Kymeti

Kymeti is a biotechnology company focused on developing innovative solutions for the treatment of rare genetic diseases. Founded in 2016, the company is headquartered in Cambridge, Massachusetts, and operates globally. Kymeti's primary research and development efforts are centered around the use of gene editing technologies, specifically CRISPR-Cas9, to correct genetic mutations that cause these diseases.

The company's pipeline includes several programs targeting various rare genetic disorders. One of its notable projects is the development of a gene therapy for spinal muscular atrophy (SMA), a severe neuromuscular disorder that affects the muscles and nerves. Kymeti's approach involves using CRISPR-Cas9 to edit the genetic code of stem cells, which are then used to generate functional neurons that can replace damaged ones in the spinal cord.

Kymeti collaborates with academic institutions, research organizations, and pharmaceutical companies to advance its research and development efforts. The company has also established partnerships with healthcare providers and patient advocacy groups to ensure that its therapies are accessible and beneficial to patients with rare genetic diseases.

Kymeti's mission is to improve the lives of patients with rare genetic diseases by providing them with effective and accessible treatments. The company aims to achieve this by leveraging its expertise in gene editing and its commitment to innovation and collaboration.