Rare Drug Development Symposium

The 2022 RARE Drug Development Symposium kicks off June 8, and Global Genes is ecstatic about the speaker lineup for all of the sessions this year. Going with this year’s theme of disrupting innovation, Global Genes is going for a different approach with sessions this year. Formats will be more laid back and inclusive, with fireside chats to start the day, and workshop-style sessions with experts and peers throughout the day that will be offered at multiple times so you don’t miss any content. 

AI-Driven Screening Platforms & New Approaches to Therapeutics

Big data, acquired over years across multiple rare diseases, and made even more meaningful by adding samples from patient communities, provides an unparalleled opportunity to accelerate progress.  AI-driven approaches, such as high throughput screening (HTS) combined with statistical modeling can drive discovery, shape predictions, and assist in the targeted design of disease therapeutics. Understanding the potential of these applications will provide deeper insight into novel ways to help shorten the ‘end-to-end’ timeline, identify both specific- and broad-based disease therapies, and yield broader insight into mechanisms of disease convergence. But, the ‘human interaction component’ still remains essential to the success of advancing such approaches. In this session, we’ll look at how machine learning can help identify potential novel therapies and approved therapies that can be repurposed, and share thoughts on how regulatory policies will need to align with this rapidly evolving area of research.

 

Darius John Adams, MD
Division Chief of Genetics and Metabolism, Goryeb Children’s Hospital 
Scientific Advisory Board Member, INADCure Foundation 

Dr. Adams sits on the scientific advisory board of the INADcure foundation and is involved in clinical trial research not only for Infantile Neuroaxonal Dystrophy but also phenylketonuria, fragile X syndrome and lysosomal storage disorders. 

Dr. Adams completed his internship and residency in Genetics at the Mount Sinai Medical Center in New York City, where he received comprehensive training in genetics and dysmorphology. He remained at Mount Sinai for an additional year to complete a fellowship in Metabolic/Biochemical Genetics. Dr. Adams is a board certified Clinical and Biochemical. Dr. Adams joined Goryeb Children’s Hospital in 2013 as the Division Chief of Goryeb Children’s Hospital Genetics and Metabolism. He also coordinates the regional NJ Newborn Screening referral center and the Lysosomal Storage Disease program at Atlantic Health System. 

Learn More:
INADCure Foundation

Simi T. Ahmed, PhD 
Senior Vice President, Strategic Partnerships, New York Stem Cell Foundation

Simi Ahmed is an immunologist with 10+ years of experience in strategic planning and scientific portfolio development in the non-profit sector.  Currently, Simi leads strategic partnerships at the New York Stem Cell Foundation (NYSCF). Partnership projects include disease modeling efforts using stem cells, iPSC enabled patient-centric drug screening, and the generation of candidate cell therapies for complex and neglected diseases, including Rare Diseases.

Prior to joining NYSCF, Simi was a portfolio lead at JDRF, where she spearheaded multi-stakeholder initiatives worldwide to accelerate therapeutic development. She received her undergraduate degree in Biophysical Chemistry from Dartmouth College, a PhD in Immunology from the Weill Cornell Graduate School of Medical Sciences and completed her postdoctoral training with Dr. James Darnell Jr. at the Rockefeller University.

Learn More:
New York Stem Cell Foundation Website
Artificial Intelligence and Robotics Uncover Hidden Signatures of Parkinson’s Disease

Christopher Hart, PhD
Co-Founder, CEO and President, Creyon Bio

Chris is an experienced leader of interdisciplinary teams with decades of experience leveraging computational methods, machine learning and AI, and deep biological insights to solve problems. Prior to creating Creyon Bio, he built the functional genomics department at Ionis Pharmaceuticals. He was responsible for the company-wide genomics and bioinformatics efforts, including the development, running, and processing of all next-generation sequencing assays, development of research software informatics, and execution and strategic leadership of translational ‘omics and exploratory drug discovery programs that included programs in rare and common diseases. As a tenure-track faculty member at New College of Florida and a researcher at Lovelace Respiratory Research Institute, he researched and taught bioinformatics and computational biology. Prior to his work in the pharmaceutical industry, he worked at the Science and Technology Policy Institute advising the White House Office of Science and Technology Policy on matters ranging from personalized medicine to international health research funding. Chris earned his Ph.D. in Biology and Computer Science Applications in Biotechnology from the California Institute of Technology and conducted post-doctoral training at Yale University.

Learn More:
Creyon Bio
Creyon Debuts to Disrupt Oligonucleotide Research 
Creyon Bio is Creating Precision Medicines for Diseases Common and Rare
Artificial Intelligence and Machine Learning to Accelerate Translational Research: Proceedings of a Workshop – in Brief
Can Machine-learning Models Overcome Biased Datasets? MIT News, Massachusetts Institute of Technology

Frederick (Rick) Monsma, PhD
Senior Vice-President of Scientific Operations, New York Stem Cell Foundation (NYSCF)

The expansion of big data acquired over years and across multiple RAREs has created an evolving rich opportunity to find meaningful solutions with “what we have in hand – now”. Computational machine-based algorithms can be leveraged to identify in an unbiased manner baseline features of data or disease models that were previously unknown or under-appreciated.  The New York Stem Cell Foundation (NYSCF) exemplifies AI-driven approaches, such as high throughput screening (HTS) combined with statistical modeling that can drive discovery, shape predictions, and assist in the targeted design of disease therapeutics.

The New York Stem Cell Foundation’s SVP of Scientific Operations, Rick Monsma, oversees all laboratory operations at NYSCF, including day to day functioning, project planning, and strategic development. Rick started his career as a post-doc at the NIH and thereafter moved to an industry position with Hoffman-LaRoche in Basel, Switzerland where he focused on the identification and characterization of G-protein Coupled Receptors. From Roche, Rick was recruited to Schering-Plough to set up and lead a group devoted to identifying ligands for orphan GPCRs. Later, he led the High Throughput Screening (HTS) group at Schering-Plough, including development of capabilities to address screening of several million compounds in 1536 well format. Following the Merck/Schering merger, Rick transitioned to leading a Cell Pharmacology group, which developed and conducted diverse cell-based assays in support of Drug Discovery projects in Neuroscience, Diabetes and Cardiovascular Diseases.

Learn More:
NYSCF’s Mission and Approach
Stem Cells 101
Rare Disease Research at NYSCF
NYSCF Drug Discovery Platform

Amina Ann Qutub, PhD
VP of Computational Biology, Rarebase 

The Rarebase team understands  that there are thousands of rare diseases, so rather than approaching them one by one, they are tackling rare drug discovery at scale. Amina’s work pioneering technologies at the interface of computer science, neurovascular biology and engineering is bringing the needed knowledge for this greater goal. 

Amina joined the Rarebase team as VP of Computational Biology in January of 2022. Leading the computational thrust of Rarebase and working closely with bench scientists, Amina and her team are developing mathematical models and an artificial intelligence platform to identify the best therapies for use in children and adults with rare diseases. By systematically interrogating the network biology of many rare genetic mutations experimentally and computationally, the team has the ability to accelerate the path to clinical treatment. Amina is also an Associate Professor in Biomedical Engineering at the University of Texas, San Antonio (UTSA), and a research thrust lead of the Artificial Intelligence MATRIX Consortium. The MATRIX consists of more than sixty researchers focused on novel artificial intelligence applications to improve human wellbeing and augment human performance. Amina received her PhD in Bioengineering from UC Berkeley and UCSF, with a major in mathematical modeling and minor in neurology, and completed her postdoc as a National Institute of Health NRSA fellow in Biomedical Engineering at Johns Hopkins University, School of Medicine. She is an American Institute for Medical and Biological Engineering Fellow, National Academies Keck Future Initiatives Awardee and National Science Foundation CAREER Awardee. She also serves as a standing member of the U.S. National Academies of Science, Engineering and Medicine’s Biotechnology Capabilities and National Security Needs.

Rarebase partners with families and patient communities affected by rare disease to search for new applications for drugs already on the market. Their goal is to find associations between these drugs and rare diseases, in order to help patients in a rapid, cost-effective and safe way. Amina is excited to harness her experience to move Rarebase’s mission forward and help accomplish the Rarebase vision of applying cutting-edge computational and experimental methods to rapidly advance therapeutic discovery for rare disorders.

Learn More:
Rarebase
Rarebase: A Path Forward for One or Many
Amina’s TedX Houston talk

 

See other workshops and speaker lineups:
Disruptive Innovations in Clinical Trials Workshop
Emerging Models & Partnerships
Fostering Successful Connections Between Stakeholders to Accelerate Progress

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