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”