Abbey Meyers Khushi Bridging RARE Awards Ceremony

About the Award

The Abbey Meyers Khushi Bridging RARE Award honors the lifetime of dedicated work and accomplishments of two stalwarts who have done groundbreaking work to foster cross-border collaborations in the field of rare genetic and undiagnosed diseases between the US, India, and globally. By honoring these legends who have advanced research in rare and undiagnosed diseases, the Indo US Organization for Rare Diseases together with the Bridging RARE planning committee seeks their vast wisdom and guidance to carry forward the mission of catalyzing cross-border collaborations for patient-focused research and the next generation of affordable orphan drug development.

About Dr. Abbey Meyers:

Dr. Abbey Meyers is a living legend who was the force behind the enactment of the US FDA Orphan Drug Act of 1983, which has had immense an impact in the US and kicked off a global crusade.

She is the founder and past President of the National Organization for Rare Disorders (NORD), a coalition of national voluntary health agencies and a clearinghouse for information about little known illnesses. Dr. Meyers currently holds the honorary title of President Emeritus of NORD. She has also served as Honorary President of the European Organization for Rare Disorders (EURORDIS) before she retired from her busy life as a leader of the rare disease movement in 2009.

About Baby Khushi

Baby Khushi was diagnosed at birth in 2012 with Trisomy 18 or Edwards syndrome. Her parents were told “she is not a viable baby and that she was not compatible with life” by the neonatologist. Baby Khushi lived for 4 days with the support of life support ventilators in the NICU. This shifted the career trajectory of Khushi’s father, Harsha Karur Rajasimha, PhD. The NIH-trained genomics data scientist turned into a serial entrepreneur co-founding the national umbrella organization for rare diseases in India in 2013, led the publication of the first peer-reviewed journal article reviewing the challenges and opportunities for rare diseases in India (Rajasimha et al., 2014). Harsha later founded the digital platform company, Jeeva Clinical Trials, Inc in Virginia to make clinical trials more efficient and universally accessible. In 2019, Harsha founded the Indo US Organization for Rare Diseases recognizing the unmet need to engage the Indian subcontinent with the patient-led orphan drug revolution in the US.

“Khushi” means Joy in multiple Indian languages. Baby Khushi’s legacy lives on as she continues to inspire her parents and the Karur family to work towards making it better for all persons affected by rare and undiagnosed diseases.

About Baby Khushi:

Meet the Awardees

Rare diseases, which encompass a wide range of conditions affecting small populations, often face significant challenges in terms of treatment options. However, advances in immunotherapies have led to remarkable improvements in clinical outcomes for several of these diseases. One of the most promising breakthroughs in this field is CAR T-cell therapy, which has the potential to offer life-saving interventions for various rare conditions. Despite the transformative potential of these therapies, their high cost has been a global challenge, limiting access for many patients in need.

CAR T-cell therapies have already transformed outcomes for patients with rare hematological diseases, demonstrating the potential for long-term remission. Expanding the availability of these therapies could be a game-changer for the treatment of rare diseases worldwide, including rare autoimmune conditions, offering hope for patients who have long struggled with limited or ineffective treatment options.

A breakthrough in this area comes from NexCAR19, a CAR T-cell therapy developed by Dr. Rahul Purwar and his team at IIT Bombay, in collaboration with Dr. Hasmukh Jain from Tata Memorial Hospital. After successfully leading a multi-center phase I/II clinical trial, the therapy received approval from India's Central Drugs Standard Control Organization (CDSCO). This has drastically reduced the treatment cost to approximately ₹30 lakh (~35,000 USD) per patient, with ongoing efforts to decrease costs further and make the therapy more accessible to a wider population

Rahul Purwar, PhD

Dr. Rahul Purwar is a Professor at the Department of Biosciences and Bioengineering at IIT Bombay. He holds a PhD in Molecular Medicine from Hannover Medical School, Germany. Dr. Rahul’s professional journey spans various prestigious institutions and roles. He started his post-doctoral fellowship at Harvard Medical School, Boston, USA. After his fellowship, he joined ImmunoGen, Inc. (Waltham, MA, USA), as a scientist before returning to India as a faculty member at IIT Bombay.

Based on his research at IIT Bombay, Dr Purwar founded ImmunoACT in 2018, first cell & gene therapy company in India. In this role, he drives strategic initiatives through accelerated process & clinical development and manufacturing, which has not only led to the successful market authorization of the country’s first CAR-T cell therapy, but also one that is fully Indigenous to India. This is the first of many planned milestones in democratizing access to advanced cell & gene therapies in India, his fundamental vision and founding principle of ImmunoACT.

Dr. Purwar’s research work is well-recognized in the scientific community, with numerous publications to his credit in esteemed journals. His contributions to the field of Biosciences & Bioengineering are commendable and his work continues to inspire many in the scientific community.

Hasmukh Jain, MD

Dr. Hasmukh Jain is a hemato-oncologist, currently working as a professor at Tata memorial centre, Mumbai. He completed his MD medicine from KMC Mangalore and DM medical oncology from the prestigious Tata memorial centre. His research work is focussed on ALL, Hodgkin’s lymphoma, artificial intelligence and cell therapy. His research has been published in national and international journals.

He was the lead PI on the pivotal trials that led to the approval of CAR-T cell therapy in India and is conducting pioneering research to study CAR-T cell therapy in the frontline and improve access.

He has received several awards in his career including the best post graduate student in internal medicine from manipal university, awards on CAR-T cell therapy research from haematology cancer consortium, indian society of medical and paediatric oncology and European group for blood and marrow transplantation.

Frank J. Sasinowski, MS, MPH, JD

Frank J. Sasinowski, has played a pivotal role in securing FDA approval for hundreds of new drugs, including over 100 new molecular entities, many of which target serious and rare diseases. Notably, he has contributed to over half of all non-oncology drugs approved through the FDA's accelerated approval pathway and has been instrumental in numerous cell and gene therapies, including the first systemic gene therapy, Zolgensma, and Lenmeldy. His expertise extends to championing the alternative effectiveness statutory standard, as seen in approvals of therapies like Duvyzat, Miplyffa, Skyclarys, Filsuvez, and Vyjuvek. Frank began his career at the FDA in 1983 as regulatory counsel in the Center for Drugs and Biologics, where he contributed to the implementation of the 1983 Orphan Drug Act and the 1984 Hatch-Waxman Act.

His influential work includes a seminal 2012 Drug Information Journal analysis titled “Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders,” which continues to be widely cited across the FDA, industry, and academia. Frank’s extensive knowledge of FDA processes, coupled with his advocacy for patient-centered drug development, grants him a unique perspective on regulatory matters. Since 2014, he has served as an Adjunct Professor of Neurology at the University of Rochester School of Medicine and has contributed his insights to numerous boards, including the National Organization for Rare Disorders (NORD) and the EveryLife Foundation for Rare Diseases. His achievements have been recognized with numerous awards, including NORD’s inaugural Lifetime Achievement Award, the FDA Award of Merit, and the 2021 Pope Francis Pontifical Hero Award for Inspiration.

Frank's academic journey includes a B.S. in Biological Sciences and Genetics from Cornell University, an M.S. in Nutritional Sciences, and an M.P.H. from the University of California at Berkeley, followed by a J.D. from Georgetown University Law Center. His influence and dedication are also reflected through his work with the IndoUSrare patient organization and on the boards of the United States Pharmacopeia (USP) and the ARM Foundation.