Main lessons from the 2018 CDKL5 Forum25 October 2018
For the past four years the Loulou Foundation hosts an annual “by invitation only” meeting where scientists and drug developers working on CDKL5 deficiency, together with representatives from patient organizations, meet to discuss the latest advances. This was the second Forum I attended, and my first since joining the Loulou Foundation.
There were major news announced during the meeting, like a new Phase 2 clinical trial in CDKL5 deficiency disorder with fenfluramine, a drug that has completed two Phase 3 clinical trials in Dravet syndrome with very strong efficacy, and the announcement that Ultragenyx will develop a gene therapy for CDKL5 deficiency disorder...
Ovid Therapeutics Receives “Company Making a Difference Award” from CDKL5 Deficiency Disorder Community24 October 2018
Ovid Therapeutics Inc., a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced it has received the 2018 CDKL5 ForumCompany Making a Difference Award for initiation of the Phase 2 ARCADE trial with OV935/TAK-935, and its commitment to the CDKL5 deficiency disorder (CDD) patient community. The award was announced today in London, UK at the CDKL5 Forum, the largest annual conference on the advancement of science and therapeutic development for CDD...Read More >>
Ultragenyx and RegenXBio Announcement for Gene Therapy to tackle CDKL5 Deficiency Disorder22 October 2018
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced it has exercised its option with REGENXBIO to develop a gene therapy to treat patients with CDD (CDKL5 Deficiency Disorder) utilizing REGENXBIO’s adeno-associated virus (AAV) vectors, including AAV9.Read More on Ultragenyx >> Read More on Regenxbio >>
Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies8 October 2018
Amicus Therapeutics today announced a major collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency and one additional undisclosed rare metabolic disorder. This relationship will combine Amicus’ protein engineering and glycobiology expertise with Penn’s adeno associated virus (AAV)...Read More >>
Ovid Therapeutics Announces Initiation of Two Phase 2 Clinical Trials of OV935/TAK-935 for Pediatric Patients with Rare Epilepsies24 September 2018
Ovid Therapeutics Inc., a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced initiations of the Phase 2 ELEKTRA and ARCADE trials for OV935/TAK-935 in pediatric patients with rare epilepsies. The company reports that the first patient has been randomized to receive either OV935 or placebo in the ELEKTRA trial and that patient screening is underway for the ARCADE trial...Read More >>
Marinus Pharmaceuticals Initiates Pivotal Phase 3 Trial in Children with CDKL5 Deficiency Disorder19 June 2018
Marinus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics to treat epilepsy and neuropsychiatric disorders, today announced the initiation of a pivotal Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD)...Read More >>