsy, are hard to tackle with traditional gene therapy. New approaches in the works include using antisense therapy to boost…

Genetic conditions like Dravet syndrome, which causes severe childhood epilepsy, are hard to tackle with traditional gene therapy. New approaches in the works include using antisense therapy to boost mRNA splicing.

✍️ Elie Dolgin

knowmag.org/3XlmYFm

FDA touts new flexible approach to reviewing cell and gene therapies The US Food and Drug Administration (FDA) has announced it will take a flexible approach to reviewing all new cell and gene therapy (CGTs) biologics license applications (BLAs) to expedite the development of these products, rather than applying its flexibilities on a case-by-case basis. @

🚀 The biggest #JPMWeek2026 win you missed: The FDA is easing manufacturing rules (21 CFR 211) for Cell & Gene Therapy.

Lower costs. Faster trials. Massive win for biotech startups. 🧬

Read: www.raps.org/news-and-art...

Belief BioMed Celebrates FDA IND Acceptance for AskBio's Gene Therapy Aimed at Pompe Disease Treatment Belief BioMed proudly announces AskBio's FDA IND acceptance for AB-1009, a gene therapy for late-onset Pompe disease, marking a vital milestone.

Belief BioMed Celebrates FDA IND Acceptance for AskBio's Gene Therapy Aimed at Pompe Disease Treatment #Shanghai #USA #gene_therapy #Belief_BioMed #AskBio

AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD) - Canada News Beep Durham, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) --

AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)

https://www.newsbeep.com/ca/397792/

Durham, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) — AB-1009 program advances to Phase 1/Phase 2; clinical trial …

A Fort Collins family is trying to raise millions to test gene therapy that could help kids trapped in bodies they can’t move Everly Green, whose family lives in Fort Collins, has a rare mutation in a gene called FRRS1L, pronounced “frizzle,” which affects how cells in her brain communicate.

A Fort Collins family is trying to raise millions to test gene therapy that could help kids trapped in bodies they can’t move trib.al/sf9KaTj

Retrograde optogenetics reveals sensorimotor convergence within a corticotectal pathway of non-human primates Yu et al. demonstrate that diverse frontal eye field signals converge on motor-selective superior colliculus neurons to generate saccades, revealing visuomotor convergence and establishing retrograde ...

I’m excited to share our new Current Biology paper! We use retrograde optogenetics in non-human primate and revealed a visuomotor convergence mechanism in the FEF-SC pathway.
www.cell.com/current-biol...

Decoupling Efficacy from Toxicity: Engineering Spatial Control in AAV-Mediated Gene Therapy www.biorxiv.org/content/10.64898/2025.12...

Child’s sudden death unnerves a promising area of gene therapy research Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient died.

Child’s sudden death unnerves a promising area of gene therapy research www.statnews.com/2025/12/15/g... via @statnews.com

EpilepsyGTx Secures $33M to Advance Targeted Gene Therapy for Epilepsy Focal refractory epilepsy (FRE) affects millions worldwide, with around 2 million patients in the US, UK, and EU alone. Individuals with FRE continue to experience seizures even after trying two appropriate and tolerated medications. These seizures originate from a specific part of the brain, making treatment challenging and leaving patients with limited options. EpilepsyGTx’s Innovative Approach Kent-based biotechnology firm EpilepsyGTx is changing the landscape of epilepsy treatment.

EpilepsyGTx Secures $33M to Advance Targeted Gene Therapy for Epilepsy

Focal refractory epilepsy (FRE) affects millions worldwide, with around 2 million patients in the US, UK, and EU alone. Individuals with FRE continue to experience seizures even after trying two appropriate and tolerated…