1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at nejm.org/doi/full/10.10…. It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (nejm.org/doi/full/10.10…).

No company is too small to partner with us. Nearly three-quarters of our clients are new and emerging companies. Connect with us to find out how we can support your development journey: bit.ly/3nJ5dfY #deliveredwith #drugapproval #drugapprovals #solvedwith #CMO

Fetal mice exposed to low doses of BPA showed signs of disrupted development in the circadian “clock” that regulates the rhythms of daily activity. Science Advances fcld.ly/o2ktjkc

Neurogene employees participated in the BDSRA Foundation Virtual 5K and mailed postcards in honor of Batten Disease Awareness Day today. How are you helping raise #Battendisease awareness today?

NIH gene therapy news today - Thermo Fisher & others in Bespoke Gene Therapy Consortium (BGTC) to optimize and streamline gene therapy for rare diseases. #raredisease #genetherapy #precisionmedicine #AAV #viralvectors #cdmo #genomics #biotechnology #NIH nih.gov/news-events/ne…

Today, the UN adopted the first-ever UN Resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” ☺️ Amazing to see unanimous global support for #RareDisease 🌏 Read more here: bit.ly/3q4hopf #NGLY1

Natural history studies are important because they may improve understanding of the disease & future gene therapy trial design. Want to learn more about a natural history study in CLN5 at University of Rochester Medicine? Call 1-866-620-8143 or email [email protected].

Neurogene is excited to share data on A Self-Regulating Gene Therapy for Rett Syndrome, an Oral Presentation at #ASGCT22! We invite you to learn about this novel #genetherapy technology at 4pm ET Wednesday, May 18. Room 202: Novel Therapeutic Targets to treat CNS Disorders.

Gene therapy helps restore hearing loss by delivering the protein EPS8 to rescue malfunctioning inner ear hair cells that transduce sound. lnkd.in/g-BHTe-2 #salkinstitute #genetherapy #AAV #precisionmedicine #viralvector #hearingloss #hearingaid

Would love to catch up IN PERSON at Global Genes next week. Let's compare rare disease notes or talk about gene therapy. I'm around Sun to Thur. #globalgenes #raredisease #ADCY5 #precisionmedicine #genetherapy #AAV #Lentivirus #celltherapy #drugdiscovery globalgenes.org/event/rare-pat…

I think having an animal in our life makes us better humans

Just heard Gail Devers speak at the Global Genes rare disease conference… “A rare disease is something NOT ENOUGH PEOPLE HAVE HEARD ABOUT. Pass the word.” lnkd.in/dDAkSvQW #GGSummit22 #raredisease #precisionmedicine #gravesdisease

Honored to be 1 of 4 families featured in rare disease documentary “Undiagnosed”. It will be premiering as a #BostonFilmFestival Virtual Screening Sep 22-25 & then shared at festivals worldwide over the next year. watch.eventive.org/bostonfilmfest… #raredisease #ADCY5 #precisionmedicine

FDA & NIH announce the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership to advancing the understanding of rare neurodegenerative diseases #raredisease #neurology fda.gov/news-events/pr…

Last minute: Grant for medical education focused on gene and gene-modified cell therapies for rare diseases. ASGCT & Pfizer Medical Education Grant: Deadline Next Week Jan 30 #genetherapy #celltherapy #viralvector #raredisease #precisionmedicine #CME cdn.pfizer.com/pfizercom/2022…

ChatGPT can help with diagnosing Alzheimer’s….really? Wow #alzheimersresearch #neurology #precisionmedicine #neuropsychiatry interestingengineering.com/innovation/cha…

AI for #drugdevelopment podcast - layman's summary re: improved success rates & accelerate cures. Great for #rarediseease. Read related article-MIT Tech Review bit.ly/3FtrdWA sciencefriday.com/segments/ai-de… #precisionmedicine #nof1 #genetherapy #celltherapy #smallmolecule

FDA Peter Marks FDA says 1. Streamline approval of platform technologies. 2. Encourage more gene therapies to seek accelerated approval. 3. Improve harmonization with regulators. 4. Support small companies in rare disease. pharmaphorum.com/views-analysis… #raredisease #precisionmedicine

Resignation letter of Peter Marks from FDA’s director for the center for biological evaluation and research

Sad day for biotech, healthcare, & vaccines. Peter Marks is one of the most even-keeled, forward-thinking physician/scientists who has improved the lives of those suffering from rare disease and common disease. #RareDisease #vaccines #mRNA #biotech npr.org/sections/shots…