Potential Best-in-Class Approach for the treatment of AATD
AATD is a hereditary disorder most commonly caused by the SERPINA1 E342K (PiZZ) mutation, which causes the liver to produce misfolded Z-AAT in place of normal M-AAT. Toxic accumulation of Z-AAT polymers in the liver can lead to hepatitis, fibrosis, and cirrhosis. At the same time, reduced levels of functional AAT in circulation leave the lungs vulnerable to progressive emphysema and chronic obstructive pulmonary disease (COPD).
Published studies estimate approximately 100,000 prevalent PiZZ individuals in the United States, with a similar number across Europe. There is currently no FDA-approved disease-modifying therapy for AATD, nor any approved therapy that addresses its liver manifestations. The only approved treatment directed at the underlying deficiency is weekly intravenous augmentation therapy, which does not address liver disease.
Our commitment to transforming the treatment of alpha-1 antitrypsin deficiency, or AATD
ABOUT SERAPHA BIO
Our potentially best-in-class approach for the treatment of AATD
SERP-01 is a proprietary in vivo base editing program currently in a Phase 1/2 clinical trial for the treatment of AATD with the PiZZ genotype, the most severe genetic mutation responsible for >90% of AATD cases. SERP-01 is designed to restore production of functional M-AAT while reducing toxic Z-AAT, with the potential to address both the liver and lung manifestations of AATD with a single intravenous administration.
Seasoned team with deep expertise spanning genetic medicines, regulatory strategy, clinical and product development, manufacturing and commercialization.
LEADERSHIP TEAM
Ken Mills
Chief EXECUTIVE OfficerKen Mills has spent over two decades in executive leadership roles in the biotechnology industry. Previously, he was President, Chief Executive Officer, and director of Tagworks Pharmaceuticals BV, a precision oncology company, from July 2024 to July 2026. Before that, he was the founding President and Chief Executive Officer of REGENXBIO from 2009 to 2024 and currently serves as the Chairman of the Board of Directors. Mr. Mills led REGENXBIO’s initial public offering in 2015 and grew the company into a leading gene therapy biotech, with end-to-end capabilities including research and development, clinical, and manufacturing. Before his 15-year tenure at REGENXBIO, Mr. Mills was the Chief Financial Officer and Vice President of Business Development at Meso Scale Diagnostics, a privately held life sciences company. Prior to Meso Scale Diagnostics, he was Director of Business Development for IGEN International, a medical diagnostics company. Mr. Mills received an SB in chemistry from the Massachusetts Institute of Technology.
Daphne Karydas
PRESIDENT & CHIEF FINANCIAL OFFICERDaphne Karydas has over 20 years of financial leadership experience across biopharmaceutical company operations, capital markets, and healthcare-focused investment banking. Previously, she was President and Chief Financial Officer of Flare Therapeutics, a transcription factor targeting oncology company, which she joined in October 2021. Prior to Flare Therapeutics, she served as the Chief Financial Officer of Syndax Pharmaceuticals Inc. from July 2020 to October 2021. From April 2017 to May 2020, Ms. Karydas held several roles at Allergan plc, including Senior Vice President of Corporate Strategy and Financial Planning & Analysis, until the company’s acquisition by AbbVie. Before that, Ms. Karydas spent more than 15 years in investment banking and asset management roles that focused exclusively on the healthcare space, including biopharmaceuticals, life sciences and medical technologies. Ms. Karydas is on the Board of Directors of COMPASS Pathways, Mineralys Therapeutics, Inc. and Epikast. Ms. Karydas earned her MBA from Harvard Business School and holds a BA and MS in chemical engineering from the Massachusetts Institute of Technology.
Weston Miller, MD
Chief medical officerWeston Miller, MD, has over two decades of experience in clinical development of in vivo and ex vivo genomic medicines for patients with rare, severe genetic disorders. Dr. Miller was previously Chief Medical Officer of Epicrispr Biotechnologies, a company pioneering programmable epigenetic medicines. Before that, he was at Graphite Bio, leading the investigation of an ex vivo gene-corrected autologous hematopoietic stem cell product for patients with severe sickle cell disease. He has also held Medical Director roles at Astellas Gene Therapies and Sangamo Therapeutics. He began his career as an associate professor of pediatrics in the division of pediatric blood and marrow transplantation (BMT) at the University of Minnesota. Dr. Miller earned his MD from Louisiana State University School of Medicine, New Orleans and holds a BS in chemistry from Stanford University.
Nina Hunter, PhD
CHief development officerNina Hunter, PhD, Chief Development Officer, has close to two decades of regulatory and drug development leadership spanning industry and government. Dr. Hunter was previously SVP, Global Regulatory Strategy and Quality at REGENXBIO, where she led global regulatory strategy and policy and oversaw regulatory affairs from early research through BLA submission and FDA review, with additional responsibility for Quality functions. Previously, she led Corporate Strategy at REGENXBIO and co-led the company’s global partnership with Nippon Shinyaku for the development and commercialization of its MPS gene therapy programs. Prior to joining REGENXBIO in 2020, Dr. Hunter spent 12 years at the U.S. Food and Drug Administration, rising to Director of the Office of Clinical Policy and Programs and supporting the work of multiple FDA Commissioners and Principal Deputy Commissioners through cross-agency policy and regulatory initiatives. She earned her PhD in Genetics from Harvard Medical School and an AB in Biochemistry from Bowdoin College.
Shiva G. Fritsch
Chief CORPORATE AFFAIRS officerShiva G. Fritsch has more than two decades of experience supporting the growth and strategic evolution of biopharmaceutical companies. Most recently, Ms. Fritsch served for nine years as Chief Communications and People Officer at REGENXBIO, where she built and led the people, communications and patient advocacy functions during the company’s advancement of a broad clinical and pre-commercial gene therapy portfolio. She oversaw talent and organizational strategy, total rewards, corporate and scientific communications, public relations, internal communications and patient engagement. Previously, Ms. Fritsch held various leadership roles at Novavax, Inc., Howard Hughes Medical Institute and Human Genome Sciences, Inc. She earned her MBA from the University of Maryland’s Robert H. Smith School of Business and holds a BA in Biology and Neurobiology from Bryn Mawr College.
