New regulations and shifts to the economy have the potential to impact industry appetites and risk tolerances for investing in therapies that serve smaller populations. Concurrently, some rare disease product developers may be facing increasing financial pressure and others may consider adjustments to company pipeline investment and research strategies as a result of these evolving trends. These shifts are also occurring while scientific advancements are increasing the potential of personalized medicine to enhance treatment effectiveness and reduce side-effects, but further subdivide the population that could benefit from the treatment. However, there is still significant progress being made in the rare disease product development and investing spaces, with many companies adapting to these changes and driving research and clinical efforts forward. This session will explore current and forward-looking strategies for pursuing and investing in rare disease research and therapeutic options that are evolving in light of these trends that are aiming to achieve regulatory, access, and financial success.

Moderator

Annie Kennedy

Chief of Policy, Advocacy and Patient Engagement, EveryLife Foundation for Rare Diseases

Speakers

Sam Blackman

Founder and Head of Research and Development, Day One Biopharmaceuticals, Inc.

Nick France

Vice President, Global Therapeutic Area Head for Genomic Medicines and Neurology, Alexion, AstraZeneca Rare Disease

Maha Katabi

General Partner, Sofinnova Investments

Ethan Perlstein

Founder and CEO, Perlara PBC; Co-Founder and CEO, Maggie's Pearl LLC