bohlen.bsky.social

Fear, monkeys, and institutional courage

When news broke of an explosion inside Harvard’s neurobiology building early Saturday morning, every scientist who works with monkeys felt it—an involuntary jolt, a spike of cortisol, the silent thought: what if it had been us? Neither animals nor people…

We desperately need new research tools to move the ball forward in Parkinson's. How about seeing dopamine in a new light? Enter 'far-red sensors' that unlock multiplex brain signals. Could a far-red dopamine sensor open the door to simultaneously watching multiple brain chemicals 'in action.'

Opportunities in Japan’s regenerative medicine, gene therapy markets

Japan has become a hub for regenerative medicine startups driven by innovation. The sector is expanding rapidly, supported by favourable regulations and strong market potential. Investment opportunities extend beyond core…

Single-cell, multi-region profiling of the macaque brain across the lifespan https://www.biorxiv.org/content/10.1101/2025.10.31.685880v1

Nonhuman primate research to lose federal funding at major European facility The Dutch Senate has ordered the Biomedical Primate Research Centre in the Netherlands to shift its funding away from primate experiments by 2030.

Dutch lawmakers have approved the phase-out of primate research at one of Europe’s biggest facilities. Neuroscientists are worried.

By Lauren Schenkman

#neuroskyence

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

Unconscionable.

AAV Quantification: Towards Faster, More Scalable Analysis - Bytes Europe Adeno-associated virus (AAV) is widely used in gene therapy as a vector to deliver genetic material into patient cells due to its specificity and low

AAV Quantification: Towards Faster, More Scalable Analysis

https://www.byteseu.com/1496260/

Adeno-associated virus (AAV) is widely used in gene therapy as a vector to deliver genetic material into patient cells due to its specificity and low immunogenicity. Accurate AAV quantification and …

Immagine associata al post

Teenager who received Elevidys, Sarepta's Duchenne gene therapy, dies - STAT
Sarepta Therapeutics said that a young man died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy

𝗔 𝗡𝗘𝗨𝗥𝗢𝗘𝗖𝗢𝗟𝗢𝗚𝗜𝗖𝗔𝗟 𝗣𝗘𝗥𝗦𝗣𝗘𝗖𝗧𝗜𝗩𝗘 𝗢𝗡 𝗧𝗛𝗘 𝗣𝗥𝗘𝗙𝗥𝗢𝗡𝗧𝗔𝗟 𝗖𝗢𝗥𝗧𝗘𝗫
By Mars and Passingham
"Understanding anthropoid foraging challenges may thus contribute to our understanding of human cognition"
Going to the top of the reading list!
doi.org/10.1016/j.ne...
#neuroskyence

AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact - BioSpace AviadoBio Looks to China for Eye-Based Gene Therapy in $413M UgeneX Pact  BioSpace

Signal or noise? > AviadoBio Looks to China for Eye-Based Gene Therapy in $413M
UgeneX Pact - BioSpace >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #pharma #biotech #pharmaceutical #healthcare

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.

#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...

FDA awards RMAT designation to Dravet syndrome gene therapy - Dravet Syndrome News FDA awards RMAT designation to Dravet syndrome gene therapy  Dravet Syndrome News

Signal or noise? > FDA awards RMAT designation to Dravet syndrome gene therapy
- Dravet Syndrome News >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #healthcare #biotech #pharma #competitivemarketing

A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3 Intrinsic innate immune barriers have evolved to suppress viral infection and can reduce effective gene delivery in gene therapy. We have developed BG147, a novel cyclosporine A analogue, optimised vi...

New preprint from our lab, led by our postdocs Dara Annett and Kate Morling in collaboration with Selwood Lab!

We developed a modified cyclosporine, BG147, which enhances lentivector gene therapy transduction, ex vivo in HSPC and in vivo in mouse photoreceptors, by degrading IFITM3. Check it out!

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial - Nature Medicine Interim trial results of MYR-101, a novel recombinant vector with selective tropism for oligodendrocytes, support its tolerability and indicate improved myelination and reduced cerebrospinal fluid N-acetyl-aspartic acid concentrations in children with Canavan disease.

Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial

www.nature.com/articles/s41...

Proud to share the Alzheimer’s Association invested $112.2M in research activities over the last year — our largest single-year commitment. From gene therapy to #BrainHealth to risk reduction to AI, we’re funding bold boundary-pushing ideas. alz.org/research

#ENDALZ

This teenager no longer needs lifelong blood transfusions thanks to gene transplant therapy Children’s Healthcare of Atlanta recently performed Georgia’s first commercial gene therapy transplant for a genetic disease requiring lifelong blood transfusions.

Children’s Healthcare of Atlanta recently performed Georgia’s first commercial gene therapy transplant for a genetic disease requiring lifelong blood transfusions. www.gpb.org/news/2025/09...

First Child Dosed in Gene Therapy Trial Dies Investigational agent aimed to treat rare gene-related encephalopathy

First Child Dosed in Gene Therapy Trial Dies.
www.medpagetoday.com/neurology/ge...

Congratulations! Thanks to all the animals and animal research that made this possible 🙌

Precigen (PGEN) Makes History: FDA Approves First-Ever Therapy Targeting Root Cause of RRP Precigen’s PAPZIMEOS therapy is transforming the lives of adults with recurrent respiratory papillomatosis, eliminating endless surgeries.

Feeling a bit tearful at the moment. A gene therapy for RRP, the airway disease I've had for 25 years now, has been developed and is now approved. For those of us who've had anything between 50-500 throat operations, this is an absolute life changer!
biotechhealthx.com/biotech-news...

Woof

www.geneonline.com/biotech-layo...

NCBN Trending: Gene therapy startup Kriya Therapeutics gets $313M https://zpr.io/V27wP4Znjfxt

Research Spotlight: Organoids Could Make Gene Therapy Trials Safer By Identifying Hidden Risks Early On - massgeneralbrigham.org Research Spotlight: Organoids Could Make Gene Therapy Trials Safer By Identifying Hidden Risks Early On  massgeneralbrigham.org

Thoughts on this? >> Research Spotlight: Organoids Could Make Gene Therapy Trials
Safer By Identifying Hidden Risks Early On -
massgeneralbrigham.org >> Comment below! #strategy #competitiveintelligence #marketing #pharma #competitivemarketing #biotech #healthcare #pharmaceutical

Ousted F.D.A. Vaccine Chief Returns to Agency Dr. Vinay Prasad’s rehiring was an unusual instance of a federal official targeted by the right-wing activist Laura Loomer being brought back into the Trump administration.

Dr. Vinay Prasad, who led the FDA’s vaccines and gene therapy division before resigning under pressure two weeks ago, is returning to the agency, a Department of Health and Human Services spokesman confirmed Saturday.

Family raises money for son's gene therapy and clinical trial to treat rare genetic mutation Paxton Purdy's parents said he has an ultra-rare neurodegenerative genetic mutation. They hope they can raise enough money to cure it and help children in similar situations.

Story about my autistic grandson, Paxton Purdy, who has a terminal genetic mutation called CLCN6. Now he’s at real risk of losing grants for his life saving gene therapy and for what? So billionaires can have more yachts! I just want to hang my head and cry!

www.cbsnews.com/losangeles/n...

AAV in Gene Therapy Pipeline 2025: Mechanism of Action, Route - openPR.com AAV in Gene Therapy Pipeline 2025: Mechanism of Action, Route  openPR.com

Thoughts on this? >> AAV in Gene Therapy Pipeline 2025: Mechanism of Action,
Route - openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #pharma #biotech #competitivemarketing #healthcare #pharmaceutical

Imlifidase enables gene therapy in Duchenne patients with AAV antibodies - European Biotechnology Magazine Imlifidase enables gene therapy in Duchenne patients with AAV antibodies  European Biotechnology Magazine

Thoughts on this? >> Imlifidase enables gene therapy in Duchenne patients with
AAV antibodies - European Biotechnology Magazine >> Comment below! #strategy #competitiveintelligence #marketing #pharma #pharmaceutical #competitivemarketing #biotech #healthcare

AAV2-BDNF Gene Therapy Restores FDG-PET Activity in Entorhinal Cortex, Phase 1 Data Show - NeurologyLive AAV2-BDNF Gene Therapy Restores FDG-PET Activity in Entorhinal Cortex, Phase 1 Data Show  NeurologyLive

Thoughts on this? >> AAV2-BDNF Gene Therapy Restores FDG-PET Activity in
Entorhinal Cortex, Phase 1 Data Show - NeurologyLive >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #pharma #healthcare #biotech #pharmaceutical

Study Highlights Need for Standardized Measurement Methods in Gene Therapy - United Kingdom News Beep Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s

Study Highlights Need for Standardized Measurement Methods in Gene Therapy

https://www.newsbeep.com/uk/28256/

Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic…

The deadly saga of the controversial gene therapy Elevidys The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

The deadly saga of the controversial gene therapy Elevidys buff.ly/tJneVRW

A good day to be inside doing science.