🧬 Genetic Therapies for Rare Diseases: Developing Ethical Regulatory Policies

🔗https://bit.ly/4sIQl1A

#Bioethics #GeneTherapy #RareDiseases #GeneticMedicine #HealthPolicy

Jennifer Doudna’s $1 Billion Plan To Bring Gene Editing To The Masses Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer Jennifer Doudna wants to build an entire ecosystem to bring these treatments mains...

Read the @forbes.com daily cover story on Dr. Doudna: www.forbes.com/sites/amyfel...

And a 2023 feature on Scribe: www.forbes.com/sites/alexkn...

#CRISPR #GeneEditing #GeneticMedicine

Subcommittee backs bill to fund genetic counseling education amid statewide shortage The Higher Education Budget Subcommittee reported CS for House Bill 1115 favorably after Representative Anderson said Florida has just 179 licensed genetic counselors and needs an education grant program to expand the workforce; Laboratory Corporation of America filed a written expression of support.

Florida is on the brink of a genetic counseling crisis, with only 179 licensed professionals in the state, but a new bill aims to change that by funding education for future counselors!

Click to read more!

#FL #WorkforceDevelopment #HealthcareEducation #GeneticMedicine #CitizenPortal

🧬 CRISPR breakthrough targets rare neurological disease! Gene editing strategy could rewrite the future of NIID treatment. Precision medicine FTW! 🧠 #GeneticMedicine #MedicalInnovation #CRISPR https://emmecola.github.io/genomics-daily

🧬 From antitarget identification to genetics-driven discovery, RNA-based strategies are redefining antiviral drug development. These insights align with key discussions at OTDC.
📍Rome, Italy |🗓️ May 25–26, 2026
🔗 oligo-nucleotide.com
#RNAtherapeutics #PrecisionMedicine #GeneticMedicine #OTDC2026

🎉 Excited to announce: Sculpta Bio has won the 2025 Alnylam BioVenture Challenge at Alnylam HQ in Cambridge!

Read full recap https://loom.ly/1CJlCMI
More on Alnylam https://loom.ly/P6fMsRU

#GeneticMedicine #BiotechInnovation #RNA #Nucleate #BioVentureChallenge

Scribe Therapeutics CEO on how CRISPR technologies are ready to move beyond rare diseases and take on the world’s biggest killers.

longevity.technology/news/can-gen...

#longevity #CRISPR #geneticmedicine #CVD #heartdisease

New data at ASN Kidney Week 2025 demonstrating the potential of ultrasound-mediated delivery. Learn about our latest results in the treatment of X-Linked Alport Syndrome, delivering full-length gene expression: sonothera.com/press-releas...

#KidneyWk #SonoThera #XLAS #ultrasound #geneticmedicine

The 2026 #MDAconference agenda is LIVE! 🧬Join global leaders in #neuromuscular research, care & advocacy March 8-11 in Orlando FL. View tracks, speakers & sessions: www.mda.org/press-releas...

#NeuromuscularResearch #GeneticMedicine #RareDisease #ClinicalCare

Huntington's disease successfully treated for first time One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.

#UniQure #genetherapy #genesilencingtechnologies #Huntingtonsdisease #Huntingtonspatients #UCL #DNA #huntingtingene #huntingtinprotein #cuttingedgemedicine #geneticmedicine #neurosurgery #mutanthuntingtin #NationalHospitalforNeurologyandNeurosurgery #UCLH
www.bbc.co.uk/news/article...

Huntington’s disease sees first-ever slowdown with gene therapy Researchers call the results “world-changing"

Huntington’s disease sees first-ever slowdown with gene therapy

#HopeForHuntingtons #EndHuntingtons #GeneticMedicine #MedicalBreakthrough #Neurogenetics #FutureOfMedicine #ScienceSavingLives

www.indiaweekly.biz/huntingtons-...

US Gitelman Syndrome Market Size, Growth Insights 2035 The US Gitelman Syndrome Market Industry is expected to grow from 186.9(USD Million) in 2024 to 883 (USD Million) by 2035.

New therapies are emerging for this rare genetic kidney disorder—personalized care changing lives. 🌟🩺
#RareDisease #Nephrology #GeneticMedicine

www.marketresearchfuture.com/reports/us-g...

Thanks for the follow @bsgmofficial.bsky.social, great to have you on here

#Genetics #GeneticMedicine #GenomicMedicine

Fighting genetic cancer with genetic precision! 🎯 Scientists identify natural compounds that counteract breast cancer mutations using molecular genetics. Ancient plant medicine validated through cutting-edge analysis.

dl.begellhouse.com/journals/439...

#GeneticMedicine #CancerGenetics

Explore the future of personalized medicine for inborn errors of metabolism. Join us on July 30 for expert insights!

Join us on July 30 for a webinar on cutting-edge gene editing therapies for metabolic disorders. Dr. Rebecca Ahrens-Nicklas will share exciting advances from research to clinical trials. Register here: spkl.io/63325AEm6W

#InbornErrors #GeneticMedicine

The British Society for Genetic Medicine will be on the 5th of November. Get your abstracts ready! #BSGM #AnnualConference #GeneticMedicine

AlphaRose Therapeutics on doing things differently Casey McPherson founded AlphaRose Therapeutics with the aim of one day developing a cure for his daughter and others like her.

Casey McPherson's journey from music to rare disease treatments inspires us! Join AlphaRose in making a difference! #RareDiseases #Biotechnology #Innovation #Healthcare #AlphaRoseTherapeutics #GeneticMedicine rarerevolutionmagazine.com/rare-insider...

Prime Editing: The Next Frontier in Genetic Medicine By Dr. Chinta SidharthanReviewed by Benedette Cuffari, M.Sc. Discover how prime editing is redefining the future of medicine by offering highly precise, safe, and versatile DNA corrections, bringing h...

Prime Editing: The Next Frontier in Genetic Medicine

thanks to @nanoappsmedical.bsky.social @amandascott2023.bsky.social @cellrep7.bsky.social
#NanoTech #GeneticMedicine #NextFrontier #SDGs
www.nanoappsmedical.com/prime-editin...

#PortfolioNews | Proud to join the $135M Series B of our #portfolio company #SpliceBio, the largest round ever raised by a Spanish biotech 🚀

🧬 Advance SB-007 for #StargardtDisease
🔬Expand #GeneticMedicine pipeline

👉 buff.ly/jey8g9O

#VC #Biotech #Innovation #ProteinSplicing

Prime Editing: The Next Frontier in Genetic Medicine Prime editing is an advanced gene editing technology that enables precise DNA corrections without introducing double-stranded breaks, significantly reducing off-target effects. This innovation holds p...

Prime Editing: The Next Frontier in Genetic Medicine www.news-medical.net/health/Prime... #PrimeEditing #GeneEditing #Genome #GeneticMedicine #CRISPR #PersonalizedMedicine #Biotechnology #Genetics #DNARepair

A baby has become the first person to be successfully treated for a rare genetic disease before birth.

#GeneticMedicine #SpinalMuscularAtrophy #PrenatalCare #MedicalBreakthrough #SMAAwareness #RareDiseases #GeneTherapy #FDAApproval

New Guidelines Aim to Improve Cystic Fibrosis Screening | Newswise All states should adopt updated screening protocols so more newborns with cystic fibrosis can be diagnosed in the first weeks of life, when interventions can have the greatest benefit, according to th...

New Guidelines Aim to Improve Cystic Fibrosis Screening
www.newswise.com/articles/new...
#cysticfibrosis #newbornscreening #geneticmedicine

Excited for #GTC2025! 💚 Gus Minto-Cowcher joins Microsoft onstage to share how Basecamp Research uses Azure VMs & AI Services—accelerated by NVIDIA GPUs—to decode complex biological data.

Catch it live: March 19, 12pm PDT @ Microsoft Theater. Don’t miss it!

#MLOps #GeneticMedicine #DrugDiscovery

The Future of Cell & Gene Therapies—Powered by AI

Basecamp Research CEO @glengowers.bsky.social is speaking at BIO-Europe Spring on how AI + the world’s largest biological dataset are shaping gene writing & programmable insertion.

For info go to bit.ly/3FpB9Df

#BIOEuropeSpring #GeneticMedicine

Presenting new data @MDAorg Clinical & Scientific Conference highlighting potential of genetic medicine using targeted, ultrasound-mediated delivery in treating DMD. Learn more: sonothera.com/press-releases #geneticmedicine #ultrasound #ultrasoundmediated #SonoThera #MDAconference #MuscularDystrophy

On #IWD2025 💪🙋‍♀️, we celebrate Dr. Alison Reynolds @ucddublin.bsky.social for her groundbreaking work in #YouthBlindness & #HereditaryBlindness. Her research in #GeneticMedicine & #ZebrafishModels is working to unlock new treatments for inherited vision conditions.🔬🧬🐠
@ucdresearch.bsky.social

ep.10/46 🇺🇸 🇮🇳 Startups Accepted by Y Combinator 2024 Three founders, Prof. Michael Dustin, Dr. Ashwin Jainarayanan, and Dr. Ashwin Nandakumar, successfully raised funds led by Felicis and Refactor. The round also included participation from Y Combinator and various notable ventures and angel investors, highlighting significant investor interest in their initiative.

Innovative cancer & genetic medicine delivery 🧬

🇺🇸 🇮🇳 Biotech startup raises $7.14M in seed funding

#BiotechStartup #CancerTreatments #GeneticMedicine #BiotechInvestments #StartupFounders #SeedFunding #TherapeuticDelivery

This week, be sure to catch TuneTx Co-Founders Charles Gersbach and Fyodor Urnov - along with Tune VP & Head of Research Blythe Sather - at PMWC 2025. Their 'Beyond the Genome' panel event on the future potential of epigenetic medicine. #PMWC2025 #geneticmedicine #epigenome

Children’s National Research Institute Academic Annual Report 2023-2024 Academic Annual Report for 2023-2024

From breakthroughs in cell and gene therapy to advancements in #BrainHealth, #GeneticMedicine and #SurgicalInnovation, we’re proud to showcase how Children’s National is leading the way in improving outcomes for children everywhere.

Gene and Cell Therapies Take Aim at Parkinson’s Disease New therapies that are less intrusive but more lasting than current interventions promise to arrest and even reverse neurodegeneration.

How can patients with #Parkinsons disease benefit from gene and cell therapies? New, less intrusive treatments are in development that could slow down - or even reverse - neurodegeneration. #geneticmedicine

My latest for GENBio:
www.genengnews.com/topics/drug-...