22-23 PHARMA - Fast and the curious This month: the NHS as a dystopian game of Tetris, the GLP-1 explosion and its impact on future meds, plus deep dives into AI in pharma, reproductive health, and Trump’s healthcare influence. Also, co...

#mRNAvaccines #GLP1 #obesity #CARTcelltherapies #oncology #PCSK9inhibitors #HepatitisC #Immunotherapy #checkpointinhibitors #RSVvaccines #Genetherapies #rarediseases #antivirals #Biosimilars #expandedaccess #biologics #HIV #Migraine #CGRPinhibitors #antifungals #precisionmedicine
zurl.co/3UDz7

Advancing the Battle against Cystic Fibrosis: Stem Cell and Gene Therapy Insights

This review examines #CysticFibrosis (CF) pathophysiology and the emerging role of #StemCell and #GeneTherapies, highlighting how these strategies could redefine CF management and improve #PatientOutcomes. #medsky

Full article here: doi.org/10.1007/s115...

CT researchers explore gene therapy that shows promise to slow aging, fight cancer With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients.

CAR T-cell and #genetherapies are reshaping #cancer care—and what’s next could be even bigger.
“If we can get more advances like that… this opens up to many, many different cancers,” -Dr. Michael Hurwitz.
ctinsider.com/business/art...

If Gene Therapies are so Revolutionary, Why Does no one Want to Pay for Them? | The Incidental Economist Gene therapies can cure once-incurable diseases, but without payment reform, America’s insurance system will keep them out of reach for many patients.

The Incidental Economist explores how to pay for high-cost #genetherapies, including “rev pay” models that spread costs over time to address large upfront prices, payer risk, & patient mobility. The piece cites CP3 research on financing for gene therapies.

theincidentaleconomist.com/wordpress/ge...

Bedford-based company secures £6m funding to reduce cost of breakthrough gene therapies - Bedford Independent A Sharnbrook-based company has completed a £6 million funding round for its 'Smart Polymer' technology that could significantly...

The company, based at Colworth Science Park in Sharnbrook, intends to use the funds to open up access to groundbreaking treatment...

#Bedford #BedfordNews #GeneTherapies

AstraZeneca raises guidance on stellar cancer drugs growth AZ reports strong financial results for 2025, and provides a first peek at the data for its oral GLP-1 contender elecoglipron

#AstraZeneca #PascalSoriot #cancertherapies #oncologyproducts #commercialperformance #blockbusterbrands #cancer #weightloss #genetherapies #antiHER2therapy #prostatecancer #breastcancer #asthma #respiratorysyncytialvirus #RSV #RSVprophylactic #lupus #phase2trials #phase3trials
zurl.co/yLEDf

🚀 #IT delivery💉bypasses the #BBB, sending #ASOs 🧬, #GeneTherapies 🧑‍🔬, #Antibodies 🛡️ & #StemCells 🌱 straight into the #Brain! A lifeline for #NDDs? @mdpiopenaccess.bsky.social #NeuroTech #BrainResearch
👉 doi.org/10.3390/biom...

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

Mark your calendar! July 30, 2025, 12-1 PM EST — webinar on platform-based therapies for inborn errors of metabolism. Featuring Dr. Rebecca Ahrens-Nicklas spkl.io/63320AEmKi

#RareDiseases #GeneTherapies

Secure your spot! Join our webinar on July 30 with Dr. Rebecca Ahrens-Nicklas to learn about advances in genomic therapies for severe metabolic diseases.

Secure your spot! Join our webinar on July 30 with Dr. Rebecca Ahrens-Nicklas to learn about advances in genomic therapies for severe metabolic diseases. spkl.io/63328AElxj #RareDiseases #GeneTherapies

Engineering Biology Mission Hubs showcase: advanced therapeutics Join members from the team leading the Engineered Genetic Control Systems for Advanced Therapeutics Mission Hub as they provide an overview of their expansive engineering biology programme.

Discover how we harness #engineeringbiology for safe and effective #genetherapies. Sign up to our upcoming webinar.

📅 Date: Monday, 9th June 2025
⏰ Time: 1pm-2pm UK time

👉 Register here: edin.ac/43O5PIC

Kindly hosted by @ukri.org showcasing our #AdvancedTherapeutics hub.

Attending #SynBioBeta2025!
Our Hub focusses on #engineeringbiology for safe and effective #genetherapies. We are looking forward to insights from the Engineered Human Therapies track, connecting with industry leaders, and exploring new opportunities.
Reach out if you’d like to connect there!

Engineering Biology Mission Hubs showcase: advanced therapeutics Join members from the team leading the Engineered Genetic Control Systems for Advanced Therapeutics Mission Hub as they provide an overview of their expansive engineering biology programme.

We're on Bluesky! Follow us for exciting updates, insights, and arising opportunities as we harness #engineeringbiology for safe and effective #genetherapies.

Join our upcoming @ukri.org - hosted webinar to learn more
📅 Date: Monday 9th June 2025
⏰ Time: 1-2pm UK time
🔗 edin.ac/43O5PIC

Unifying observational research with a next-generation virtual approach Explore the unification of observational research with a next-generation virtual approach in decentralised clinical trials (DCTs). Learn more about the benefits and advancements in this innovative met...

#Observationalstudies #interventionaltrials #celltherapies #genetherapies #AI #clinicaltrials #integratedendtoendtrials #DCTmodels #interventionaltrialdesigns #digitaltechnologies #patientparticipation #virtualobservationalstudies #hybridobservationalstudies
pharmaphorum.com/patients/uni...

After firing of #PeterMarks, new FDA commissioner emphasizes importance of #GeneTherapies (consistent with Peter Marks and new CBER acting director):

endpts.com/new-fda-comm...

New study helps researchers engineer better gene therapies This study will make basic research on AAV vectors for gene delivery and preclinical gene therapy studies in mice more efficient and reproducible.

A new study helps researchers engineer better gene therapies. bit.ly/4jhW2Pw #GeneTherapies #BCMResearch

Kick-off of the #GeneH project in Coimbra, Portugal. A new European Project by a consortium involving Academia, Industry, Government Agencies and Patient Organizations focused on #GeneTherapies for #RareDiseases. Stay tuned to learn more
#research #innovation

Also here! 🙋‍♀️🤩 Looking forward to meeting many friends and colleagues again and hearing about recent progress in the fields of #rarediseases and #genetherapies 😊

A professional profile graphic featuring Samantha Malka, NIHR Moorfields Bioresource Centre’s Genomic Research Manager. The design includes a photo of Samantha wearing glasses and an orange top, standing outdoors with a blurred cityscape in the background. The graphic incorporates hexagonal elements in teal, white, and orange, along with the NIHR Moorfields Biomedical Research Centre logo.

This #RetinitisPigmentosa Month, we’re spotlighting Sam Malka’s research on improving diagnosis for Black African communities. Her discovery of a genetic change opens the door to future #GeneTherapies.

Find out more: buff.ly/3CHwgV8

@ucleye.bsky.social #GenomicsEngland

Unlocking the Potential of Receptor-Based Approaches in Diabetes Treatment Announcing a new article publication for BIO Integration journal.   Diabetes, a common metabolic condition, poses a substantial health burden worldwide. To revolutionize diabetes management, enhance g...

From #GLP1 agonists to #GeneTherapies, innovative drug therapies are revolutionizing #diabetes management. Explore the future of #personalizedmedicine now!

Read full article 👉 bio-integration.org/novel-diabet...

#DiabetesResearch #biotech #precisionmedicine

On #WorldCancerDay, we acknowledge the stories behind every cancer diagnosis—resilience, loss, hope, and healing. These stories drive us to develop intelligent #GeneCircuits for next-gen cell and #genetherapies, aiming to transform outcomes for cancer patients. #UnitedByUnique

Genes & Diseases: A Leading Journal in Molecular and Translational Medicine Discover the cutting-edge research published in Genes & Diseases, a leading journal in molecular and translational medicine. This video introduces the journa...

Explore Genes & Diseases, a top journal in molecular and #translationalmedicine . Uncover the molecular bases of #humandiseases and experimental #therapeutics through high-quality, peer-reviewed research.

Watch this video: https://buff.ly/3EfhC7K

#openaccessjournal #GeneTherapies #sciencejournal

UK sees 70% increase in phase I advanced therapy clinical trials in 2024 - PharmaTimes Cell and Gene Therapy Catapult reveals significant growth in UK trials

#advancedtherapyclinicaltrials #CellandGeneTherapyCatapult #CGTCatapult #UKtrials #UK #clinicaltrials #phaseItrials #AdvancedTherapyMedicinalProductClinicalTrialsDatabase #ATMPClinicalTrialsDatabase #globaltrials #Europeantrials #Genetherapies #ATMPclinicaltrials
pharmatimes.com/news/uk-sees...

#ICYMI: In an interview with CSPC, Cate Murray, SCN President & CEO, shared a vision for the future of #RegenMed.

She highlighted advances in #CellTherapy, #TissueEngineering, and #GeneTherapies offering hope for Type 1 #diabetes, #MuscularDystrophy, and more! 🔬

After years of delay, Novartis’ intrathecal Zolgensma hit its phase 3 goal, improving motor function in SMA Type 2 (ages 2–18).

Novartis paid billions for AveXis—delays highlight the complexity of gene therapies.

Hopefully, this positive news drives more investment in the space #genetherapies

📢 Huge News! 📢

The MRC has launched two centres of research excellence to transform gene therapies: IDRM’s Paul Riley will co-direct the MRC/BHF CoRE in Advanced Cardiac Therapies & Stephan Sanders will direct the MRC CoRE in Therapeutic Genomics.
Read more: lnkd.in/g-3CmAsP
#GeneTherapies

From March 12-13, join us for this free, virtual event to inform and educate those who can benefit from #celltherapy and #genetherapy treatments.

From March 12-13 at Empowering Patients 2025, we'll explore:

✔️The Value of Patients + Advocates
✔️Why Are Clinical Trials for #RareDiseases Different?
✔️Developing #GeneTherapies as Platform Technologies

Register for FREE! #ASGCTAdvocacy #PatientEducation www.asgct.org/events/empow...

Going to #ASH24 this looks like a great session for #celltherapies and #biomanufacturing looking forward to seeing the new #genetherapies

This is just a first small step for #AI in #PrecisionMedicine. We’re working on many more AI-guided CAR-T cell approaches and hope that #GenerativeAI can democratize and greatly accelerate binder design for new #celltherapies and #genetherapies, especially in the context of cancer resistance. (7)

In the first preprint from my and @grunewald.bsky.social labs @TU_Muenchen, we show how this could work for new #celltherapies and #genetherapies. (2)

Deadline is today for abstracts for talks for our GRC Cilia, Mucus & Mucociliary Interactions in Pomona, California 16-21/02/25! Organized by myself, Katharina Ribbeck, Marcus Mall & Sudipto Roy! #cilia #biophysics #RareDisease #biofilms #GeneTherapies #Mucus #PCD #CF

www.grc.org/cilia-mucus-...