Leadership Team
Chief Executive & Medical Officer
Chief Executive & Medical Officer
Chief Executive & Medical Officer
Dr. Brandon M. Henry, M.D.
Dr. Brandon M. Henry, M.D., is a physician–scientist specializing in the clinical development of AAV gene therapies for rare and ultra-rare disorders. His work bridges academic and industry domains, with a focus on advancing clinical development strategies for neurodevelopmental, neurodegenerative, neuromuscular,
Dr. Brandon M. Henry, M.D.
Dr. Brandon M. Henry, M.D., is a physician–scientist specializing in the clinical development of AAV gene therapies for rare and ultra-rare disorders. His work bridges academic and industry domains, with a focus on advancing clinical development strategies for neurodevelopmental, neurodegenerative, neuromuscular, and inborn errors of metabolism, where innovative gene therapy approaches have the potential to transform previously untreatable conditions.
Dr. Henry brings deep expertise in AAV vector biology, CNS-directed delivery, immune risk mitigation, and long-term safety monitoring, with extensive experience designing and executing complex pediatric gene therapy trials. He has led multiple AAV programs across academic, biotech, and nonprofit settings, guiding assets from preclinical translation through IND submission, clinical development, and toward registration, with a particular emphasis on rigorous safety frameworks, novel endpoint development, and regulatory alignment for transformative neurologic therapies.
Chief Business Officer
Chief Executive & Medical Officer
Chief Executive & Medical Officer
Amber Freed
Amber is passionate about building an ecosystem that can systematically help fund and consolidate research and treatment efforts. Amber brings a wealth of lived experience from driving her own son's gene therapy from bench to the clinic. Since her son's diagnosis in 2018 Amber has single-handedly repurposed a drug, driven m
Amber Freed
Amber is passionate about building an ecosystem that can systematically help fund and consolidate research and treatment efforts. Amber brings a wealth of lived experience from driving her own son's gene therapy from bench to the clinic. Since her son's diagnosis in 2018 Amber has single-handedly repurposed a drug, driven multiple translational treatments forward, and become a leader within the rare disease community.
Amber is the proud mother of adorable twins, Miss Riley James and Mr. Maxwell Norman. Maxwell was just 18 months old when she received his devastating diagnosis of SLC6A1, a rare neurological disease. Amber left her career in equity research analysis the day Maxwell was diagnosed and dedicated her life to finding a cure. Amber serves as the Founder and CEO of SLC6A1 Connect.
Chief People Officer
Chief Executive & Medical Officer
Chief People Officer
Effie Parks
Effie has become a guiding light in the rare disease community following her son Ford's diagnosis with CTNNB1 syndrome. She transformed her family's journey into a crusade for advocacy, support, and empowerment for families navigating similar challenges. As the host of the "Once Upon a Gene" podcast, Effie has been recognized
Effie Parks
Effie has become a guiding light in the rare disease community following her son Ford's diagnosis with CTNNB1 syndrome. She transformed her family's journey into a crusade for advocacy, support, and empowerment for families navigating similar challenges. As the host of the "Once Upon a Gene" podcast, Effie has been recognized for several awards including WEGO Health and Podcast Magazine for her impactful storytelling and resource-sharing in the realm of rare genetic disorders.
Effie extends her advocacy through speaking engagements at medical and patient advocacy conferences, sharing her experiences and insights from her work to bridge the gap between all rare disease stakeholders. Her skill in community engagement, developed through her advocacy, empowers her efforts in building a supportive network and raising awareness.
