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...
endpts.com/new-fda-comm...
April 3, 2025 at 7:14 AM
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...
endpts.com/new-fda-comm...
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-...
www.grc.org/cilia-mucus-...
November 25, 2024 at 8:34 AM
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-...
www.grc.org/cilia-mucus-...
📢 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
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
December 11, 2024 at 10:40 AM
📢 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
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
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)
November 26, 2024 at 1:36 PM
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)
Also here! 🙋♀️🤩 Looking forward to meeting many friends and colleagues again and hearing about recent progress in the fields of #rarediseases and #genetherapies 😊
March 17, 2025 at 1:53 PM
Also here! 🙋♀️🤩 Looking forward to meeting many friends and colleagues again and hearing about recent progress in the fields of #rarediseases and #genetherapies 😊
Going to #ASH24 this looks like a great session for #celltherapies and #biomanufacturing looking forward to seeing the new #genetherapies
Thank you to Dr. Marcela Maus for previewing the Therapeutic #GeneEditing of #StemCells in #ClassicalHematology and #MalignantHematology session at #ASH24!
#ASHinaFlash brings you micro doses of exciting #hematology breakthroughs!
#HemeOnc #HemeSky #HemeSky
www.youtube.com/watch?v=A6Mx...
#ASHinaFlash brings you micro doses of exciting #hematology breakthroughs!
#HemeOnc #HemeSky #HemeSky
www.youtube.com/watch?v=A6Mx...
ASH in a Flash - Therapeutic Gene Editing of Stem Cells in Classical & Malignant Hematology
YouTube video by American Society of Hematology
www.youtube.com
December 4, 2024 at 1:00 PM
Going to #ASH24 this looks like a great session for #celltherapies and #biomanufacturing looking forward to seeing the new #genetherapies
A new study helps researchers engineer better gene therapies. bit.ly/4jhW2Pw #GeneTherapies #BCMResearch
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.
bit.ly
April 1, 2025 at 8:50 PM
A new study helps researchers engineer better gene therapies. bit.ly/4jhW2Pw #GeneTherapies #BCMResearch
PhD defended! 🎓@orbu100.bsky.sociale tackled a major bottleneck in gene delivery—showing Septin 9 and ESCRT proteins play key roles in DNA escape from endosomes. Insights that could improve #GeneTherapies. Congrats, Emmanuel!
PI: Julie Hollien
#PhDLife26 #MCEB @uofu-science.bsky.social @Utah.edu
PI: Julie Hollien
#PhDLife26 #MCEB @uofu-science.bsky.social @Utah.edu
May 1, 2026 at 7:00 PM
PhD defended! 🎓@orbu100.bsky.sociale tackled a major bottleneck in gene delivery—showing Septin 9 and ESCRT proteins play key roles in DNA escape from endosomes. Insights that could improve #GeneTherapies. Congrats, Emmanuel!
PI: Julie Hollien
#PhDLife26 #MCEB @uofu-science.bsky.social @Utah.edu
PI: Julie Hollien
#PhDLife26 #MCEB @uofu-science.bsky.social @Utah.edu
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...
Full article here: doi.org/10.1007/s115...
March 27, 2026 at 3:00 AM
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...
Full article here: doi.org/10.1007/s115...
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
February 4, 2025 at 1:59 PM
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
#Novartis #SMA #spinalmuscularatrophy #genetherapy #Itvisma #SMAgenetherapy #onasemnogeneabeparvovec #patientpopulation #EC #SMApatients #Zolgensma #geneticneuromusculardisease #STEERstudy #STRENGTHtrial #SMAcommunity #patientadvocacy #SMAEurope #reimbursement #genetherapies
zurl.co/NupjG
zurl.co/NupjG
Novartis' new SMA gene therapy cleared in EU
Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.
pharmaphorum.com
July 6, 2026 at 10:03 AM
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)
November 26, 2024 at 2:07 PM
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)
🚀 #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...
👉 doi.org/10.3390/biom...
September 25, 2025 at 5:27 AM
🚀 #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...
👉 doi.org/10.3390/biom...
A groundbreaking collaboration between the Jackson Laboratory, Broad Institute, and Boston Children’s Hospital is set to revolutionize rare-disease therapies by transforming laboratory discoveries into clinical realities.
Click to read more!
#CT #CitizenPortal #ClinicalTrials #GeneTherapies
Click to read more!
#CT #CitizenPortal #ClinicalTrials #GeneTherapies
Jackson Laboratory outlines new Center for Therapeutic Genetics to speed rare-disease therapies
Jackson Laboratory's Matthew Simon described a new Center for Therapeutic Genetics — a collaboration with the Broad Institute and Boston Children's Hospital — to develop models, editing approaches and translational pipelines aimed at moving gene-based therapies into clinical trials.
citizenportal.ai
September 25, 2026 at 2:42 PM
A groundbreaking collaboration between the Jackson Laboratory, Broad Institute, and Boston Children’s Hospital is set to revolutionize rare-disease therapies by transforming laboratory discoveries into clinical realities.
Click to read more!
#CT #CitizenPortal #ClinicalTrials #GeneTherapies
Click to read more!
#CT #CitizenPortal #ClinicalTrials #GeneTherapies
The @alleninstitute.org is launching a new initiative with $400 million in funding and the ultimate goal of developing #GeneTherapies for neurodegenerative diseases including Alzheimer’s, Parkinson’s, Huntington’s, ALS, and Lewy body dementia.
🤔 Learn more: https://bit.ly/4e6VIBU
🤔 Learn more: https://bit.ly/4e6VIBU
June 5, 2026 at 7:22 PM
The @alleninstitute.org is launching a new initiative with $400 million in funding and the ultimate goal of developing #GeneTherapies for neurodegenerative diseases including Alzheimer’s, Parkinson’s, Huntington’s, ALS, and Lewy body dementia.
🤔 Learn more: https://bit.ly/4e6VIBU
🤔 Learn more: https://bit.ly/4e6VIBU
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.
📅 Date: Monday, 9th June 2025
⏰ Time: 1pm-2pm UK time
👉 Register here: edin.ac/43O5PIC
Kindly hosted by @ukri.org showcasing our #AdvancedTherapeutics hub.
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.
edin.ac
May 30, 2025 at 2:50 PM
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.
📅 Date: Monday, 9th June 2025
⏰ Time: 1pm-2pm UK time
👉 Register here: edin.ac/43O5PIC
Kindly hosted by @ukri.org showcasing our #AdvancedTherapeutics hub.
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
#research #innovation
March 21, 2025 at 3:33 PM
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
#research #innovation
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!
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!
April 25, 2025 at 4:29 PM
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!
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!
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
Novartis paid billions for AveXis—delays highlight the complexity of gene therapies.
Hopefully, this positive news drives more investment in the space #genetherapies
January 6, 2025 at 1:40 PM
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
Novartis paid billions for AveXis—delays highlight the complexity of gene therapies.
Hopefully, this positive news drives more investment in the space #genetherapies
🧬 #GeneTherapies are revolutionary for #RareDiseases. But regulatory systems were developed for small molecules, not advanced therapies.
💡 ARDAT developed evidence to support the regulatory assessment of ATMPs, helping launch a #ClinicalTrial for a rare disease treatment.
👉 link.europa.eu/cgmm6P
💡 ARDAT developed evidence to support the regulatory assessment of ATMPs, helping launch a #ClinicalTrial for a rare disease treatment.
👉 link.europa.eu/cgmm6P
June 25, 2026 at 9:13 AM
🧬 #GeneTherapies are revolutionary for #RareDiseases. But regulatory systems were developed for small molecules, not advanced therapies.
💡 ARDAT developed evidence to support the regulatory assessment of ATMPs, helping launch a #ClinicalTrial for a rare disease treatment.
👉 link.europa.eu/cgmm6P
💡 ARDAT developed evidence to support the regulatory assessment of ATMPs, helping launch a #ClinicalTrial for a rare disease treatment.
👉 link.europa.eu/cgmm6P
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...
December 9, 2024 at 5:53 PM
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...
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 we can get more advances like that… this opens up to many, many different cancers,” -Dr. Michael Hurwitz.
ctinsider.com/business/art...
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.
ctinsider.com
March 26, 2026 at 2:54 PM
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 we can get more advances like that… this opens up to many, many different cancers,” -Dr. Michael Hurwitz.
ctinsider.com/business/art...
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
Join our upcoming @ukri.org - hosted webinar to learn more
📅 Date: Monday 9th June 2025
⏰ Time: 1-2pm UK time
🔗 edin.ac/43O5PIC
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.
edin.ac
April 10, 2025 at 9:57 AM
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
Join our upcoming @ukri.org - hosted webinar to learn more
📅 Date: Monday 9th June 2025
⏰ Time: 1-2pm UK time
🔗 edin.ac/43O5PIC
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...
theincidentaleconomist.com/wordpress/ge...
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.
theincidentaleconomist.com
March 24, 2026 at 1:38 PM
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...
theincidentaleconomist.com/wordpress/ge...
#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! 🔬
She highlighted advances in #CellTherapy, #TissueEngineering, and #GeneTherapies offering hope for Type 1 #diabetes, #MuscularDystrophy, and more! 🔬
CSPC 2024 Interview with Cate Murray
YouTube video by Canadian Science Policy Centre
www.youtube.com
January 17, 2025 at 9:10 PM
#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! 🔬
She highlighted advances in #CellTherapy, #TissueEngineering, and #GeneTherapies offering hope for Type 1 #diabetes, #MuscularDystrophy, and more! 🔬