aav
(7)
Beacon Therapeutics
Beacon Therapeutics is a clinical-stage ophthalmic gene therapy company building AAV-based treatments to preserve and restore sight for people with rare and prevalent retinal diseases that cause blindness. Formed in 2023 by Syncona around the assets of Applied Genetic Technologies Corp (AGTC) and complementary science from the University of Oxford, its lead program, laru-zova (AGTC-501), is a one-time gene therapy for X-linked retinitis pigmentosa now in registrational trials, with additional programs targeting geographic atrophy (dry AMD) and cone-rod dystrophy.
Joel Schneider
Joel Schneider is the founder, president and CEO of Carbon Biosciences, a Waltham, Massachusetts biotech building gene therapies from parvovirus-derived vectors rather than the more common adeno-associated viruses. He launched the company out of stealth in 2022 with a $38 million Series A led by Agent Capital, pairing his operating background with the science of gene therapy pioneer Robert Kotin. Trained as a stem cell biologist with a Rutgers Ph.D. and a Harvard postdoc, Schneider was the first employee at Solid Biosciences before founding Carbon to push vectors that carry larger genetic payloads, target specific tissues such as the lung, and sidestep the pre-existing immunity that limits current gene therapies. Carbon's lead program targets cystic fibrosis.
Cyagen Biosciences
Cyagen Biosciences is a life-sciences contract research organization that builds custom genetically engineered rodent models - knockout, knockin, conditional, and humanized mice and rats - for academic labs and drug developers. Founded in 2006 and headquartered in Santa Clara, California, with major operations in China, Japan, and Europe, Cyagen has grown into one of the world's largest suppliers of custom mouse and rat models, complemented by stem-cell products and a fast-growing gene-delivery arm, VectorBuilder, that designs and manufactures viral vectors from research through clinical CDMO work.
Ramon Salsas Escat
Ramon Salsas Escat is the Chief Business Officer of GEMMA Biotherapeutics (GemmaBio), the Philadelphia gene-therapy company spun out of Jim Wilson's lab to widen global access to AAV therapies for rare inherited diseases. A chemist by training who earned a PhD in computational and systems biology at MIT and an MBA at IESE, Salsas has led or co-led more than 20 transactions across AAV gene therapies, CNS disorders, oncology and manufacturing platforms, with cumulative deal value north of $4 billion in upfront payments and milestones. He helped build the business engine at Penn's Gene Therapy Program, served as SVP of Business Development at G2 Bio, and now runs business development, alliance management, legal and IP at GemmaBio, where he played a pivotal role in the company's founding and its $34 million seed round.
克睿基因 Cure Genetics
克睿基因 Cure Genetics is a clinical-stage biotech founded in Suzhou in 2016 that develops cell and gene therapies for solid tumors and genetic diseases. It runs two proprietary platforms: AIMS, an allogeneic CAR-NKT cell therapy approach aimed at hard-to-treat solid tumors, and VELP, a directed-evolution AAV vector platform that builds high-quality viral libraries for tissue-targeted gene delivery to the nervous system, heart, and kidney. Its lead programs include CGC729 (anti-CD70 CAR-NKT for renal cell carcinoma) and CGC-602 (a PD-1-IL-2v bispecific antibody).
Yuanyuan Xu
Yuanyuan Xu is a structural biologist turned biotech founder who left the protein-crystallography bench at Tsinghua and Yale to build Cure Genetics (克睿基因), a Suzhou clinical-stage company chasing cell and gene therapies for solid tumors and genetic disease. She founded it in 2016, raised a $60M Series B in 2021, and bets on two homegrown platforms: an invariant-natural-killer-T cell therapy (AIMS CAR-NKT) and a directed-evolution engine for new AAV capsids (VELP).
Sandy Macrae
Sandy Macrae is a Scottish physician-scientist who has led Sangamo Therapeutics, the Brisbane, California gene-editing pioneer, as President and CEO since June 2016. Trained at Glasgow and Cambridge - where Nobel laureate Sydney Brenner supervised his PhD - he ran emerging-markets R&D at GSK and was Global Medical Officer at Takeda before staking his career on zinc finger technology and genomic medicines for rare and neurological diseases.