Established in November 2019, Doylestown-based GeneLancet Biosciences develops in vivo gene and cell therapies built around its proprietary single-molecule guide RNA, or LgRNA. The company is advancing LgRNA-conjugate-guided STAR and DuoSTAR genome editors for precise treatments aimed at serious diseases including neurodegenerative disorders, cancer and chronic hepatitis B. Its platform emphasizes scalable, high-purity guide RNA and editing approaches designed to avoid double-strand DNA breaks and support repeat or multiplex treatment strategies.
AUM LifeTech, IncPhiladelphia-based AUM LifeTech develops RNA-targeting therapeutics through its proprietary AUM silence antisense oligonucleotide platform, which is designed to silence or regulate disease-linked RNA without an external delivery vehicle. Founded by biotechnology entrepreneur Veenu Aishwarya, the company is pursuing preclinical treatments for genetically defined diseases, including an anti-FOXP3 cancer-immunotherapy program developed with Children’s Hospital of Philadelphia. Its work also includes a Parkinson’s disease program conducted with the University of Pennsylvania.PHILADELPHIA, PA
Rocket Pharmaceuticals, IncRocket Pharmaceuticals is a Cranbury, New Jersey biotechnology company developing and manufacturing genetic therapies intended to address the root causes of rare diseases. Its integrated research and manufacturing operation includes roughly 30,000 square feet of laboratories and a 103,720-square-foot facility built in 2022, with programs spanning inherited cardiovascular and blood disorders. Rocket’s first FDA-approved gene therapy, KRESLADI, treats severe leukocyte adhesion deficiency-I.NEW YORK, NY
LineaRxFormed in 2018 to commercialize Applied DNA Sciences’ more than 20 years of enzymatic DNA expertise, LineaRx produces therapeutic LineaDNA™ through a proprietary, cell-free PCR process. Its linear DNA is made as an alternative to plasmid-based manufacturing, with applications including mRNA production and gene and cell therapies. Production is supported by a GMP-compliant facility operating within ISO 9001- and ISO 13485-based quality systems.STONY BROOK, NY
Andelyn BiosciencesBorn from decades of gene-therapy research at Nationwide Children’s Hospital, Columbus-based Andelyn Biosciences is a contract development and manufacturing organization that develops and produces viral vectors and plasmids for cell and gene therapies. Spun out as an independent company in January 2020, Andelyn carries a patient-centered origin story into work spanning early development through clinical and commercial manufacturing. Its two Ohio facilities support a record that includes more than 500 cGMP clinical batches and the production experience behind an FDA-approved systemic gene therapy.COLUMBUS, OH