cystic-fibrosis
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Carbon Biosciences
Carbon Biosciences is a Waltham, Massachusetts preclinical genetic-medicine company building gene therapies on non-AAV parvovirus vectors. Founded by Longwood Fund with gene-therapy pioneers John Engelhardt and Robert Kotin, it uses a proprietary vector engine (PAVE) to design capsids from the broad parvovirus family - including human bocavirus - that carry larger genetic payloads, target specific tissues, and provoke little pre-existing immunity, opening the door to re-dosing. Its lead program targets cystic fibrosis by delivering the full-length CFTR gene to the lung, with a second vector aimed at cardiac disease.
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.
Krystal Biotech, Inc.
Krystal Biotech is a Pittsburgh-based commercial-stage biotechnology company that pioneered redosable, off-the-shelf gene therapy delivered directly to the body. Its lead product VYJUVEK became the first FDA-approved topical and redosable gene therapy in 2023, treating the rare skin disease dystrophic epidermolysis bullosa. Built on a proprietary HSV-1 vector platform (STAR-D), the company runs its own GMP manufacturing and is extending the same delivery technology into respiratory disease, oncology, ophthalmology and aesthetics.
Aluna
Aluna is a San Francisco digital health company that turns daily breathing into data. Its FDA-cleared, palm-sized spirometer pairs with a gamified app and a clinician dashboard so people with asthma, COPD and cystic fibrosis - and their doctors - can track lung function (FEV1) at home and catch trouble before it becomes an emergency. Founded by three UC Berkeley alumni and originally named Knox Medical Diagnostics, Aluna raised about $27M total, including a $15.3M Series B in 2023, and was acquired by Huma in May 2025.
ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines company using its Selective Organ Targeting (SORT) lipid nanoparticle platform to deliver mRNA and gene-correction therapies beyond the liver. Its lead programs target cystic fibrosis and primary ciliary dyskinesia - rare respiratory diseases where existing modulators leave many patients without options.
David Lockhart
David J. Lockhart, Ph.D. is President and Chief Scientific Officer of ReCode Therapeutics, a clinical-stage biotech pioneering nonviral lipid nanoparticle (LNP) delivery of mRNA and gene-editing payloads to the lungs for genetic respiratory diseases including cystic fibrosis and primary ciliary dyskinesia. With more than 25 years in drug discovery and genomics — from co-founding Ambit Biosciences to leading science at Amicus Therapeutics — Lockhart brings rare-disease chops and deep genomics expertise to ReCode's SORT LNP platform, which has raised $345M and dosed its first patients in a Phase 1 PCD trial.