Megacystis Microcolon Intestinal Hypoperistalsis Syndrome

Three New RFPs Now Open for Qualified Researchers of AVM, LYS, and MMIHS DANBURY, Conn., Sept. 19, 2023 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) announced three new grant funding opportunities related to the following rare diseases: Arteriovenous Malformation (AVM), Levy-Yeboa Syndrome (LYS), and Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS). NORD's Research Grants Program provides seed-money grants to qualified investigators for scientific and clinical research into diseases for which there are few other sources of funding. These grants are competitive and international. Continue Reading The National Organization for Rare Disorders (NORD) announces three new research opportunities for AVM, LYS, and MMIHS. Tweet this Photo of a medical researcher looking through a microscope With funding from the Tyler James Abizeid Foundation, NORD is accepting applications for one grant of up to $35,000 US, for scientific and/or clinical research studies related to Arteriovenous Malformation (AVM). Deadline for initial applications is Monday, October 16. View more information and apply here. With funding by the Maxwell Family, NORD is accepting applications for one grant of up to $40,000 US, for scientific and/or clinical research studies related to Levy-Yeboa syndrome (LYS). Deadline for initial applications is Monday, October 16. View more information and apply here. With funding from the MMIHS Foundation, NORD is accepting applications for one grant of $30,000 US for scientific and/or clinical research studies related to megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS). Deadline for initial applications is Monday, October 16. View more information and apply here. Grants will be awarded to qualified researchers to initiate small scientific research studies or clinical trials, the results of which could be used to obtain funding from the NIH, FDA, or other funding agencies, or to attract a corporate sponsor. "Since 1989, NORD's grants have led to the development of two FDA-approved treatments and many peer-reviewed publications. Over 200 grants have been awarded, totaling over $9 million in approved funding. The vision behind these critical study grants is to ultimately lead to new diagnostics, treatments, and cures for rare diseases," said Aliza Fink, Director, Research Programs, NORD. To learn more or submit to the 2023 NORD Research Grants Cycle, visit rarediseases.org/advancing-research/request-for-proposals/ About the National Organization for Rare Disorders (NORD) With a 40-year history of advancing care, treatments and policy, the National Organization for Rare Disorders (NORD) is the leading and longest-standing patient advocacy group for the 25-30 million Americans living with a rare disease. NORD, a 501(c)(3) nonprofit, is dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 330 patient organization members, is committed to improving the health and well-being of people with rare diseases by driving advances in care, research and policy. For more information, please visit . SOURCE National Organization for Rare Disorders (NORD)

L’AQUILA, Italy, Dec. 12, 2022 (GLOBE NEWSWIRE) -- Genomic Biopharma Inc., a genomic medicine-based drug discovery and development company, has partnered with Associazione Poic e Dintorni APS (“POIC e dintorni APS”), a patient organization that supports the advocacy of chronic intestinal pseudo-obstruction (CIPO), to enable access to whole genome sequencing (WGS) and demonstrate the utility of genomic information to treat and cure the most severe disorders of gut motility, including: ACTG2 mediated Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS) Chronic Intestinal Pseudo Obstruction (CIPO) Associazione Poic e dintorni APS is an Italian patient organization that supports the advocacy of CIPO, a rare and disabling disease characterized by a severe myogenic, neurogenic or idiopathic motility defect that affects the entire digestive tract, associated with periods of intestinal obstruction and inability to feed the patients. Through the collaboration, all members of POIC e dintorni APS and their parents will have access to whole genome sequencing. Among rare, severe and disabling intestinal diseases, Genomic Biopharma will target one of its first drug candidates, demonstrating the clinical utility of genomic information in drug discovery and development for the treatment of rare diseases. At the American Society of Human Genetics conference in October, Genomic Biopharma presented a poster that identified siRNA sequences as good candidates for the development of a new therapeutic approach in MMIHS and underscored a translational impact for future strategy to treat the underlying cause of the disease. “For those living with ultra rare diseases, in particular, the only approach is to take the broadest and most comprehensive look at the person’s genomic pro whole genome sequencing,” said Mattia Capulli, PhD, co-founder of Genomic Biopharma. “It is our mission to push genomics to new frontiers in order to develop treatments for diseases for which there are no cure or resolutive pharmacological therapies. In partnering with progressive organizations like Poic dintorni APS, we will pave the way to cure the most severe disorders of gut motility.” “As a mother of a daughter affected by CIPO since her birth and as President of a patient organization that supports the advocacy of chronic intestinal pseudo-obstruction, I am very excited about the potential that this partnership with Genomic Biopharma offers us. It clearly shows the joint-commitment to improve patients' quality of life,” said Ornella Spada, President and co-founder of Poic e dintorni APS. “Starting today, the word hope has a new and encouraging meaning for families, caregivers and patients affected by CIPO.” Another goal of the partnership is to raise awareness of MMIHS and CIPO around the world in order to bring together the world’s most knowledgeable experts from clinicians to patient advocates to truly foster the development of an international alliance of pediatric and adult patients. About Genomic Biopharma Inc. Genomic Biopharma Inc., a company created by Dante Genomics, is a drug discovery and development company building a pipeline of drug programs focused on bringing to market treatments and therapies for infectious and rare diseases. The Company develops its pipeline based on findings from Dante Genomics’ proprietary database of whole genomes. Genomic Biopharma’s core technology, the GBI AI, uses predictive algorithms and genomic data mining to support the identification of novel RNA-based therapeutics. About Associazione Poic e dintorni APS Associazione Poic e dintorni APS is an Italian patient organization that supports the advocacy of chronic intestinal pseudo-obstruction (CIPO), a rare and disabling disease characterized by a severe myogenic, neurogenic or idiopathic motility defect that affects the entire digestive tract, associated with periods of intestinal obstruction and inability to feed the patients. Its mission is to improve the quality of life of patients and to accelerate scientific research to find a cure. Poic e dintorni APS relies also on its Scientific Advisory Board leadership, made up by 18 members with different clinic-scientific background and internationally acknowledged. Learn more about CIPO here. Contact: Genomic Biopharma Inc. info@genomicbiopharma.com Associazione Poic e dintorni APS alex@poic-e-dintorni.org

– Genomic Biopharma Inc. to present abstracts on two drug candidate programs at the 2022 American Society of Human Genetics (ASHG) Annual Meeting –NEW YORK, Oct. 26, 2022 (GLOBE NEWSWIRE) -- Dante Genomics, a global leader in genomics and precision medicine, today announced the creation of Genomic Biopharma Inc., a separate drug discovery and development company to distinguish the separate business streams of each entity. Genomic Biopharma Inc. will present two abstracts on two drug programs at the 2022 American Society of Human Genetics (ASHG) Annual Meeting, the first of which is a top scoring abstract at the meeting titled: Abstract Title: A computational approach to design a COVID-19 vaccine against a predicted SARS-CoV-2 variant: high immunogenicity, efficacy and safety of DELLERA vaccine Presentation Date/Time: Wednesday, October 26, 2022, 3:00 PM - 4:45 PM Pacific Time Abstract Title: Novel siRNA-based therapeutic approach for Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS) Presentation Date/Time: Thursday, October 27, 2022, 3:00 PM - 4:45 PM Pacific Time “Dante Genomics has delivered genomic insights and diagnoses to thousands of individuals, ending many years-long diagnostic odysseys,” said Mattia Capulli, Chief Scientific Officer of Dante Genomics. “Through the creation of Genomic Biopharma Inc. we will realize our vision to support the discovery and development of personalized medicine, so that no one is diagnosed with a disease for which there is no treatment.” Genomic Biopharma Inc. was created as a fully integrated drug discovery and development company, with a pipeline of drug programs for treatments and therapies focused on infectious and rare diseases. The Company develops its pipeline based on findings from Dante Genomics’ proprietary database of whole genomes. Genomic Biopharma’s core technology, the GBI AI, uses predictive algorithms and genomic data mining to support the identification of novel RNA-based therapeutics. “Personalized medicine requires an integrated approach from data to clinical trials,” said Andrea Riposati, CEO of Dante Genomics. “We created Genomic Biopharma as a separate, independent company, to achieve the vision of personalized medicine and unlock the new era of genomic data based therapeutics.” Dante Genomics provided initial funding and resources to Genomic Biopharma Inc. to kick off the new entity’s endeavors and programs. The two companies are now independent. About Genomic Biopharma Inc. Genomic Biopharma Inc. is a drug discovery and development company building a pipeline of drug programs focused on bringing to market treatments and therapies for infectious and rare diseases. The Company develops its pipeline based on findings from Dante Genomics’ proprietary database of whole genomes. Genomic Biopharma’s core technology, the GBI AI, uses predictive algorithms and genomic data mining to support the identification of novel RNA-based therapeutics. Contact:info@genomicbiopharma.com www.genomicbiopharma.com