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Homepage Cure GPX4 Research Conference
Loading Events
  • « Global Genes Data DIY
  • IndoUSrare Open house for Indian Patient Advocacy Groups »

Cure GPX4 Research Conference

July 14, 2021
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Overview

We are excited to invite you to the Cure GPX4 Research Conference on March19th. This one day conference is aimed at bringing a diverse group of researchers, clinicians and industry partners who are working on finding a cure for the disease caused by mutations in GPX4. The goal of the conference is to share updates from different areas of GPX4 research, discuss planned activities and facilitate dynamic exchange of ideas to build a cure.

Goal

Goal is to create a complete roadmap for therapy development by the end of the day. The roadmap will include identifying experiments necessary to understand disease, identifying small molecule drugs, and exploring the use of emerging technologies like gene therapy, ASOs etc to treat this condition. To accomplish this goal, we will have workshop-style free form discussions spread across three sessions.

Each session starts with a 10min “context” talk to get participants up-to-speed with the science underlying GPX4 with regard to its role in health and disease. This can provide the necessary background to all attendees to successfully participate and brainstorm. This will be followed by a ~40min brainstorming session. With the emphasis on informal freeform discussions, brainstorming sessions is where we expect everyone to contribute their expertise to help shape the roadmap.

 The agenda, brainstorming session structure, and other activities are tailored to run a very collaborative online meeting respecting constraints such as multiple time zones, technology issues etc. We have dedicated time for tech-checks, ample breaks, dedicated note taking person, and a dedicated tech support person to ensure participants can focus on discussions.


Agenda

07:30 – 07:45 am Tech Check

Introduction & Keynote (08.00 to  09.00 am)

07.45 – 08.15 am Introductions and Welcome by Sanath Kumar Ramesh

08.15 – 08.45 am Keynote Speaker: Dr. David Fajgenbaum “Chasing my cure”

08.45 – 09.00 am Clinical Presentation of SSMD disease and Patient Needs from therapy by Dr. Kristen Wigby

09.00 – 9.30 am Break

Session 1: Basic Science (09.30 to  10.30 am)

09:30 – 09:35 am Explain the objective of the session by Sanath Kumar Ramesh

09.35 – 09.50 am “Context Talk” on GPX4 science by Dr. Brent Stockwell

09.50 – 10.30 am Workshop & Brainstorming: Basic Science moderated by Dr. Brent Stockwell & Dr. Elias Arner

10.30 – 10.45 am Break

Session 2: Small Molecule Drug Development (10.45 to  11.45 am)

10.45 – 10:50 am  Explain the objective of the session by Sanath Kumar Ramesh

10.50 – 11.05 am “Context Talk” GPX4 Mouse Models by Dr. Qitao Ran

11.05 – 11.45 am Workshop & Brainstorming: Small Molecule Drug Development moderated by Dr. Ethan Perlstein & Dr. Matt Hall

11.45 – 12.15 pm Break

Session 3: Emerging Therapeutic Approaches (12.15 to 01.15 pm)

12.15 – 12:20 pm  Explain the objective of the session by Sanath Kumar Ramesh

12.20 – 12:35 pm  “Context Talk” on building gene therapy treatments by Dr. Miguel Esteves

12.35 – 01:15 pm Workshop & Brainstorming: Emerging therapeutic approaches moderated by Dr. Plavi Mittal & Dr. Miguel Esteves

01:15 – 01.45 pm Prioritize Roadmap & Closing Remarks


Session Structure

  • Explain the objective (5mins)

  • Context Talk (10mins + 5mins Q&A) to orient participants by covering enough topics necessary for brainstorming

  • Brainstorm (40min)

    • Moderators will explain the objective of the session and start discussion with the first open ended question.

    • Discussions are freeform like a small “team meeting”. Moderators’ role is to guide discussion to meet the objective.

    • Participants can ask questions on chat or audio.

    • If new information is identified or a decision made, add it to “Roadmap Chart”

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Copyright ©2022 Indo US Organization for Rare Diseases. All rights reserved. IndoUSrare is a registered 501(c)(3) charity organization.
MS. KANAN LATHI

Kanan Lathia is an Ambassador and scientific consultant at Indousrare.She has spent 20+ years as a research scientist focusing on neurodegenerative diseases, cancer, and HIV. She has also worked as a project manager supporting teams in building brain maps of the first 1000 days of babies. Kanan’s passion for rare diseases is driven by a personal cause. Her work with Dup15q Alliance strengthened her passion for giving back to rare disease families. Kanan hopes to scale the organization’s efforts by securing funding and building a patient alliance membership program. Kanan loves yoga and spending time between the US and India.

 Dr. Krishna Ramaswamy

Dr. Krishna Ramaswamy is a life science professional with experience in research, management consulting, and business development in academia and industry. She has a Ph.D. in Biological Sciences from University at Buffalo, SUNY, and pursued postdoctoral research in cell biology from the University of Southern California, Los Angeles. She has worked as a consultant for the biotech/pharma industry and worked with Dr. Harsha and ORDI in the past for about 2 years. Dr. Krishna currently resides in Hyderabad India and has a keen interest in the rare diseases space and would like to bring awareness about the various diseases, diagnoses and treatments that are available and those in the pipeline. She first began working in the rare disease space during her postdoctoral training and has worked on peroxisome biogenesis disorders under the guidance of Dr. Joe Hacia at USC.

Ms. Neeraja

Neeraja is in her junior year of high school, studying at a School in Gurgaon, India. She has completed all 8 grades in a set of vocal examinations conducted by Trinity College as the captain of the Student Council in her school, currently serving the post of House Captain. Neeraja aims to find solutions to the problems associated with the fields of healthcare, technology, and sustainable development. She is passionate about teenage welfare and has founded a youth-centric organization called ‘Discover Yourself’ through which she aims to inspire, and collaborate with young people across the world and wants to bring awareness regarding various teenage and adolescent issues

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