Drug Repurposing for Rare
We are incredibly grateful for the generosity of all our sponsors and supporters of the Drug Repurposing for Rare Diseases Conference 2020.
2020 Corporate Partner
Healx is an AI-powered and patient-inspired technology company, accelerating the discovery and development of rare disease treatments. Their AI drug discovery platform leverages public and proprietary biomedical data and features the world’s leading knowledge graph for rare diseases.
Combining their technology with patient insights and drug discovery expertise, Healx’s mission is to advance 100 rare disease treatments towards the clinic by 2025.
LifeArc is a self-funded medical research charity. Our work advances the translation of early science along the path to healthcare treatments or diagnostics. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines. Our success allows us to explore new approaches to advise, research and fund translation.
Our model is built on collaboration, and we partner with a broad range of groups including medical research charities, research organisations, industry and academic scientists. We are motivated by patient need and scientific opportunity.
Newron is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, Italy.
In addition to Xadago®/safinamide for Parkinson’s disease, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia.
Sarizotan - a potential first ever drug approved for Rett Syndrome
Sarizotan, a new chemical entity licensed from Merck KGaA, is a highly selective compound for specific serotonin or dopamine receptors that modulates the activity of these neurotransmitters in the brain. As Sarizotan was originally developed in another indication, the compound was licensed with an extensive safety and tolerability data package. Sarizotan could become the first drug approved for Rett syndrome; a debilitating genetic disorder with no specific treatment options.
Newron is currently performing the “Sarizotan Treatment of Apneas in Rett Syndrome” (STARS) study, a potentially pivotal clinical study performed in study centers in the US, Europe, Asia and Australia to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome and its impact on Quality of Life.
As part of its commitment to the rare disease patient community, Newron is partnering with the global Rett community to work on the largest International Burden of Illness (BOI) study. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. The learnings can help identify improved intervention programs and services designed to complement the Rett care pathway.
Ergomed and PSR Orphan Experts joined forces in 2017 to create a leading orphan drug development team that allows our clients to implement patient-driven solutions in their clinical trials. We recognise that clinical studies in rare diseases are not just smaller versions of conventional trials, and have specific requirements. Our unique expertise has helped us support over 100 clinical trials and observational studies in rare and ultra-rare indications.
We understand the challenges faced during orphan drug development, as well as how important the patient and their families’ experiences are in order to keep them engaged in potentially life changing trials. Our input to delivering new treatments, our Patient Engagement Officer and recently formed Patient Organisation Advisory Board demonstrate our dedication and passion for the area of rare diseases, in business as well as at the patient level. We are proud to be experts in the regulatory and clinical development of orphan drugs, working closely with patients, regulators, key opinion leaders, drug development companies and other stakeholders to streamline the development of safe and effective therapies. WE CARE ABOUT RARE!
Sobi is an international rare disease company dedicated to providing access to innovative treatments that make a significant difference for people with rare diseases. We have two business areas: Haemophilia and Specialty Care. Our research and product portfolio is primarily focused on haemophilia, inflammation, and genetic and metabolic diseases.
The United Kingdom and Republic of Ireland team is based in Cambridge and Sobi has been rated as the best company in Haemophilia in the UK through an annual independent survey in 2016, 2017 and 2018 (1st, 1st & 2nd) and one of the UK’s great places to work in 2017 and 2018.
Our sponsors have provided financial support for this event. This event has been organised by Findacure and our sponsors have not determined the content or organisation. All funds received by pharmaceutical companies have been used in accordance with the ABPI code.
How can you get involved?
In supporting Findacure’s vital work, your organisation will make a life changing difference to the millions of rare disease patients and their families. By partnering with our Drug Repurposing for Rare Diseases Conference, you will be providing an opportunity for all members of the rare disease community to meet, form new collaborations, and spread awareness of rare diseases and repurposing developments.
What opportunities are available?
We have a range of sponsorship and exhibition packages, each tailored to the needs of our supporters. Whatever you’re looking for, whether it be to show your support for the rare disease field, promote a new service, or establish your credentials as a leader in repurposing, we have a package for you.
For more information, contact our Head of Operations, Mary Rose Roberts.
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