Alnylam was founded in 2002 on a bold vision to turn scientific possibility into reality, which is now marked by its robust discovery platform and deep pipeline of investigational medicines. Alnylam is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options.
Alnylam aims to support doctors’ and patients’ understanding of Hereditary ATTR amyloidosis to address this devastating disease. Following the success of the 1st European meeting for ATTR amyloidosis, Alnylam is delighted to continue supporting this unique meeting and wishes the organisers, physicians and patients exceptional success.
Akcea Therapeutics , Inc., an affiliate of Ionis Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases. Akcea is advancing a mature pipeline of six novel drugs, including TEGSEDI™ (inotersen), WAYLIVRA™ (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. Akcea is building the infrastructure to commercialize its drugs globally. Akcea is a global company headquartered in Boston, Massachusetts. Additional information about Akcea is available at www.akceatx.com.
Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos is developing AG10, a potentially disease-modifying therapy for the treatment of ATTR. AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR.
AG10 is being studied in patients with ATTR-CM in an open-label extension of a Phase 2 clinical trial and in a pivotal, global Phase 3 clinical trial (ATTRibute-CM) that is currently enrolling.
Together with patients and physicians, Eidos aims to bring a safe, effective and disease-modifying treatment for ATTR to market.
Intellia Therapeutics is a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products.
Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.
Patients organisations and groups
Grupo Argentino de Familias con Amiloidosis (Argentina)
Amyloidosis Australia (Australia)
Leben mit Amyloidose-Amyloidosis (Austria)
Associação Brasileira de Paramiloidose (Brazil)
Canadian Amyloidosis Support Network (Canada)
Fundacion Colombiana Enfermedades Huerfanas (Colombia)
Association Française Contre l’Amylose (France)
Patientenverband “FAP e.V “ (Germany)
Amyloidosis Israel Org (Israel)
Associazione Italiana Amyloidosi Familiare Onlus (Italy)
Mitisirube no kai (Japan)
FAP Macedonia (Macedonia)
Fundacion de Familias con Amyloidosis in Mexico (Mexico)
Stichting Amyloidose Nederland (The Netherlands)
New Zealand ATTR Amyloidosis Patients Association (New Zealand)
Associaçao Portuguesa de Paramiloidose (Portugal)
Hereditary amyloidose hwanwoohoe (South Korea)
Asociacion Balear Enfermedad Andrade / Asociación Española de Amiloidosis / Asociación Valverdeña de la Enfermedad de Andrade (Spain)
Association FAMY-Norrbotten (Sweden)
ATTR Amyloidosis Patients Association (UK)
Amyloidosis Support Groups (US)
FAP Venezuela (Venezuela)