Myotonia Congenita

TARRYTOWN, N.Y. and WASHINGTON, March 11, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Society for Science (the Society) announced that Matteo Paz,18, of Pasadena, CA, won the top award of $250,000 in the 2025 Regeneron Science Talent Search, the U.S.’s oldest and most prestigious science and math competition for high school seniors. Now in its 84th year, the competition celebrates and rewards young innovators who are applying their Science, Technology, Engineering and Math (STEM) talent and leadership skills to push the boundaries of discovery and address today’s pressing challenges. Forty finalists, including Matteo, were honored this evening during an award ceremony at the National Building Museum in Washington, D.C, where they were awarded more than $1.8 million in prizes for their groundbreaking research, exceptional problem-solving skills and potential to shape the future of STEM. Matteo Paz, 18, of Pasadena, CA, won first place and $250,000 for designing machine-learning algorithms to efficiently comb through 200 billion entries of raw NEOWISE infrared full-sky data. By analyzing tiny changes in infrared radiation, the AI sorted the objects into 10 classes. He found 1.5 million new potential objects. Second place and $175,000 went to Ava Grace Cummings, 18, of Smithfield, NC, for creating a fruit fly model of STAC3 disorder, or Native American myopathy (a rare genetic muscle disease). She found that the common nettle herb, alone or combined with the experimental drug Tirasemtiv, improved movement in both adult flies and larvae. Third place and $150,000 went to Owen Jianwen Zhang, 18, of Bellevue, WA, for solving a long-standing math problem about objects called 3-uniform hypergraphs. He proved a maximum value for how many 3-uniform hypergraphs can have similar structures but differing connections. Owen's results have applications in computer science. "Congratulations to the winners of this year's Regeneron Science Talent Search," said Maya Ajmera, President and CEO, Society for Science and Executive Publisher, Science News. "The remarkable creativity and dedication of these students bring renewed hope for our future. Driven by their ingenuity, these young scientists are developing groundbreaking solutions that have the potential to transform our world and propel society forward." The Regeneron Science Talent Search provides a national platform for high school seniors to showcase original, innovative STEM research that proposes novel solutions to real-world issues. Finalists are evaluated for their scientific rigor, originality, critical thinking, leadership potential and commitment to drive meaningful impact across crucial STEM fields. “The Science Talent Search changed my life. At my high school, STS winners were treated like star athletes, and I never imagined I would belong in such an amazing group of kids who were operating at a whole different level than I had ever seen,” said George D. Yancopoulos, co-Founder, Board co-Chair, President and Chief Scientific Officer of Regeneron and a 1976 Science Talent Search finalist and top winner. “The experience of competing in STS and being named a top winner gave me the confidence to devote my life to science. So, congratulations to this year’s finalists and winners, you are America’s best and brightest. I hope this moment inspires you to push boundaries, challenge assumptions and use your brilliance to change the world.” Other top honors from the competition include: Fourth Place: Logan Lee, 18, of Honolulu, HI received a $100,000 award for helping sterile male mosquitoes survive in the wild. These males are important in mosquito control. Logan improved their survival by giving them a transplant of wild mosquito bacteria. His transplant helped the sterile mosquitoes grow faster and survive better in the wild. Fifth Place: Rivka Lipkovitz, 18, of San Francisco, CA received a $90,000 award for using statistical modeling to study U.S. voter ID laws. She found that presidential election turnout dropped by 2.4% in states that passed strict laws after 2008. Turnout for midterm elections increased. Knowing how laws affect turnout can help shape future policies. Sixth Place: Melody Heeju Hong, 17, of Wantagh, NY received a $80,000 award for developing a powerful, flexible statistical model for mapping sites called trans-methylation quantitative trait loci (trans-mQTL) within the human genome. These sites are key to understanding the interplay between genes and environment in disease and aging. Seventh Place: Kevin Shen, 18, of Olympia, WA received a $70,000 award for building a custom flight computer to control a 3D-printed airplane with oblique wings. These aircraft can be more fuel-efficient but are hard to control. His oblique-wing aircraft and flight computer improved flight efficiency by 9.2%. Eighth Place: Minghao Zou, 18, of Santa Clara, CA received a $60,000 award for simulating protons to probe environments that produce subatomic particles called neutrinos. He created an algorithm mimicking extreme astrophysical conditions, such as electromagnetic and gravitational forces and interactions with nearby particles. He verified it using known cases of particle motion. Ninth Place: Thanush Patlolla, 17, of Cary, NC received a $50,000 award for approximating the density of electrons using a finite nuclear model. Using a mathematical strategy called a density function, he created a model to map electrons in a nuclear simulation. The map increased the accuracy of energy distribution predictions by 0.6%. Tenth Place: Ray Zhang, 17, of Chantilly, VA received a $40,000 award for studying how to better treat drug-resistant Fusarium fungal infections. Ray studied how the fungus builds sticky communities of cells that resist drug treatment. He also found that using a combination of drugs better controlled the fungus. Akilan Sankaran, 17, of Albuquerque, NM was named the Seaborg Award winner and selected to speak on behalf of the Regeneron Science Talent Search Class of 2025. The 40 finalists chose Akilan as the student who best exemplifies their class and the legacy of nuclear chemist Glenn T. Seaborg, who won the Nobel Prize for Chemistry in 1951 and served on the Society’s Board of Trustees for 30 years. All other finalists received $25,000. All 40 finalists join a distinguished group of Science Talent Search alumni, many of whom have gone on to achieve world-changing careers in STEM, with some earning esteemed honors, including the Nobel Prize, National Medal of Science, and MacArthur Fellowship. In total, Regeneron awarded $3.1 million in prizes, including $2,000 to each top scholar and their school. Learn more about Regeneron Science Talent Search at https://www.societyforscience.org/regeneron-sts/ and learn about all their research projects at our Virtual Public Showcase. For media resources, visit https://www.societyforscience.org/2025-regeneron-science-talent-search-media-kit About Society for Science Society for Science is a champion for science, dedicated to promoting the understanding and appreciation of science and the vital role it plays in human advancement. Established in 1921, Society for Science is best known for its award-winning journalism through Science News and Science News Explores, its world-class science research competitions for students, including the Regeneron Science Talent Search, the Regeneron International Science and Engineering Fair and the Thermo Fisher Scientific Junior Innovators Challenge, and its outreach and equity programming that seeks to ensure that all students have an opportunity to pursue a career in STEM. A 501(c)(3) membership organization, Society for Science is committed to inform, educate and inspire. Learn more at www.societyforscience.org and follow us on Facebook, Twitter, Instagram and Snapchat (Society4Science). About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases. Regeneron believes that operating as a good corporate citizen is crucial to delivering on our mission. We approach corporate responsibility with three goals in mind: to improve the lives of people with serious disease, to foster a culture of integrity and excellence and to build sustainable communities. Regeneron is proud to be included on the Dow Jones Sustainability World Index and the Civic 50 list of the most “community-minded” companies in the U.S. Throughout the year, Regeneron empowers and supports employees to give back through our volunteering, pro-bono and matching gift programs. Our most significant philanthropic commitments are in the area of science education, including the Regeneron Science Talent Search and the Regeneron International Science and Engineering Fair (ISEF). For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Media Contacts Joseph Brown, Regeneron 386-283-1323, joseph.brown2@regeneron.com Gayle Kansagor, Society for Science 703-489-1131, gkansagor@societyforscience.org Source: Regeneron Pharmaceuticals, Inc.

HAYWARD, Calif., April 09, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced it will host a virtual R&D Day, on Thursday, April 18, 2024 from 9:00 AM to 11:00 AM ET. To register, click here. The event will feature two key opinion leaders who will discuss the clinical symptoms and the natural history of oculopharyngeal muscular dystrophy (OPMD), a rare genetic muscle disorder. The key opinion leaders will also review the clinical and radiographic methods employed to evaluate disease progression and discuss the current treatment landscape for patients diagnosed with OPMD. Bernard Brais, MDCM, PhD - Professor, Department of Neurology and Neurosurgery, Montreal Neurological Institute, McGill University Health CentreEmily Plowman, PhD, CCC-SLP, FASHA - Professor, Department of Otolaryngology - Head and Neck Surgery, The Ohio State University College of Medicine The event will focus on the Company’s clinical development program for its investigational gene therapy agent BB-301, which is currently being evaluated in a Phase 1b/2a clinical trial for the treatment of OPMD-derived dysphagia. Key areas of focus will include discussions of clinical, radiographic, and subject-reported assessments of disease progression, clinical study design, and the specific primary and secondary endpoints that will be used to quantify subject improvement over the course of the BB-301 clinical development program. A live question and answer session will follow the formal presentations. About Bernard Brais, MDCM, PhD Bernard Brais, MDCM, PhD, Professor, Department of Neurology and Neurosurgery at Montreal Neurological Institute (MNI), McGill University Health Centre (MGH, MNH) is Director of the Rare Neurological Diseases Group. He completed his MDCM, neurology residency and PhD at McGill. He is also trained as a historian of neurosciences and genetics. His research largely focuses on the genetic basis of neurogenetic disorders with founder effects in Quebec, with an increasing focus on disorders with ataxic manifestations such as Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS). Since 2007, he has headed a team of researchers on ARSACS. Dr. Brais has played important roles in identifying causal genes for Oculopharyngeal muscular dystrophy (OPMD), Hereditary Sensory and Autonomic Neuropathy type II (HSANII), Limb Girdle Muscular Dystrophy with Quadriceps atrophy (LGMD2L), Pol III-related leukodystrophies, and ZAK congenital myopathy. About Emily Plowman, PhD, CCC-SLP, FASHA Emily Plowman, PhD, CCC-SLP, FASHA, Professor, Department of Otolaryngology – Head and Neck Surgery, The Ohio State University College of Medicine is Director of the Aerodigestive Research Core across its two sites at the Ohio State University and University of Florida and Director of the Wexner Medical Center Dysphagia Program. She is an internationally recognized expert in the field of dysphagia who has held uninterrupted funding from the National Institutes of Health (NIH) since commencing her academic career in 2009. Her current research at OSU and UF are supported by the National Institute of Aging, National Institute of Nursing Research, National Institute of Cancer, Department of Defense, and the ALS Association. Dr. Plowman has authored over 85 peer-reviewed scientific manuscripts, given over 600 lectures worldwide, and obtained over 30 external research grants. In addition to her own research, Dr. Plowman is passionate about mentoring the future generation of clinician scientists and her mentorship efforts were recently recognized by the National Institutes of Health with the NINDS Story Landis Award for Outstanding Mentorship by a Neuroscientist (2022) and the University of Florida Doctoral Mentor of the Year award (2021). She was inducted into the American Speech and Hearing Association as a Fellow in 2022 and was elected to be the incoming President of the international Dysphagia Research Society for 2026. About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. About Benitec Biopharma, Inc. Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com. Forward-Looking Statements Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of pre-clinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements. These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company's ability to protect and enforce its patents and other intellectual property rights; the Company's dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company's ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of the COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus and similar events, which may adversely impact the Company's business and pre-clinical and future clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. Investor Relations Contact: Irina KofflerLifeSci Advisors, LLC(917) 734-7387ikoffler@lifesciadvisors.com

CAMBRIDGE, Mass. and ROSTOCK, Germany and BERLIN, March 08, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its conference schedule for March 2023. CENTOGENE representatives will be attending events in Brazil, the U.S., Saudi Arabia, Thailand, and the U.K. and will be available to discuss Pharma, CRO, and Diagnostic collaboration opportunities. The Company invites attendees to schedule one-on-one meetings in advance. Please see additional details below: 3rd Brazilian Congress of Neurogenetics 2023 Dates: March 9-11, 2023 Location: Frei Caneca Convention Center, São Paulo, Brazil CENTOGENE’s Presentations: Title: Increased diagnostic precision with insights from a rare disease biodatabank Speaker: Prof. Peter Bauer, CENTOGENE's Chief Medical & Genomic Officer When: Friday, March 10, 2023, 8:30 a.m. – 8:55 a.m. BRT Where: Room Turquesa 2 Title: Multiomics: A new era of rare disease diagnostics Speaker: Prof. Peter Bauer, CENTOGENE's Chief Medical & Genomic Officer When: Friday, March 10, 2023, 3:00 p.m. – 3:35 p.m. BRT Where: Room Turquesa 1 Title: ROUNDTABLE - My personal view on the future of neurogenetics in the next 5-10 years Speaker: Prof. Peter Bauer, CENTOGENE's Chief Medical & Genomic Officer When: Saturday, March 11, 2023, 3:50 p.m. – 5:05 p.m. BRT Where: Room Turquesa 1 and 2 To learn more about the 3rd Brazilian Congress of Neurogenetics 2023 and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( ). American College of Medical Genetics & Genomics (ACMG) Annual Clinical Genetics Meeting Dates: March 14-18, 2023 Location: Salt Palace Convention Center, Salt Lake City, U.S. CENTOGENE’s Poster Presentations: Title: Beyond genomics: Using RNA-seq in filter cards to unlock the clinical relevance Speaker: Jorge Pinto Basto, Senior Medical Director at CENTOGENE When: Thursday, March 16, 2023, from 10:30 a.m. – 12:00 p.m. MDT Where: Exhibit Hall (Abstract #P427) Title: Impact of processed pseudogene insertions in genetic testing as cause of monogenic diseases: Insertion in CLCN1 gene causing myotonia congenita Speaker: Jorge Pinto Basto, Senior Medical Director at CENTOGENE When: Friday, March 17, 2023, from 10:30 a.m. – 12:00 p.m. MDT Where: Exhibit Hall (Abstract #P576) For information about the ACMG Annual Clinical Genetics Meeting and to arrange a one-on-one meeting, please refer to the event webpage ( ). World Rare Disease Day Conference 2023 Dates: March 15-16, 2023 Location: King Salman Abdul Aziz Medical City, Medina, Saudi Arabia To learn more about the World Rare Disease Day Conference 2023 and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( ). 6th Annual Gene Therapy for Rare Disorders 2023 Summit Dates: March 20-23, 2023 Location: Sheraton Boston Hotel, Boston, U.S. For information about the 6th Annual Gene Therapy for Rare Disorders 2023 Summit and to arrange a one-on-one meeting, please refer to the event webpage ( ). The 6th Asian Congress on Inherited Metabolic Diseases 2023 Dates: March 22-24, 2023 Location: Arnoma Grand Bangkok, Bangkok, Thailand To learn more about the 6th Asian Congress on Inherited Metabolic Diseases 2023 and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( ). Pharma USA 2023 Dates: March 28-29, 2023 Location: Pennsylvania Convention Center, Philadelphia, U.S. For information about Pharma USA 2023 and to arrange a one-on-one meeting, please refer to the event webpage ( ). Research & Innovation 2023 Meeting Dates: March 29-30, 2023 Location: Hinxton Hall Conference Centre, Hinxton, U.K. CENTOGENE’s Presentations: Title: Using patient data to enable drug discovery in rare diseases Speaker: Guido Hartmann, VP Global Head Research and Development at CENTOGENE When: Wednesday, March 29, 2023, 11:10 a.m. BST Where: Rosalind Franklin Room To learn more about the European Laboratory Research & Innovation Group’s (ELRIG) Research & Innovation 2023 conference and set up an in-person meeting with the CENTOGENE team, please visit the event webpage ( ). About CENTOGENE CENTOGENE’s mission is to provide data-driven, life-changing answers to patients, physicians, and pharma companies for rare and neurodegenerative diseases. We integrate multiomic technologies with the CENTOGENE Biodatabank – providing dimensional analysis to guide the next generation of precision medicine. Our unique approach enables rapid and reliable diagnosis for patients, supports a more precise physician understanding of disease states, and accelerates and de-risks targeted pharma drug discovery, development, and commercialization. Since our founding in 2006, CENTOGENE has been offering rapid and reliable diagnosis – building a network of approximately 30,000 active physicians. Our ISO, CAP, and CLIA certified multiomic reference laboratories in Germany utilize Phenomic, Genomic, Transcriptomic, Epigenomic, Proteomic, and Metabolomic datasets. This data is captured in our CENTOGENE Biodatabank, with nearly 700,000 patients represented from over 120 highly diverse countries, over 70% of whom are of non-European descent. To date, the CENTOGENE Biodatabank has contributed to generating novel insights for more than 260 peer-reviewed publications. By translating our data and expertise into tangible insights, we have supported over 50 collaborations with pharma partners. Together, we accelerate and de-risk drug discovery, development, and commercialization in target and drug screening, clinical development, market access and expansion, as well as offering the CENTOGENE Biodatabank Licenses and Insight Reports to enable a world healed of all rare and neurodegenerative diseases. To discover more about our products, pipeline, and patient-driven purpose, visit and follow us on LinkedIn. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the U.S. federal securities laws. Statements contained herein that are not clearly historical in nature are forward-looking, and the words “anticipate,” “believe,” “continues,” “expect,” “estimate,” “intend,” “project,” and similar expressions and future or conditional verbs such as “will,” “would,” “should,” “could,” “might,” “can,” and “may,” are generally intended to identify forward-looking statements. Such forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause CENTOGENE’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward- looking statements. Such risks and uncertainties include, among others, negative economic and geopolitical conditions and instability and volatility in the worldwide financial markets, possible changes in current and proposed legislation, regulations and governmental policies, pressures from increasing competition and consolidation in our industry, the expense and uncertainty of regulatory approval, including from the U.S. Food and Drug Administration, our reliance on third parties and collaboration partners, including our ability to manage growth, execute our business strategy and enter into new client relationships, our dependency on the rare disease industry, our ability to manage international expansion, our reliance on key personnel, our reliance on intellectual property protection, fluctuations of our operating results due to the effect of exchange rates, our ability to streamline cash usage, our continued ongoing compliance with covenants linked to financial instruments, our requirement for additional financing, or other factors. For further information on the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to CENTOGENE’s business in general, see CENTOGENE’s risk factors set forth in CENTOGENE’s Form 20-F filed on March 31, 2022, with the Securities and Exchange Commission (the “SEC”) and subsequent filings with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and CENTOGENE’s specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise. Media Contact: CENTOGENE Ben Legg Corporate Communications Press@centogene.com Lennart Streibel Investor Relations IR@centogene.com