Industry Partners
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide.
Genentech is a biotechnology company dedicated to pursuing groundbreaking science to discover and develop medicines for people with serious and life-threatening diseases. Our transformational discoveries include the first targeted antibody for cancer and the first medicine for primary progressive multiple sclerosis.
Considered the founder of the industry, Genentech, now a member of the Roche Group, has been delivering on the promise of biotechnology for more than 40 years.
Roche believe’s it’s urgent to deliver medical solutions right now – even as they develop innovations for the future. They are passionate about transforming patients’ lives. They are courageous in both decision and action. And they believe that good business means a better world.
That is why they come to work each day. They commit themselves to scientific rigour, unassailable ethics, and access to medical innovations for all. They do this today to build a better tomorrow.
Letter to the community-First Patient dosed!
GeneTx Biotherapeutics was setup as a subsidiary of the Foundation for Angelman Syndrome Therapeutics for the development and commercialization of an investigational antisense drug, GTX-102, for the treatment of Angelman syndrome (AS).
Press Release on 5/19/21 and FAQ’s
Webinar on the KIK-AS Clinical Trials of GTX-102
Ionis is guided by world-class scientists and business leaders whose passion to innovate is matched only by their commitment to deliver transformational medicines to patients who need them.
Ionis is THE leading innovator in RNA-targeted therapeutics and was purposely designed to:
Create a better, more efficient drug discovery platform
Establish a new innovation-centered business model, and
Maximize the value of every medicine we create and get it to the people who need it most as quickly and as efficiently as possible
Neuren is developing new therapies for neurodevelopmental disorders with high unmet need. To learn more about the trial go to https://angelmanstudy.com.au/
Clinical Trial Phase One Press Release
Ovid Therapeutics is a biopharmaceutical company focused exclusively on developing impactful medicines for patients and families living with rare neurological disorders.
Letter from President and CEO, Jeremy Levin on 12/2/20
Update on Clinical Trial Status due to COVID-19
PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs that regulate gene expression by targeting post-translational control mechanisms in orphan diseases.
Impact of COVID-19- Response from PTC
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are no approved therapies.
Sarepta is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short.
Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, and StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, announced their collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system (CNS) and neuromuscular targets. Learn more.
StrideBio, Inc. is a gene therapy company focused on creating and developing novel adeno-associated viral (AAV) vector technologies. Our STRucture Inspired DEsign approach holds the potential to generate unique AAV capsids capable of overcoming the challenges of pre-existing neutralizing antibodies in patients. This powerful new approach has broad application, enabling gene addition, gene silencing and gene editing modalities for rare diseases.
Sage is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the brain. We are pursuing new pathways with the goal of improving brain health, and our depression, neurology and neuropsychiatry franchise programs aim to change how brain disorders are thought about and treated. Our mission is to make medicines that matter so people can get better, sooner. Learn More
At Taysha Gene Therapies, we believe the patient always comes first. This is why we are singularly focused on discovering, developing and commercializing gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), both in rare and large patient populations. We were founded in partnership with The University of Texas Southwestern Medical Center (UT Southwestern) to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we have created a powerful engine to develop transformative therapies with the potential to dramatically improve patients’ lives. Learn More
Healx went from being an idea to a reality when our co-founders, Dr Tim Guilliams and Dr David Brown, met a man named Nick Sireau. More than a decade earlier, Nick had set up a patient group to help save his sons from an extremely rare genetic disorder. He worked tirelessly, but the drug discovery process was slow and went into the millions of dollars, and this is a problem many patient groups still face today. And the truth is that most patient groups don’t find success. Tim and Dave had long been advocating the benefits of drug repurposing, in combination with the power and speed of AI. Nick became their inspiration – he was the living story of the therapeutic need their abilities could meet. This encounter took place in 2014 and we’ve been growing ever since. Today, we’re a 50+ strong, world-class team of drug discoverers, AI engineers, and bioinformaticians, all working together to accelerate the discovery of new treatments for rare diseases. Learn More
Dr. John S. Kovach, CEO of Lixte, said “AS is a rare disease affecting approximately 20,000 persons in the US and approximately 500,000 worldwide. Its genetic cause, a mutation in a maternal gene called UBE3A, has been understood for some time but the molecular abnormality resulting from the genetic lesion has now been shown to be increased concentrations of protein phosphatase 2A (PP2A), the target of Lixte’s investigational compound, LB-100. This was a completely unanticipated finding. We can only hope that LB-100 may be of benefit to patients afflicted with this devastating life-long disorder.” Lixte Biotechnology Holdings, Inc. announced an agreement with the Foundation for Angelman Syndrome Therapeutics (FAST) to collaborate in supporting preclinical studies of the potential benefit of Lixte’s proprietary lead clinical compound, LB-100, in a mouse model of Angelman Syndrome (AS). Learn More
Radius Health is a global biopharmaceutical company focused on addressing unmet medical needs in the areas of bone health and neuroscience. Our team works collaboratively and relentlessly to advance our therapies with the goal of improving the lives of patients, their families, and their caregivers.
The Radius portfolio consists of commercial and clinical development assets that include mid to late-stage drug candidates.