Cure GPX4 Research Conference
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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 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.
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
Explain the objective (5mins)
Context Talk (10mins + 5mins Q&A) to orient participants by covering enough topics necessary for brainstorming
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”