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A crisis in rare disease: How the current investment landscape is stifling rare disease development

One in ten people suffer from rare diseases in the United States. There is a big need for therapeutic advancement for the rare disease community and, oftentimes, treatments for these diseases are the patient’s last hope. However, there is a crisis happening today in rare disease. The current public markets are stifling innovation and drug development for rare diseases. This webinar will examine the current markets, their effect on the innovation in rare disease drug development, and what we can do to help.

  • Learn about the current state of investments for the rare disease sector
  • Understand the market’s effect on innovation in rare disease
  • Hear from leading experts on what we do as a community to help drive innovation forward
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Alex Karnal

Alex Karnal

Founder, Braidwell

Alex Karnal is Co-founder and Chief Investment Officer of Braidwell, a life science-focused investment firm dedicated to serving the people and organizations that transform human health. Braidwell provides flexible capital and thoughtful partnership to companies at all stages of development, at all parts of the capital structure, across public, private, and structured capital markets.

Prior to co-founding Braidwell, Alex was a Partner, Managing Director and the Co-Portfolio Manager at Deerfield Management, from 2005 until 2021. Beyond his direct Deerfield investment activities, Alex helped conceptualize and implement the Cure, Deerfield’s healthcare innovation campus at 345 Park Avenue South. During the COVID-19 global health crisis, Alex was appointed by Governor Ned Lamont to the Reopen Connecticut Advisory Group to provide counsel on the reemergence of the state’s economy, community and education systems. He is a co-founder and board chairman of The Institute for Life Changing Medicines, a nonprofit dedicated to developing medicines for people suffering from less common, life-threatening, diseases around the world. Alex serves on the Board of the Biotechnology Innovation Organization, and previously served on the Boards of the Museum of Science, the Children’s Health Fund as Vice Chairman, New York Academy of Medicine, Discovery Labs, and Recovery Centers of America, the latter two of which he co-founded. He is also a member of the Board of the Greenwich Country Day School. Prior to Deerfield, he was a member of Merrill Lynch’s Global Equity Linked Products Group, as well as its Capital Markets team. He received a B.S. from the Massachusetts Institute of Technology and an M.S. from Johns Hopkins University.

Chidozie Ugwumba

Chidozie Ugwumba

Managing Partner, SymBiosis

Chidozie Ugwumba serves as Managing Partner of SymBiosis, a venture capital firm focused on investments in biotherapeutics. At SymBiosis, Mr. Ugwumba leads sourcing, due diligence, and execution of investments, and serves as an advisor to SymBiosis’ portfolio companies. He has led numerous investments in therapeutics companies in the US and Europe, across disease areas including CNS, cardiac, dermatology, GI, infectious disease, and oncology, and across modalities such as precision medicine, and cell, gene, and regenerative therapy. Prior to SymBiosis, Mr. Ugwumba led the Direct and Impact Investment Group of WIT, LLC and worked on the Private Credit and Infrastructure teams at Partners Group, a global private investment manager. Mr. Ugwumba earned an MBA from the Johnson Graduate School of Management at Cornell University, and a BA in Political Science from Amherst College.

James Wilson

James Wilson

Director, Perelman School of Medicine, UPenn

Dr. Jim’s commitment to patients with rare diseases stemmed from his days as a graduate student and medical student at the University of Michigan between 1977 and 1984. He was discouraged by the lack of available treatments for rare disease patients but optimistic that science could yield solutions with his initial focus being gene therapy. He completed his training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute where he began his work in gene therapy.

After launching his faculty career at the Howard Hughes Medical Institute at the University of Michigan, he moved to the University of Pennsylvania in 1993. Jim’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for the translation of these technologies into the clinic. He has published more than 600 papers and is named on more than 1,200 patents worldwide. Jim is the founder and President of a 501(c)3 called Health Through Fitness in Orphan Diseases.

Tony Khoury

Tony Khoury

EVP, Project Farma

Tony Khoury is an experienced industry leader in life sciences, specifically, biologics and advanced therapies. As a member of the Project Farma leadership team, Tony has spearheaded the firm’s growth in the biologics and most recently next-generation medicines. Tony has extensive experience with small and large molecules including biologics, biosimilars, and cell and gene therapies. He has worked with small and large life science organizations including startups, academic centers, financial institutions and CMOs/CROs. He has led global programs greater than $350+ million in the following disciplines: project management, turnkey facility builds, automation, validation, engineering and serialization track and trace. Recently, Tony has pioneered the industrialization of advanced therapies including two FDA-approved cell and gene therapies. Tony has led the creation of Project Farma’s Advanced Therapy Manufacturing Playbook which has helped develop the largest cell and gene therapy manufacturing footprint in the world.

Anshul Mangal

Anshul Mangal

President, Project Farma & Precision ADVANCE

Anshul is the President of Project Farma (PF) and Precision ADVANCE. He founded and grew PF into a leading global biologics and advanced therapy engineering consulting firm. In addition, Anshul has served as general counsel and managed all corporate legal transactions. Under Anshul's leadership, PF pioneered the industrialization of advanced therapies including two notable, commercially approved cell and gene therapies, and created PF's Advanced Therapy Manufacturing Playbook. This playbook helped develop the largest cell and gene therapy manufacturing footprint in the world. To date, PF has led 20+ facility builds with over $1BB in capital spend, selected and managed 60+ CDMO engagements, and partnered with over 40+ advanced therapy organizations.

Project Farma was acquired by Precision Medicine Group in 2020 to be the cornerstone of Precision ADVANCE. ADVANCE is a collection of Precision's interconnected services and complementary teams that uniquely focuses on the complexities of clinical, regulatory, manufacturing, and commercial needs to successfully bring a cell or gene therapy to market. Anshul is also dedicated to advancing next-generation medicines through his philanthropic efforts at the Institute for Life Changing Medicines, various patient-advocacy organizations, and the Alliance for Regenerative Medicine.