Setting the new standard in gene therapy

Our purpose is to change the lives of people affected by devastating diseases.

About Affinia Therapeutics

We are building a world-class gene therapy company.

Our proprietary rational design platform enables us to methodically engineer the next generation of AAV vectors and gene therapies. We’re finding solutions to the greatest challenges the field is facing, from tissue selectivity and immunogenicity, to manufacturing yields and payload capacity.

We have an experienced team that is focused on discovering, developing and manufacturing breakthrough medicines to transform people’s lives.

Our pipeline

Our science can benefit many therapeutic areas. Our initial focus is on devastating diseases affecting the central nervous system and affecting muscle.

Central Nervous System (CNS)

Genetic CNS disorders are among the most debilitating and difficult diseases to treat. Gene therapy holds great promise, but the availability of vectors that efficiently deliver the therapeutic cargo to diseased cells is limiting progress. With our unique platform, we are engineering the next generation of AAV vectors that penetrate more broadly and deeply into the CNS. Our new treatments will transform the lives of people with neurological diseases.

Central Nervous System (CNS)

Muscle

Gene therapies have shown the potential to slow the progression of disease and, in some cases, return lost function to patients suffering from genetic muscle disorders. The field now urgently needs improved vectors that not only deliver the therapeutic cargo to diseased cells, but do so safely and efficiently. We are applying our unique platform to develop these next generation vectors and realize the full potential of genetic medicines for people affected by muscular dystrophies.

Muscle

Partnerships

Our platform is delivering novel vectors that unlock new opportunities across cell types, tissues and therapeutic modalities. Forging strategic partnerships, such as our recently announced collaboration with Vertex Pharmaceuticals, helps us realize the full potential of our science and benefit more people.

Leadership team

Laura has authored or co-authored over 50 INDs and three BLAs. She was most recently the Senior Vice President of R&D at Arcellx. Prior to that, she was the Executive Director of the University of Pennsylvania’s Gene Therapy Program, where she led the preclinical development of more than 20 gene therapy programs across CNS, liver metabolic, ocular and respiratory diseases. Before this, she was Vice President, Translational Sciences at MedImmune. Laura is an author on more than 60 publications. She is a veterinary pathologist and molecular virologist by training, with a D.V.M. from the University of Wisconsin, Madison and a Ph.D. from Johns Hopkins School of Medicine.

Luk is a renowned scientific leader in AAV gene therapy research and development and a Scientific Co-Founder of Affinia Therapeutics. He has authored over 80 publications, holds more than 12 patents in the field and is a Co-Inventor of AAV9, a vector used in many gene therapies. Luk is also Co-Founder of gene therapy companies GenSight Biologics, Odylia Therapeutics and Akouos. He is the Grousbeck Family Associate Professor of Gene Therapy at Mass. Eye and Ear and Harvard Medical School and Director of the Grousbeck Gene Therapy Center. Luk trained at University of Leuven, Belgium and the University of Pennsylvania in biochemical engineering and molecular medicine.

Paula has led strategy and operations for more than 15 programs advancing them through meaningful milestones ranging from discovery to clinic and approvals to launches. She spent most of her career at Biogen in roles of increasing responsibility across new product planning, program management and franchise management, culminating as Senior Vice President of the Rare Disease group. In this role, she managed a portfolio of clinical and commercial assets across hemophilia, spinal muscular atrophy and other rare genetic diseases with annual revenues over $750 million. More recently, Paula was the Chief Operating Officer of gene therapy company, Decibel Therapeutics, and a Board member of gene therapy company, Nightstar Therapeutics, until its acquisition by Biogen in 2019 for $800 million. Paula is a current Board member of Prothena. She received her B.A. from Amherst College and her M.B.A. from Harvard University.

Rick has helped bring transformative medicines to patients in more than 10 diseases across multiple successful biotech companies. Before joining Affinia Therapeutics, Rick was Chief Business Officer at AveXis where he played a significant role in the company's build and sale to Novartis for $8.7 billion and the launch of Zolgensma®, its flagship gene therapy product. Prior to AveXis, Rick was Chief Business Officer at Catabasis Pharmaceuticals and before that, a Senior Vice President in the commercial group at InterMune until its acquisition by Roche. Before this, Rick held roles of increasing responsibility at MedImmune (AstraZeneca) and at Centocor (J&J). Before joining the industry, Rick practiced as a clinical pharmacist in a hospital setting. He received his B.S. in Pharmacy from the University of Iowa and his M.B.A. from the Wharton School.

Rob has closed more than 50 strategic transactions across a broad range of R&D collaborations, licensing agreements and mergers and acquisitions. Before Affinia Therapeutics, Rob was a Principal at the law firm Faber, Daeufer & Itrato PC, co-leading its R&D collaborations and licensing practice and working with prominent gene therapy clients. Prior to this, he was Vice President and Head of Business Strategy for GSK’s biotech partnering unit, and was Assistant General Counsel at GSK supporting Worldwide Business Development and R&D Operations. Earlier in his career, Rob held multiple roles including Biotech Patent Attorney at Akin, Gump, Strauss, Hauer, & Feld and Research Biochemist in Bioprocess R&D at Merck. Rob received his B.S. in Biochemistry from Albright College, M.S. in Biochemistry from Rutgers University and J.D. from Temple University.

Rob has managed the design, development and operations of manufacturing for biologics, vaccines and gene therapies making medicines that have changed the lives of millions of patients. He most recently led the design, build and operations of a state-of-the-art 170,000-square-foot GMP-grade gene therapy manufacturing plant for clinical and commercial supply at AveXis, where he was Vice President, North Carolina Site Head. Before AveXis, Rob was the U.S. Head of Technical Development Operations for Novartis/GSK Vaccines, where he was responsible for the delivery of several viral vaccine development programs using a variety of production platforms. He also spent 17 years on the biologics manufacturing team at Amgen. Rob received his B.A. in Biology from the University of California, Berkeley.

Scientific
co-founders

Aaron is an Associate Professor at the University of California, San Francisco (UCSF) and a Scientific Co-Founder of Affinia Therapeutics. He is a surgeon-scientist and a pioneer in the application of high-throughput sequencing technologies to critical problems in biomedicine. Aaron has authored more than 30 publications spanning the fields of genomics, cancer biology, stem cell biology and skull base surgery. He received both his Ph.D. in Biomedical Sciences and his M.D. from UCSF, and completed his residency in Otolaryngology-Head and Neck Surgery and fellowship in Otology, Neurotology and Skull Base Surgery at Harvard Medical School, as well as a post-doctoral fellowship at the Broad Institute of MIT and Harvard.

Botond is a founding Director of the Institute of Molecular and Clinical Ophthalmology in Basel, Professor at the Faculty of Science and Medicine at the University of Basel and a Scientific Co-Founder of Affinia Therapeutics. He is an author on more than 80 publications in the areas of gene therapy, regulatory elements and neuronal circuits. Botond is the recipient of multiple awards and a member of many advisory and editorial boards. Botond received his M.D. at the Semmelweis Medical School in Budapest, Hungary, and his Ph.D. in Neurobiology at the University of California, Berkeley.

Luk is a renowned scientific leader in AAV gene therapy research and development and a Scientific Co-Founder of Affinia Therapeutics. He has authored over 80 publications, holds more than 12 patents in the field and is a Co-Inventor of AAV9, a vector used in many gene therapies. Luk is also Co-Founder of gene therapy companies GenSight Biologics, Odylia Therapeutics and Akouos. He is the Grousbeck Family Associate Professor of Gene Therapy at Mass. Eye and Ear and Harvard Medical School and Director of the Grousbeck Gene Therapy Center. Luk trained at University of Leuven, Belgium and the University of Pennsylvania in biochemical engineering and molecular medicine.

Board of directors

Dave is a partner at Atlas Venture where he has co-founded and led the growth of multiple biotech companies. He was Co-Founder and Chief Executive Officer of Arteaus Therapeutics (acquired by Eli Lilly) and of Annovation Biopharma (acquired by The Medicines Company) and a founding board member of Delinia (acquired by Celgene). Dave is a Co-Founder and Board member of Surface Oncology, Cadent Therapeutics and Q32 Bio. He also sits on the Board of Xilio Therapeutics. Dave received his B.A. from Stanford University and M.D. from Harvard Medical School.

Ed is a general partner at New Enterprise Associates (NEA) where he has invested in multiple companies with successful exits including Lumena (acquired by Shire), Ziarco (acquired by Novartis), Motus Therapeutics (acquired by Allergan) and Plexxikon (acquired by Daiichi Sankyo). Before joining NEA, Ed was Executive Vice President, Corporate Development and Venture at MedImmune and part of the leadership team that sold the company to AstraZeneca for $15.6 billion. Prior to MedImmune, he held roles of increasing responsibility at Inhale Therapeutic Systems and at Glaxo Wellcome. Ed is a Board member of multiple biotech and pharmaceutical companies, including gene therapy companies Akouos and Shape Therapeutics. He received his B.S. in Chemistry from North Carolina State University.

Elliott is a renowned scientific and industry leader who recently served on the board of gene therapy pioneer Spark Therapeutics for five years. During that time, the company developed and launched Luxturna®, the first AAV gene therapy in the United States, and was acquired by Roche for $4.3 billion. Elliott is a former Executive Vice President and Director of Bristol-Myers Squibb (BMS) where he also served as Chief Scientific Officer and President of R&D from 2004 to 2013. Under Elliott’s leadership, 14 new medicines were approved at BMS and in 2012, Scrip Intelligence named him the best R&D Chief in the pharmaceutical industry. Elliott serves as a Senior Advisor to the healthcare team of New Enterprise Associates and is a Board member of Adaptimmune, Surface Oncology and Vir Biotechnology. He co-chairs the Scientific Advisory Board for Amgen and is a member of the Scientific Steering Committee for the Sean Parker Institute for Cancer Immunology. Elliott received his B.S., M.S. and Ph.D. in Industrial Engineering from Purdue University and M.D. from the University of Chicago.

Luk is a renowned scientific leader in AAV gene therapy research and development and a Scientific Co-Founder of Affinia Therapeutics. He has authored over 80 publications, holds more than 12 patents in the field and is a Co-Inventor of AAV9, a vector used in many gene therapies. Luk is also Co-Founder of gene therapy companies GenSight Biologics, Odylia Therapeutics and Akouos. He is the Grousbeck Family Associate Professor of Gene Therapy at Mass. Eye and Ear and Harvard Medical School and Director of the Grousbeck Gene Therapy Center. Luk trained at University of Leuven, Belgium and the University of Pennsylvania in biochemical engineering and molecular medicine.

Rick has helped bring transformative medicines to patients in more than 10 diseases across multiple successful biotech companies. Before joining Affinia Therapeutics, Rick was Chief Business Officer at AveXis where he played a significant role in the company's build and sale to Novartis for $8.7 billion and the launch of Zolgensma®, its flagship gene therapy product. Prior to AveXis, Rick was Chief Business Officer at Catabasis Pharmaceuticals and before that, a Senior Vice President in the commercial group at InterMune until its acquisition by Roche. Before this, Rick held roles of increasing responsibility at MedImmune (AstraZeneca) and at Centocor (J&J). Before joining the industry, Rick practiced as a clinical pharmacist in a hospital setting. He received his B.S. in Pharmacy from the University of Iowa and his M.B.A. from the Wharton School.

Robert is a partner at F-Prime Capital where he has invested in multiple companies with successful exits including Accuri Cytometers (acquired by Becton Dickinson), Ikano Therapeutics (acquired by UCB) and Topaz (acquired by Sanofi Pasteur). Prior to F-Prime, he worked extensively in both academia and in industry. In industry, Robert held senior roles in R&D and business development at pharmaceutical and medical device companies. He is the author of more than 100 scientific papers and an inventor on six patents. Robert serves on the Board of multiple portfolio companies including Farapulse, Tempest Therapeutics and Xilio Therapeutics. He received his B.A. degree in Physics from Harvard University, Ph.D. in Physics from the Massachusetts Institute of Technology and M.B.A. from Columbia University.

Sean is Chairman of the Board of Directors at Affinia Therapeutics. Previously, he served as Chief Executive Officer of gene therapy company AveXis until its acquisition by Novartis for $8.7 billion. Sean took the company through an IPO and built it into a fully integrated global organization with research, clinical, regulatory, manufacturing, and commercial capabilities. He was previously Chief Business Officer of InterMune, Chief Commercial Officer of Reata and Ovation Pharmaceuticals and President of Lundbeck’s U.S. affiliate. Sean serves on the Boards of Encoded Therapeutics, Itsari Oncology, Taysha and Ventas. He received his B.A. in Biology from John Carroll University.

Join us

Guided by knowledge and fueled by passion, we stand determined to deliver what AAV gene therapy can — and will — do to improve human health. Our mission is to turn great scientific discoveries into even greater medicines — bringing meaningful change to people suffering from devastating diseases.

Job listings

Contact us

*Required

First Name is Required.

Last Name is Required.

Email is Required.

Please enter a valid phone number.

Message is Required.