Invest in Cures San Diego 2023

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9:05am: Opening Keynote: Derisking Rare Disease

Derisking rare diseases is of utmost importance as an astounding number of 300 million individuals worldwide are burdened with these conditions, yet effective cures remain elusive. To combat this global healthcare crisis, it becomes imperative to seek sustainable solutions that not only alleviate the suffering of these patients but also make the most efficient use of capital to maximize their impact. By carefully deploying resources and investments, we can optimize the outcomes of research, development, and treatment efforts, ensuring that every dollar spent brings us closer to discovering effective therapies and minimizing the anguish endured by those affected. Dr. Levine will speak about his experience in the field over the last 35 years.

• James Levine, PhD, MD,  President Fondation Ipsen

Speaker Bio

 

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9:30am:  The Rise of Impact Investing — How Disease Foundations are Translating Science Into Cures

Impact investing in healthcare has been a growing trend over the last several years and for good reason.  A mission-driven approach to investing in startups has proven to be a successful method for advancing science beyond the lab and into the clinic. Funds affiliated with disease foundations often include backing from a vast network of experts, access to patient populations, a drive for results, and patient advocacy that transcends business objectives. Traditional drug development investors often neglect rare disease populations due to small market size and challenges in conducting clinical trials, proving this alternative approach to be particularly advantageous. In this session, learn from a panel of impact investors on what they look for in potential investments, how they syndicate with other investors, and what makes their approach to working with startups unique. 

• Deborah Smith, PhD, JD, Partner, Wilson Sonsini, Goodrich, & Rosati (moderator)
• Rusty Kelley, PhD, MBA, Managing Director, Retinal Degeneration Fund (Foundation Fighting Blindness)
• Dirk Landgraf, PhD, Vice President, Dementia Discovery Fund
• Teri Willey, MBA, Managing Director, Pathway to Cures (National Hemophilia Foundation)
• Russell Wyborski, PhD, Director, IBD Ventures Investments (Crohn’s and Colitis Foundation)

Speaker Bios

 

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10:25am:  Mission-Driven Funding – The Collaborative Approach of Venture Philanthropy

The driving force behind disease foundations is improving patient outcomes. The affiliated venture arms created by many of these foundations share the long-term commitment and dedication needed to support the mission of their parent organization. This patient-centric objective is reflected in the investment horizon and overall strategy of the fund and ultimately influences how these foundational funds support their portfolio companies. This session explores the collaborative approach of two disease foundation funds and their investments.

• Christina Waters, PhD, MBA, CEO RARE Science (moderator)
• Linda Martin, President, Mission Cure Capital
• Jessica Gibson, MBA, Ariel Precision Medicine
• Susan Hedstrom, Executive Director, Foundation For Prader-Willi Research
• Neil Cowen, PhD, MBA, Sr. Vice President of Drug Development, Soleno Therapeutics

Speaker Bios

 

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12:15pm:  ASO Technology – Hope for the N of 1 Populations

Hot off the press, entrepreneur, scientist and philanthropist, Stanley Crooke, PhD, will discuss his recent publication in Nature Biotechnology that explores ASO (Antisense Oligonucleotide) technology and its unique ability to treat ultra-rare patients.  He will also shed light on the  measures taken to ensure safe treatment of these micro-populations and address what future reforms are needed to continue to improve the medical community’s ability to support these patients.

• Stanley Crooke, PhD, Founder & CEO, n-Lorem

 

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12:40pm:  Innovation in Rare Diseases — Novel Approaches that Address Unmet Medical Needs

Advancements in personalized medicine have revolutionized the biomedical industry’s ability to treat even the rarest conditions. Antisense Oligonucleotides and computational biology hold promise of developing therapies for the ultra-rare disease populations that have historically had little hope of treatment. Unfortunately, economic barriers to developing therapies for ultra-rare conditions often prevent these treatments from becoming a reality for the patients who need them most. Innovation is needed not only in the science but in the economics of drug development to truly revolutionize rare disease medicine. In this session, meet leaders in the rare disease space who have made huge strides in lowering the cost of developing treatments and forging partnerships to impact treating rare disease populations.

• Dawn Barry, MBA, Co-Founder and President, Luna (moderator)
• Stanley Crooke, PhD, Founder & CEO, n-Lorem
• Jeff Milton, MCS, Founder and CEO, La Jolla Labs
• Kelley Dalby, M.Ed, Associate Director Natural History and Diagnostics, Epilepsy at Praxis Precision Medicines, Inc.

Speaker Bios

 

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1:35pm:  Accelerating Pediatric Cures — Building the Framework for Faster Diagnosis and Treatment

Genetic abnormalities account for 72% of rare diseases with two-thirds having childhood onset. Next-generation genetic sequencing and data analytics have dramatically impacted the ability to identify mutated genes immediately and the rapidly advancing field of gene and cell therapy offers hope for treatments and cures to patients and families impacted by rare diseases. Identifying and diagnosing these genetic abnormalities at birth can jump start a race against the clock to find treatments and cures for debilitating and deathly diseases. Despite these medical advancements, many families burdened by these rare diseases are financially disadvantaged and lack the means to access such technologies. In this session, learn how foundational support is paving the way for worldwide institutional collaboration to help rapidly diagnose underserved pediatric patient populations and how impact investing is propelling the translation of these diagnoses into commercial treatments. 

• Linda Strause, PhD, Principal/Founder, Strategic Clinical Consultants (moderator)
• Andrew Meadow, MBA, MA,  General Partner, Health Innovation Capital
• Sharon Terry, President and CEO, Genetic Alliance
• Wendy Benson, MBA, Chief Strategy & Innovation Officer, Rady Children’s Institute for Genomic Medicine
• Kristin Stephenson, MHA, JD, Director, Office of Strategy & Alliances – Pediatric Translational Neuroscience Initiative , St. Jude Research

Speaker Bios

 

 

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ONE ON ONE MATCHMAKING OPPORTUNITIES

California Life Sciences in collaboration with LaunchBio is dedicated to creating opportunities for startups and small companies to connect with Disease Foundations, Corporate Partners, and other funding sources for rare diseases. To that end, selected companies can apply by August 11, 2023 to meet virtually with the following organizations to discuss partnering opportunities. Attendance at the forum is not required to apply.

Brain Tumor Investment Fund (National Brain Tumor Foundation)

Chiesi

Cystic Fibrosis Foundation Fund

Dementia Discovery Fund

Foundation for Prader Willi Research Venture Philanthropy Fund

Health Innovation Capital

IBD Ventures (Crohn’s and Colitis Foundation)

Mission Cure Capital

Pathway To Cures (National Hemophilia Foundation)

Retinal Degeneration Fund (Foundation Fighting Blindness)

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