French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy – Labiotech.eu (blog)
Posted: June 22, 2017 at 4:41 pm
NobleGnthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease.
Dedicated to rare diseases since 1990 and more recently to gene therapy, Gnthon is one of veryfew not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is well established as a nonprofit, credit for which is due toits creator, the French Muscular Dystrophy Association, AFM Tlthon. The company has nowteamed up with the (in)famous American company, Sarepta, to work on a gene therapy for Duchenne Muscular Dystrophy (DMD).
DMD first affects the shoulder and upper arm muscles and the muscles of the hips and thighs. (Source: mda.org)
When I last spoke to Gnthons CEO,Frdric Revah, he told me that while the majority of the companys financial support comes from Tlthon, an increasing amount comes fromsuch partnerships.We get more and more support from industrial partnerships as we outlicense more of our drugs, explains Revah. However, Tlthon will always remain the main source of our funding; the funding from out-licensing is a complement.
But is Sarepta the best partner? While the American biotech can boast about its FDA-approved drug for DMD, Exondys 51, (which is just sold to Gilead for $125M,) this achievement isdubious:not only was the key clinical trial tiny, the FDAadmittedthat patients did not receive aclear benefit fromthe drug in the study. These circumstances prompted arenowned journalist to suefor thedocuments pertaining to the decision, an attack thatSareptas stock into a downward spiral in May.
Nevertheless, under the terms of the partnership agreement,Gnthon will trust Sarepta as a potential co-developer ofits DMD candidates, all of which are preclinical. The French biotechhas been developing amicro-dystrophin gene therapythat has proven itself applicable to the disease. As it countswith Europes largest cGMP vector manufactory, YposKesi, andone of the worlds largest research and clinical groups developing rare disease therapies, Gnthon is an attractive partner for any companies prospecting in the field.
Sareptas pricing practices may also offendthe sensibilities of a nonprofit, since theyraised the hackles of the drug pricing patrol with a plan to charge $300k per year for Exondys 51. According to STAT News, the companysCEO, Edward Kaye, saidthis figure isin the middle of the range for rare disease drugs,and given the sensitivity to pricing, we tried to be reasonable when looking at all the costs.
Though financial details of the agreement have not been disclosed, Gnthon may have enough influence to sway Sareptaaway from gouging.Our main goal is to ensure that patients have access to drugs and that they are affordable. Price should not be an obstacle,Revah told me.
Whatever we do here, we hope to apply the same tech to diseases that affect more people, like sickle cell anemia and cancer,he continued. With a crowded arena of companies like CRISPR Therapeutics, AMO Pharma, andDebiopharmall battling to bring the next DMD drug to market, having a back up plan via a platform seemssensible.
Images via Alila Medical Media, Anatomy Insider / shutterstock.com
Original post:
French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy - Labiotech.eu (blog)
- Gene Therapy Could Prevent Blindness [Last Updated On: June 10th, 2010] [Originally Added On: June 10th, 2010]
- Gene Brodland Sits with Cambridge Who's Who in a Revealing Interview [Last Updated On: June 16th, 2010] [Originally Added On: June 16th, 2010]
- Researchers Make Colon Cancer Breakthrough [Last Updated On: July 20th, 2010] [Originally Added On: July 20th, 2010]
- Pro abortion- Antiabortion myth8 - Fly to India for safe abortion! [Last Updated On: October 16th, 2010] [Originally Added On: October 16th, 2010]
- New Fertility Test / Whooping Cough Alert / Gene Therapy for Depression [Last Updated On: October 21st, 2010] [Originally Added On: October 21st, 2010]
- Alzheimer's Breakthrough? [Last Updated On: October 23rd, 2010] [Originally Added On: October 23rd, 2010]
- Audio Genetics Lab - Native Flute - MP3Tera Forums [Last Updated On: December 8th, 2010] [Originally Added On: December 8th, 2010]
- Gene Therapy - Cortical Studios [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- There Shall Be Physicians for the Spirit: USC Institute for Genetic Medicine Art Gallery [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- IRRI: Rice genetic diversity and discovery [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Research Symposium: Mork Depart - 2006 - Video 1 [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Mendelian Genetics [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Ayurveda [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Drs. Kaspar and MacKenzie discuss the promise and path forward for SMA Gene Therapy [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- 3. Genetic Engineering [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Prof. Martinez Cruzado Lecture Part 3 "Amerindian Gene Study In Puerto Rico" [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Gene Therapy Research Makes Nationwide Children's Worthy of Wellstone Center [Last Updated On: May 8th, 2011] [Originally Added On: May 8th, 2011]
- Gene Therapy Example [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Sweet Tooth Gene [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- OHSU's video of new gene therapy method developed at the Oregon National Primate Research Center [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Gregor Mendel's Punnett Squares [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Ethical Concerns With Genetic Engineering [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Dr. Laura Niklason on the importance of her AFAR grants for telomerase gene therapy research [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- What is the future of genetic medicine? [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Prof. Martinez Cruzado Lecture Part 2 "Amerindian Gene Study In Puerto Rico" [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Jewish DNA - Genetic Research and The Origins of the Jewish People [Last Updated On: May 9th, 2011] [Originally Added On: May 9th, 2011]
- Future of genetic engineering - by Futurist Dr Patrick Dixon. Genetic mutations and genetic disorders. Gene science by conference keynote speaker [Last Updated On: May 11th, 2011] [Originally Added On: May 11th, 2011]
- annstewart82's Genetic Medicine and God [Last Updated On: May 13th, 2011] [Originally Added On: May 13th, 2011]
- Genetic research could unlock breeding seasons in sheep [Last Updated On: May 13th, 2011] [Originally Added On: May 13th, 2011]
- Genetics 101 Part 1: What are genes? [Last Updated On: May 13th, 2011] [Originally Added On: May 13th, 2011]
- Molecular and Cellular Foundations of Medicine, 1 of 2 [Last Updated On: May 13th, 2011] [Originally Added On: May 13th, 2011]
- Dan Arking of Johns Hopkins Medicine [Last Updated On: May 17th, 2011] [Originally Added On: May 17th, 2011]
- Genetics : How Is Gene Therapy Done? [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Gene Therapy Shows Promise for Blindness [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Tomato suicide gene therapy [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Cancer Alternative Treatment - Gene Therapy for Cancer a Report from Channel 4 News [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Muscular Dystrophy Gene Therapy: ScienCentral News Video [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Sickle Cell Anemia -- Hope from Gene Therapy [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Challenges of gene therapy [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Pain Gene Therapy [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Gene therapy success 'reverses' blindness [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- DNA Gene Therapy [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- The Neural Circuitry of Perception [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Resetting Metabolism- Nuclear Receptors and AMPK: A Lecture by Ronald Evans, PhD [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Islands at Risk (Part 2) - Genetic Engineering in Hawai'i [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Gene Therapy journal videocast from ASGCT 2011 Xiao Xiao on gene therapy for muscular dystrophy [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- HYBRID HUMANS-Hair Follicle Gene Therapy [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Genetics 101 Part 4: What is phenotype? [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Lloyd Pye - Ancient Genetic Engineering [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Study Designs: Genetic Association Studies [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Gene Therapy for Genetic Disease: The Long and Winding Road [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Introduction to Population Genetics [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Islands at Risk (Part 1) - Genetic Engineering in Hawai'i [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Genetics Based Research on Treatment-Resistant Epilepsy [Last Updated On: May 20th, 2011] [Originally Added On: May 20th, 2011]
- Science in Action: Gene Therapy for Color Blindness [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- Gregg Semenza of Johns Hopkins Medicine on HIF 1 [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- The Sleepiness Gene [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- Blind Gene Therapy [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- Richard Dawkins and Dr Yan on genetic ancestry (extended version) - Bang Goes the Theory - BBC One [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- Joshua Mendell of Johns Hopkins Medicine [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- Genetic/Genomic Faculty Champion Initiative (PM session) [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- euronews science - Epigenetics [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- The Genetic Age, Panel 1 [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- Biobanking and Bioethics: When Genetics Research Hits the Courts [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- Gene Therapy journal videocast from ASGCT 2011 Darren Wolfe on gene therapy for pain.m4v [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Genetics 101 Part 3: Where do your genes come from? [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- ASHG 2010 Mtg.: "Complex Disease Genetics Research in Populations" (Dr. Carlos Bustamente) [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Public Talk - Prof Leonard Seymour, Oxford [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- A New Era in Medicine: Genetics [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Genetic Engineering Animation [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Linda Brzustowicz - Genetic Causes of Schizophrenia [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Hadassah Gene Therapy Center [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Gene Therapy journal videocast from ASGCT 2011 Robin Ali on gene therapy for retinal disease [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- UF cardiologists study gene-modified stem cells to help Dobermans with common heart condition [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Akhilesh Pandey of Johns Hopkins Medicine [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- China's Cancer Drug - China [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Genetic Therapy Restored Boy's Sight [Last Updated On: May 24th, 2011] [Originally Added On: May 24th, 2011]
- Molecular and Cellular Foundations of Medicine Class, 2 of 2 [Last Updated On: May 24th, 2011] [Originally Added On: May 24th, 2011]
- Talking Research - Professor George Ebers - Vitamin D and genetics in MS [Last Updated On: May 24th, 2011] [Originally Added On: May 24th, 2011]
- Designing Humanity - Genetic Engineering [Last Updated On: May 30th, 2011] [Originally Added On: May 30th, 2011]