cystic-fibrosis
(4)
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.
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.