bohlen.bsky.social

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,
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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 https://www.biorxiv.org/content/10.64898/2025.12.26.696588v1

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…

We are reaching an inflection point in gene therapies where money and businesses are the thing that is causing gene therapies to fail, not the science.

Gene therapy trial death unnerves promising area of brain research - United States News Beep Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies reported they had engineered viruses that could

Gene therapy trial death unnerves promising area of brain research

https://www.newsbeep.com/us/350182/

Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies…

Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market is projected to reach USD 12.87 billion by 2034 - openPR.com Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market is projected to reach USD 12.87 billion by 2034  openPR.com

Signal or noise? > Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market
is projected to reach USD 12.87 billion by 2034 -
openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #healthcare #pharmaceutical #biotech #pharma

Early Results Amaze for One-Time Dravet Syndrome Treatment Drug aims to correct genetic defect

Young children with Dravet syndrome improved dramatically with a novel gene therapy, according to initial results from a phase I/II trial reported at #AES2025. @amepilepsysoc.bsky.social
www.medpagetoday.com/meetingcover...

Tripartite AAV Systems for EYS Retinal Gene Therapy https://www.biorxiv.org/content/10.64898/2025.12.03.692187v1

Reviewing the tissue distribution, transfection rates and #Immunogenicity of recombinant adeno-associated virus serotype 8 (#AAV8) in gene therapy, with insights into the applications and challenges of using AAV8 as a vector.

#OpenAccess: doi.org/10.1016/j.ge...

I'm obviously biased, but completely agree! Hope you are doing well, Doug!

Precerebellar paramedian tract cell groups connections: I. Oculomotor internuclear projections - Brain Structure and Function Brain Structure and Function - The role of internuclear neurons residing in the medial rectus subdivision of the oculomotor nucleus and projecting to the abducens nucleus is poorly understood. As a...

Here are the papers:
link.springer.com/article/10.1...

link.springer.com/article/10.1...

Together, the findings reveal a unified, bilateral pathway through which the brainstem shares eye-movement commands with the cerebellum.
This shared access point may help the cerebellum compare intended vs. actual eye movements and fine-tune them in real time.

And it doesn’t stop there:
These internuclear pathways also send inputs to both the flocculus and the fastigial nucleus, two cerebellar structures critical for refining conjugate and vergence eye movements.

Both OINs and AINs send efference copy signals far beyond their traditional motoneuron targets.
We show they project to the paramedian tract (PMT) cell groups, a set of precerebellar nuclei long known but not fully appreciated in this context.

Classically, oculomotor (OIN) and abducens (AIN) internuclear neurons are thought of mainly as the neurons that link the two eyes for coordinated horizontal movements.
But it turns out they do much more.

For the oculomotor nerds out there… we’re excited to share our paired papers on precerebellar circuitry!
These two studies look at how internuclear pathways talk to the cerebellum and what that means for eye movement control. 👀🧠 #neuroskyence #science