Agenda

5:30 pm - 10:00 pm EST (November 2, Sunday)
4:30 am - 8:30 am IST (November 3, Monday)

Emcees for the evening: Rohan Honganoor, Sirichandana Yakkala

  • Invocation song by Ms Alisha Thomas, Vocalist/ Voice Coach
  • A Warm Welcome by Hosts and Partners 
    • Amy Adams, PhD, Institute for Biohealth Innovation, George Mason University
    • Harsha Rajasimha, PhD, IndoUSrare
  • A message from Frank Sasinowski, JD, MPH, IndoUSrare and HPM
  • Entertainment - Performances
    • Ms Alisha Thomas, Vocalist/Voice Coach
  • Patient Keynote Address by Pesh Patel, A Kidney Life
  • Keynote Address by Gary A. Puckrein, PhD, National Minority Quality Forum
  • Entertainment - Performances 
    • Dance performance by Rishitha Voleti
  • Invited Talk by Caitlyn Barrett, PhD, Milken Institute
  • Patient Lived Experience: Mrs and Mr. Mohamed Duklef, Living with Epidermolysis Bullosa
  • Keynote Address from AMA: Dr. Bobby Mukkamala, MD, Board of Directors, American Medical Association
    • Introduction to Speaker: Dr. Krishna Madiraju, Member FSMB Education Committee, Member Virginia Board of Medicine, & Century Pediatrics Inc.
  • Family Experience: Nandita Roy and Saikat Sarkar, SSPE family from India
    • Introduction to the Speakers by Lisa Shea, Immigrants First Law Firm
  • Entertainment - Performances
    • Dance performance by the Voleti Sisters
  • IndoUSrare Board Members take a bow
    • Nara Govindarajan, MS, IndoUSrare and UWorld
    • Reena Kartha, MS, PhD, IndoUSrare and University of Minnesota
    • Juhi Naithani, MBA, IndoUSrare and bGlobal Consulting
    • Mathew Thomas, MBBS, IndoUSrare and MTT Consulting (Retired USFDA Country Director for the India office)
    • Anish Bhatnagar, MD, Soleno Therapeutics
    • Frank Sasinowski, JD, MPH, IndoUSrare and HPM
    • Harsha Rajasimha, PhD, IndoUSrare and Jeeva Clinical Trials
  • Entertainment -  Performances
    •  Vocal performance - Sasanka Sreedevi Naresh
  • Abbey Meyers Khushi Bridging RARE Award Ceremony
    Guest of Honor: Suhas Subramanyam, Hon US Representative, Virginia District 10
    • Special message from Frank Sasinowski, JD, MPH, a legend in rare disease
    • Talk by Dr. ⁠Harsha Rajsimha
    • Remarks by Guest of Honor
    • Award presentation to Honorees 2025:
      • Madhulika Kabra, MD, ICMR, India
      • Priya Kishnani, MD, Duke University Medical Center
      • Pramod K. Mistry, MD, PhD, FRCP, Yale University Medical Center
      • Rajesh Gooty, Community Impact Awardee
    • Group Photo with the Guest of Honor
  • Remarks: Henry Eickelberg, Treasurer, Loudoun County, VA

Who is a patient advocate?

Goal setting for patient advocates

What has patient advocacy achieved?

Role of patient advocacy in drug development

How to be an effective communicator as a patient or caregiver?

Involving Industry and Academics in Drug Development

Jean Campbell, Professional Patient Advocates in Life Sciences (PPALS)

Dr. Ratna Devi, DakshamA Health

Mohua Chakraborty Choudhury, PhD, MPH, IISc

David Rintell, Ed.D, BridgeBio

Nita Patel, BSN, Ex-Amicus Therapeutics

Nahid Zaman, Astrazeneca India

Dr. B.S. Charan, DGHS

Occupational and physical therapies relevant to rare diseases

Self-care, managing emotions, and guilt

Cristol O’Loughlin, RaregiversTM

Vaishali Pai, Tamahar Trust

Team Premas Life Sciences

Debjani Saha, Premas Life Sciences

Dr. Rahul Ramekar, Premas Life Sciences

Lakshman Teja, Premas Life Sciences

Dr. Vikas Sharma, Premas Life Sciences

Priyanka Singh, Premas Life Sciences

Ratna Dua Puri,MD, DM, SIAMG

Neerja Gupta, MD, DM, AIIMS, Delhi

Co-Chairs

Priyankana Mukherjee, PhD, IKP Eden

James A. Levine, MD PhD, Foundation Ipsen

  1. Art Exhibit - in partnership with Beyond the Diagnosis
  2. Prayer and Invocations
  3. Inauguration and Lamp Lighting
  4. Abbey Meyers Khushi Bridging RARE Awards Ceremony
  5. Keynotes
  6. Patient Community Voices
  7. Entertainment
  8. Dinner and Networking

Chief Guest:

Dr. Chandra Sekhar Pemmasani - Minister of State for Rural Development and Communication

Keynote - Abbey Meyers Khushi Bridging RARE Award Honorees:

Dr. Rahul Purwar, Dr. Hasmukh Jain

Frank Sasinowski, JD, MPH

Felicitation
Dr. Abbey Meyers, Founder, NORD

Patient community voices:

  1. Gautam Dongre, NASCO

  2. Shikha Metharamani PWSA - India

  3. Sunil Ladwa, FOP India

Performances:

  1. Tabla recital - Saksham Mishra

  2. BharataNatyam performance - Mansa Gautam and Raunika Negi

  3. Vocal performances - Chetan Chawla and Ms Ridisha

Close of Day 1

Conference Day 1 - Monday, 3 November 2025

Harsha K Rajasimha, MS, PhD, Summit Chairperson

Amy Adams, PhD, Institute for Biohealth Innovation, George Mason University

Victoria Gamerman, PhD, RWD Insights

Rakesh K. Mishra, PhD, Tata Institute for Genetics and Society (TIGS)

  • Define inequities
  • What rare diseases are disproportionately prevalent in Latin America, Middle East, South Asia, and Pakistan
  • What is the value exchange with these regions?
  • What is the hesitancy to inclusivity?
  • Role of patient registries, data digitization
  • Solution to achieve global equity

Moderator: Harsha K Rajasimha, PhD, IndoUSrare

Moderator:
Sara Tylosky, MBA, Farmacon Global

Tina Aswani-Omprakash, MPH, South Asian IBD Alliance

Mohamed Duklef, RPh, MSc, Center for Orphan Drug Research (CODR), UMN

Prof. Huma Cheema, MRCP, DPGN, University of Child Health, Lahore, Pakistan

Networking
10:00 am to 10:15 am ET
8:30 pm to 8:45 pm IST

Screening and Diagnosis

  • Scenario in India, US, Nepal, and Pakistan
  • Emerging and affordable technologies

Moderator: Reena Kartha, MS, PhD, IndoUSrare and University of Minnesota

Plenary keynote: Madhulika Kabra, MD, ICMR (AIIMS, New Delhi)

Speakers:
Nishitha Pillai, MD, Department of Pediatrics, University of Minnesota

Tshaka Cunningham, PhD, Athari Bio

Vaadiyar Madhusudhan, PhD, LifeArc

Bijaya Mishra, MD, Rare Disease Society, Nepal, and BPKIHS, Nepal

Bushra Afroze, MBBS, DCH, MCPS, FCPS, The Aga Khan University Hospital

  • Management of ultra-rare and novel diseases
  • Emerging novel therapies for rare diseases
  • Drug repurposing for accessible therapies
  • Prioritizing Innovation, Accessibility, and Affordability
  • Success stories and most promising opportunities

Moderator:
Donney John, PharmD, NOVA ScriptsCentral

Speakers:

Neerja Gupta, MD, DM, AIIMS, New Delhi

Narendra Chirmule, PhD, SymphonyTech Biologics

Kuntal Sen, MD, Children’s National Hospital, Washington, DC

Lunch
12:00 pm to 12:55 pm EST
10:30 pm to 11:30 pm IST

  • Role of patient advocacy in orphan drug development
  • Inclusion of Trial Sites in India and LMICs
  • Manufacturing
  • Commercial launch & Pricing of orphan drugs in India and LMICs
  • Priorities for US-India Partnerships

Moderator: Jill Yersak, PhD, Soleno Therapeutics

Speakers: Karla MacDonald, Entrada Therapeutics

Sanket Shah, MBBS, PhD, Jazz Pharma

Parva Purohit, PhD, Amneal Pharmaceuticals

Sonit Handa, MD, MS, MBA, Biovance Consulting LLC

  • Grand challenges in rare diseases for AI and Tech
  • Context of patient journey
  • Global federated learning
  • 6E principles of AI
  • Hype vs. Reality
  • Benefits of AI vs. HI
  • How can industry effectively adopt AI
  • Regulatory clarity or blur
  • Priorities for US-India partnership

Session Sponsor: Venkatesh Krishnaiah, CloudThrottle

Moderator :Harsha Rajasimha, PhD, Jeeva Clinical Trials

Panelists:

  • Christopher P. Boone, PhD, FACHE, FHIMSS, Oracle Corporation
  • Mandeep Kaur, MD, MS, Valion Consulting
  • Himashu Kansara, TheGoodAI

Networking, Exhibits, and Posters
3:00 pm – 3:30 pm EST
1:30 am - 2:00 am IST (+1)

Indo US RARE Working Group Discussion

Developing a Blueprint for US-India Strategic Cooperation for Rare Diseases Research and Orphan Drugs Development

WORKING GROUP 1: NBS, Screening, Diagnosis, Patient Registries, Referral Networks

WORKING GROUP 2: Strategy to Achieve Equitable Representation of Indian Diaspora in CRAACO, Clinical Trials

WORKING GROUP 3: Legislative, Regulatory, Financial Advocacy, the ASK: Indian and US Legislators, CDSCO, and FDA. E.g., ASHA program for Cancer Clinical Trials.

WORKING GROUP 4: BioSecure ACT and Inflation Reduction Act of USA and Food/Drugs/Cosmetics Act and NPRD of India - How India can play a greater role in affordable Innovation, R&D, and Manufacturing?

Action Items from Working Groups

Summaries to be published in Proceedings Article

BREAK OUT WORKING GROUPS with Whiteboarding

All participants have selected a working group to lead or contribute or participate.

Working Group Leaders

4:30 pm EST onwards
Biotech and Beer Networking Event with Local DMV Area BioHealth Community

Hosted By the Institute for BioHealth Innovation and VA BIO

Close of Day 1

Conference Day 2 - Tuesday, 4 November 2025

Reena Kartha, MS, PhD, IndoUSrare and UMN

 

  • Overview of IndoUSrare, our vision mission and impact
  • Learnings from 5 years of the IndoUSrare Patient Concierge
  • QnA Session
Speakers:

Nisha Venugopal, PhD

Ramya T Karur, MSc

Harsha Rajasimha, PhD

Speakers:

Mathew T Thomas, MBBS, IndoUSrare & MTT Consulting

Isaac Rodriguez-Chavez, PhD, MHSc, MSc, 4Biosolutions Consulting

Startup Pitches
Patient Stories

10:00 am to 10:30 am EST
8:30 pm to 9:00 pm IST

Introduction by Frank Sasinowski, JD, MPH, IndoUSrare and Hyman Phelps McNamara PC

Harsha K Rajasimha, IndoUSrare

John F. Crowley, Biotechnology Innovation Organization (BIO)

Networking Coffee
11:00 am – 11:15 am EST
9:30 pm to 9:45 pm IST

Moderator: Nara Govindarajan, MS, IndoUSrare and UWorld

Speakers :
Jamie Sullivan, MPH, EveryLife Foundation for Rare Diseases

Ratna Devi, MBBS, MBA, DakshamA Health

Session Sponsor: Ishan Anand, Esq, Immigrants First

  • Current state of underrepresentation and access
  • Case studies: Friedreich’s Ataxia, Ehlers Danlos
  • Role of Patient advocacy in Industry
  • Affordability and opportunities for Indo-US collaboration

Moderator: Jenifer Waldrop, MSHRD, Rare Disease Diversity Coalition (RDDC)

Panelists:

Prof. Lara Bloom, The Ehlers-Danlos Society

David Rintell, EdD, BridgeBio

Jennifer Farmer, MS, Friedreich's Ataxia Research Alliance

Sambit Mohanty, Entero Healthcare Solutions Ltd

Lunch
12:30 pm - 1:30 pm EST
11:00 pm (4 Nov) - 12.00 am IST (5 Nov)

Kapil Patel

Monica Dudley-Weldon, BS, MSL VA Commonwealth Health Research Board (CHRB) Appointee

  • Personal stories and perspectives
  • Current state of clinical trials in rare diseases
  • Unique challenges and barriers for rare disease clinical trials
  • Innovative trial designs
  • Patient access with decentralized tools
  • Voice of the patients, advocacy
  • Case studies
  • Solutions space to address inefficiency, inclusivity, and access

Plenary Keynote:
Harsha Rajasimha, PhD, IndoUSrare and Jeeva Clinical Trials

Moderator:
Isaac Rodriguez-Chavez, PhD, MHSc, MSc, 4Biosolutions Consulting

Panelists:
Hemang Maniar, Duke Clinical Research Institute (DCRI)

Gurdyal Kalsi, MD, MFPM (Hon), AskBio Consult

Del Smith, PhD, Acclinate

  • CNS perspective
  • Perspectives from across the globe
  • Session Sponsor: Entrada Therapeutics

Moderator: Terry Jo Bichell, PhD, MPH, COMBINED Brain

Plenary Keynote:

Prof Sheffali Gulati, MD, FRCPCH (UK), FAMS, FIAP, FIMSA, AIIMS New Delhi

Speakers :
Brian Tseng, MD, POLG Foundation

Nandita Roy & Saikat Sarkar, SSPE Family

Sunitha Malepati, CACNA1A Foundation

Networking Coffee
3:45 pm - 4:00 pm EST
2:15 am - 02:30 am IST (+1)

Pitches and Presentations

Pitching Companies

Apply here:
https://bit.ly/pitch4rare2025

Vote of Thanks

Harsha K Rajasimha, PhD

Summit Concludes