Collaboration based on new findings from the Regeneron Genetics Center® showing variant in HSD17B13 gene is associated with reduced risk of chronic liver diseases
TARRYTOWN, NY and CAMBRIDGE, MA, USA I March 21, 2018 I Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced a collaboration to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis (NASH) and potentially other related diseases. The discovery collaboration is based on a new Regeneron publication in the New England Journal of Medicine identifying for the first time a variant in the HSD17B13 gene that is associated with reduced risk of chronic liver diseases. RNA interference (RNAi) therapeutics represent a novel and innovative approach to specifically target and silence any gene involved in the cause or pathway of human disease; this approach could potentially mimic the naturally occurring loss-of-function genetic variation in HSD17B13 seen in people who are protected from NASH disease progression.
“At Alnylam, we are dedicated to advancing RNAi therapeutics as a new class of medicines for patients with few or no treatment alternatives,” said John Maraganore, Ph.D., Chief Executive Officer at Alnylam. “As we transition Alnylam toward commercialization in rare diseases, the prospect of collaborating with a scientific leader like Regeneron on innovative medicines for more prevalent diseases like NASH makes perfect strategic sense. We believe the exquisite specificity afforded by the RNAi mechanism of action and our industry-leading, proprietary GalNAc-conjugate approach for delivery to the liver is an unparalleled combination for developing an RNAi therapeutic toward genetically-validated targets in NASH.”
Under the discovery collaboration, Regeneron will contribute research on the hepatocyte-expressed, genetically-validated HSD17B13 target, and Alnylam will leverage its RNAi therapeutics platform to identify compounds directed to this target. Regeneron and Alnylam intend to enter into a separate, fifty-fifty collaboration to further research, co-develop and commercialize any therapeutic product candidates that emerge from these discovery efforts.
“Our Regeneron Genetics Center is delivering new targets that will require new approaches beyond our biologics capabilities. Since we are committed to following the science, we are pleased to join together with an equally science-minded company with a novel RNAi therapeutic approach that appears well-suited to impact this particular target,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. “NASH is a major cause of death in this country, with no current treatment options. We’re eager to build on the exciting science delivered by the Regeneron team in the hopes of helping patients with debilitating and life-threatening chronic liver diseases.”
Chronic liver disease and cirrhosis are leading causes of morbidity and mortality in the United States, accounting for over 38,000 deaths in 2014.1 The most common precursors to cirrhosis are alcoholic liver disease, chronic hepatitis C and nonalcoholic fatty liver disease (NAFLD). About 3 to 12 percent of adults in the United States have NASH,2 a more severe type of NAFLD, and prevalence is increasing due to rising rates of obesity.3,4 There is a great need for new medicines that treat non-viral forms of chronic liver disease, as there are currently no approved drugs for these conditions.
This is Regeneron and Alnylam’s second recent collaboration founded on genetic exploration. In January 2018, Alnylam, alongside multiple other leading life sciences companies, joined Regeneron’s pre-competitive consortium to sequence 500,000 individuals in the UK Biobank health resource and subsequently make the data publicly available to the global research community.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, and hepatic infectious diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including four product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 700 people in the U.S. and Europe and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune® which produces optimized fully-human antibodies, and ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
SOURCE: Regeneron Pharmaceuticals
Post Views: 251
Collaboration based on new findings from the Regeneron Genetics Center® showing variant in HSD17B13 gene is associated with reduced risk of chronic liver diseases
TARRYTOWN, NY and CAMBRIDGE, MA, USA I March 21, 2018 I Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) today announced a collaboration to identify RNAi therapeutics for the chronic liver disease nonalcoholic steatohepatitis (NASH) and potentially other related diseases. The discovery collaboration is based on a new Regeneron publication in the New England Journal of Medicine identifying for the first time a variant in the HSD17B13 gene that is associated with reduced risk of chronic liver diseases. RNA interference (RNAi) therapeutics represent a novel and innovative approach to specifically target and silence any gene involved in the cause or pathway of human disease; this approach could potentially mimic the naturally occurring loss-of-function genetic variation in HSD17B13 seen in people who are protected from NASH disease progression.
“At Alnylam, we are dedicated to advancing RNAi therapeutics as a new class of medicines for patients with few or no treatment alternatives,” said John Maraganore, Ph.D., Chief Executive Officer at Alnylam. “As we transition Alnylam toward commercialization in rare diseases, the prospect of collaborating with a scientific leader like Regeneron on innovative medicines for more prevalent diseases like NASH makes perfect strategic sense. We believe the exquisite specificity afforded by the RNAi mechanism of action and our industry-leading, proprietary GalNAc-conjugate approach for delivery to the liver is an unparalleled combination for developing an RNAi therapeutic toward genetically-validated targets in NASH.”
Under the discovery collaboration, Regeneron will contribute research on the hepatocyte-expressed, genetically-validated HSD17B13 target, and Alnylam will leverage its RNAi therapeutics platform to identify compounds directed to this target. Regeneron and Alnylam intend to enter into a separate, fifty-fifty collaboration to further research, co-develop and commercialize any therapeutic product candidates that emerge from these discovery efforts.
“Our Regeneron Genetics Center is delivering new targets that will require new approaches beyond our biologics capabilities. Since we are committed to following the science, we are pleased to join together with an equally science-minded company with a novel RNAi therapeutic approach that appears well-suited to impact this particular target,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. “NASH is a major cause of death in this country, with no current treatment options. We’re eager to build on the exciting science delivered by the Regeneron team in the hopes of helping patients with debilitating and life-threatening chronic liver diseases.”
Chronic liver disease and cirrhosis are leading causes of morbidity and mortality in the United States, accounting for over 38,000 deaths in 2014.1 The most common precursors to cirrhosis are alcoholic liver disease, chronic hepatitis C and nonalcoholic fatty liver disease (NAFLD). About 3 to 12 percent of adults in the United States have NASH,2 a more severe type of NAFLD, and prevalence is increasing due to rising rates of obesity.3,4 There is a great need for new medicines that treat non-viral forms of chronic liver disease, as there are currently no approved drugs for these conditions.
This is Regeneron and Alnylam’s second recent collaboration founded on genetic exploration. In January 2018, Alnylam, alongside multiple other leading life sciences companies, joined Regeneron’s pre-competitive consortium to sequence 500,000 individuals in the UK Biobank health resource and subsequently make the data publicly available to the global research community.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, and hepatic infectious diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including four product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 700 people in the U.S. and Europe and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to six FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, heart disease, allergic and inflammatory diseases, pain, cancer, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune® which produces optimized fully-human antibodies, and ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
SOURCE: Regeneron Pharmaceuticals
Post Views: 251
