The World’s Most Important Orphan Drug & Rare Disease Event – Less Than 2 Weeks Away!
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The World’s Most Important Orphan Drug & Rare Disease Event – Less Than 2 Weeks Away!
Dr. Mathew T. Thomas is a medical doctor who retired from the U.S. Food and Drug Administration (FDA) in June 2023, after more than 34 years in regulatory, management, and leadership positions. He founded MTT Consulting LLC in September 2023 and offers expert advice as an independent consultant for domestic and international entities engaged in FDA regulated medical product development. His expertise includes basic and clinical research, IRB functions, GCP protocol development, review of regulatory compliance, use of artificial intelligence, mock inspections, data integrity audits, post-inspectional remediations, and CAPAs. He is on the Board of Directors for IndoUSrare and on the GEO Network committee of the U.S. FDA Alumni Association.
In the FDA, he served as a medical reviewer for new drugs, a scientific reviewer for BIMO inspections, a health science administrator for orphan product development, the India Country Director of the U.S. FDA, and USDHHS India Health Attaché at the U.S. Embassy in New Delhi.
Ramya Tejasimha Karur is Bangalore-based research professional with Masters (MSc) in Biotechnology and Graduation in Microbiology from Bangalore University with post-graduate certification in Public Health nutrition. She has worked on various research projects ranging from Breast and Nasopharyngeal Cancer genetics, Asthma and COPD genetics, Genetic toxicology, and Public Health Nutrition, to Maternal and Child Health, and Epidemiology. She was also involved in public health research projects during her work at the Institute of Health Management Research Bangalore, India (IIHMR); Unilever, King’s College, London; and Southampton General Hospital UK.
Her interest lies in neonatal research and aspires to do a Ph.D. in studying the risk factors associated with congenital deaths due to rare diseases.
As a Project Associate at IndoUSrare, Ramya hopes to help patients in India gain access to life-saving therapies through research, outreach, and advocacy efforts and to drive policy changes in favor of rare disease patients in India.
Nisha Venugopal is Program Manager at IndoUSrare. She has a PhD in Life Sciences from the CSIR – Centre for Cellular and Molecular Biology (CCMB), India, where she studied the role of the primary cilium in adult skeletal muscle stem cells. Her postdoctoral research at the Institute for Bioinformatics and Applied Biotechnology, India involved a detailed analysis of global clinical trial registries. Nisha hopes to use her experience in the cell biology of rare diseases and policy research to support and help patients gain access to life saving therapies through research, outreach and advocacy efforts to drive policy changes in favor of rare disease patients in India.
Dr. Stephen Groft is a Senior Advisor to the Director, NCATS at NIH. He helped establish the Office of Orphan Products Development at FDA in 1982, a rare diseases initiative at NIH in 1989, Director of the Office of Rare Diseases Research (ORDR) from 1993-2014. Activities included the development of the Genetic and Rare Diseases Information Center (GARD), IRDiRC, ICORD, the RDCRN, assistance with the Undiagnosed Diseases Program and expanding the ICD XI to include more rare diseases.
His major focus has been on stimulating research with rare diseases and orphan products and developing information for patients, families, health care providers, research investigators, the biopharmaceutical industry, and the public. The emphasis included information about rare diseases, active and completed clinical trials, ongoing research, and patient advocacy groups. He has authored 85 journal articles, book chapters and the co-editor of 2 books on Rare Diseases Epidemiology. He has also made more than 320 presentations on rare diseases.
Dr. Reena Kartha is an Associate Professor in the Department of Experimental and Clinical Pharmacology and Associate Director of Translational Pharmacology in the Center for Orphan Drug Research (CODR), College of Pharmacy, University of Minnesota. She has a Masters in Biotechnology from Tamil Nadu Agricultural University, India, and a PhD in Cellular and Molecular Biology from the Indian Institute of Science, Bangalore.
Her research focuses on understanding the pathophysiological role of oxidative stress and inflammation in rare inherited metabolic disorders as well as the pharmacology of agents that target these molecular pathways. She has mentored and supervised undergraduate, graduate and pharmacy students conducting directed research and direct the CODR research seminars during the school year. She completed a rare disease clinical research training program supported by the NIH Rare Disease Clinical Research Network and was the recipient of the 2017 University of Minnesota Clinical and Translational Sciences Institute Outstanding Junior Mentor of the year award. Her career goal is to help patients with rare diseases by developing affordable and accessible therapies, train the next generation of healthcare providers and increase awareness about rare diseases
Ravi Mistry is a leader, entrepreneur, angel investor, educator, and mentor. His professional career has spanned several industries, including life sciences, technology, non-profits, and academics, in various capacities as Founder, Board Chairman/Director, President, Vice President, Sr. Director, Manager, and Adjunct Professor.
Currently, Ravi serves as Funds Management and Operations Officer of Impact India Investment Partners, LLC (3iPartners), a Silicon Valley based fund investing in early-stage impact-oriented startups in India with focus on AgriTech, HealthTech, EduTech, and Livelihoods. Ravi served as Vice President of Business Development & Operations at MedTree LLC, a Silicon Valley based life sciences start-up. Prior to MedTree, Ravi served as Founding Executive Director of Indian Institute of Technology Gandhinagar (IIT GN) Foundation. Ravi was member of founding team of Virident Systems, a Silicon Valley based technology startup that was acquired by HGST/Western Digital in 2013. Prior to Virident, he held management positions in the life sciences industry at DiscoveRx and BioImagene.
Ravi is an active angel investor and member of the TiE Angel Group Silicon Valley and TiE Global Angels. He is Former President and Board Member of EPPIC Global Network, an all-volunteer life science professional organization in Silicon Valley/Bay Area. He has been active with other Bay Area biopharma organizations such as California Life Sciences Association (CLSA – previously known as BayBio), a life sciences organization representing California bio industry, Chinese Bioscience Association (CBA), and Chinese-American Bio/Pharmaceutical Society (CABS). Ravi also advises and mentors IIT GN alumni both in India and in the US.
He is Gold Medalist from Gujarat University, India from where he holds BE degree in Civil Engineering. He holds Master’s degree in Structural Engineering from Stanford University, and an MBA in Finance from CSUEB.
Rajesh Gooty is an Executive VP of a Professional IT Services Firm Serving Both Public & Private Sectors, Headquartered in Washington DC. His seasoned expertise in Enterprise strategic planning, management & professional consulting led to several successful business transformation, procurement transformation, operational process improvement, organization design, operating model design, business case development, strategic sourcing & eProcurement change initiatives resulting in substantial operational efficiencies and cost-saving benefits for corporate clients in diverse verticals of Health Care, Pharmaceuticals, Financial Services, Government, Insurance, Hi-Tech, Oil and Gas, Entertainment and Manufacturing.
An Accomplished Business Partner, Forceful influencer, Strategic Thinker who thrives on tough challenges & translates visions and strategies into Actionable Deliverables, Value Added, and Result Oriented goals. Stays abreast of evolving business, leadership & human resource trends to gain a competitive edge. Effectively identifies compliance issues for business risk mitigation. As a Business & Technology Professional, Focused in Sales & Staffing Strategies, building Customer relationships, leveraging the knowledge of Client, Culture, and Business, facilitate change, and improve employee performance from fast-paced start-ups to Large Reputed organizations in both Public & Private Sectors of Diverse Verticals. He organizes & synthesizes data for effective business analyses, readily gains trust & credibility within varied, diverse levels in an organization, Forges Partners effectively with all levels of management, upon identifying & leveraging Synergies. With An Extensive knowledge of procedures for business transactions within the Federal Government & State Level Agencies.
A Driven Senior Leader With two Decades of Demonstrated Success, Seasoned Expertise & Exceptional Competence in Sales, Strategy & Talent Acquisition. Innovative, Resourceful, Skillful at executing strategy & bringing forth a commitment from Team Members to Achieve Excellence. The Realm of Seasoned experience has evolved from Government Contract Management (Onsite Project Management / Proposal Writing) to Corporate Division / Service Offering Management (Program Management, Proposal Management) Translating To A Strategic Business Development Executive that excels at Capture & Change Management executing, supporting full life cycle Multi-Million Revenues. He established a successful track record of designing & delivering large cross functional business transformation initiatives from strategy through execution while building strong collaborative team environments.
Dr. Akkaraju V. Sarma is a family medicine doctor in Bensalem, Pennsylvania. He received his medical degree from Universidad Autonoma de Ciudad Juarez and has been in practice for more than 20 years.
Life long commitment to help needy and enhanced my abilities being a physicians. Likewise, help structuring health sustaining programs like National Marrow Drive Registries, American Red Cross Blood Drives and similar activities.
Nara Govindarajan is a Techie & a Leader in Software Development. He currently works in a leadership role at UWorld LLC in Texas. He is a volunteer for numerous non-profit organizations and charitable causes.
Nara holds a Master’s degree in Computer Science from Virginia Tech. He did his Bachelor’s degree in Computer Science and Engineering from PSG College of Technology, Bharathiyar University, Coimbatore, India
Ms. Juhi Naithani is Principal at bGlobal Consulting and provides support to organizations ready to Go Global. She has public and private sector experience in business strategy across a range of disciplines, with her first job in India in B2B industrial sales, and subsequent experience in the United States in consulting, academia and economic development.
Prior to founding bGlobal Consulting, Juhi was Assistant Director of International Business Investment at the Fairfax County Economic Development Authority (FCEDA), Virginia, USA, where she managed the India office of FCEDA, implementing new outreach strategies and events to bring emerging technology companies to the United States.
Juhi has served as visiting faculty of marketing and international business at numerous universities over the last 20 years, fostering higher education at the undergraduate and graduate levels.
A strong proponent of entrepreneurship, Juhi was a member of the Board of Directors of the TiE (The Indus Entrepreneur) DC chapter for four years through 2022.
Juhi has an undergraduate degree from Delhi University, India and an MBA from Quinnipiac University, Connecticut, USA
Dr. Harsha Rajasimha has authored 15 high impact journal publications, 2 book chapters, and inventor on 2 patents. He is often a keynote speaker, convener of #DigitalOMICS meetup around the Capital Beltway and part of numerous conference organizing committees. Dr. Rajasimha has received numerous Awards for his work in genomics, precision medicine and rare diseases including a travel award to join the NGO Committee on Rare Diseases at the United Nations headquarters, New York City in Feb 2019, Lead India Foundation 2020 Award for leadership and outstanding achievement in rare diseases, NTT DATA Healthcare Life Sciences Silver award for outstanding achievement 2017, Sanofi Genzyme patient advocacy leadership award 2016, Bioscience company of the year 2014, NEI director’s innovation award 2012 for building their genomics cyber infrastructure. Harsha earned his M.S. in Computer Science (2004) and Ph.D. in Genetics, Bioinformatics and Computational Biology (2007) from Virginia Tech.
Driven by a personal cause in 2012, Dr. Rajasimha decided to apply his years of post-doctoral clinical genomics data research experience at National Institutes of Health and industry product development experience to develop products and solutions to help accelerate clinical research.
Frank J. Sasinowski, M.S., M.P.H., J.D., has helped secure FDA approval for hundreds of new drugs, including more than 100 new molecular entities, often for serious and rare diseases. Frank has been involved in more than 50% of all the drugs FDA approved by way of the accelerated approval pathway (outside of cancer). Frank is also involved in many cell and gene therapies and aided significantly on the first approved systemic gene therapy, Zolgensma, as well as the other “curative” gene therapy Lenmeldy. Finally, Frank has helped spearhead the implementation of the alternative effectiveness statutory standard including through his involvement in approvals of Duvyzat, Miplyffa, Skyclarys, Filsuvez and Vyjuvek.
Frank joined FDA in 1983 as regulatory counsel in the Center for Drugs and Biologics, where he was key to implementing both the 1983 Orphan Drug law and the 1984 Hatch-Waxman law. In 1987, he left the FDA as Deputy Director of health policy in Commissioner’s office and joined Hyman, Phelps & McNamara.
In its March 2012 issue, the Drug Information Journal published Frank’s seminal analysis on therapies for rare disorders: “Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders.” Other papers of his on Accelerated Approval and Orphan Drugs are regularly cited by FDA, industry and academia as well. Thanks to his extensive FDA experience, both from within the Agency & from a sponsor’s perspective, as well as his passionate advocacy for integrating the voice of the patient in developing medicines, Frank possesses a nuanced & deep understanding of the drug regulatory process.
Since 2014, Frank has been Adjunct Professor of Neurology at U. of Rochester School of Medicine. His work has been widely recognized. (e.g., Frank was asked by both political parties to testify at the May 2014 inaugural congressional hearing on the 21st Century Cures law). In May 2013, the National Organization for Rare Disorders (NORD) awarded Frank its first ever Lifetime Achievement Award. In 2000, Frank was elected to NORD’s Board of Directors, where he served as Chair and as Vice Chair & was on its Board until 2016. In 2017 Frank joined the Board of the Everylife Foundation for Rare Diseases where he currently is its Vice Chair. In October 2012, President Obama recognized Frank’s contributions to the President’s Council of Advisors on Science and Technology (PCAST) report, “Propelling Innovation in Drug Discovery, Development and Evaluation.” Frank is a founding Board member of the IndoUSrare patient organization, representing those in India and in the US with rare conditions. Frank has also served on the Board of Directors of the United States Pharmacopeia (USP) & the ARM Foundation and has served on the board of several biotechnology companies.
Dr. Anish Bhatnagar is currently the Chief Executive Officer of Soleno Therapeutics (NASDAQ: SLNO). Soleno is dedicated to the development of treatment for rare diseases. Their lead program is for DCCR, a once-a-day tablet, for the treatment of Prader Willi Syndrome. Dr. Bhatnagar is a physician with over 20 years of experience in the biopharmaceutical, medical device, and diagnostic industries. His experience spans the development of biologics, drugs, drug-device combinations, and diagnostic as well as therapeutic medical devices. His prior experience includes working at Coulter Pharmaceuticals, Inc. from 1998 to 2000 and Titan Pharmaceuticals, Inc., and Capnia Inc. He is the author of several peer-reviewed publications, abstracts, and book chapters. He obtained his medical degree at SMS Medical College in Jaipur, India, and completed his Residency and Fellowship training in the U.S. at various institutions, including Georgetown University Hospital and the University of Pennsylvania.
Dr. Anish Bhatnagar is currently the Chief Executive Officer of Soleno Therapeutics (NASDAQ: SLNO). Soleno is dedicated to the development of treatment for rare diseases. Their lead program is for DCCR, a once-a-day tablet, for the treatment of Prader Willi Syndrome. Dr. Bhatnagar is a physician with over 20 years of experience in the biopharmaceutical, medical device, and diagnostic industries. His experience spans the development of biologics, drugs, drug-device combinations, and diagnostic as well as therapeutic medical devices. His prior experience includes working at Coulter Pharmaceuticals, Inc. from 1998 to 2000 and Titan Pharmaceuticals, Inc., and Capnia Inc. He is the author of several peer-reviewed publications, abstracts, and book chapters. He obtained his medical degree at SMS Medical College in Jaipur, India, and completed his Residency and Fellowship training in the U.S. at various institutions, including Georgetown University Hospital and the University of Pennsylvania.