The Myrtelle Difference

Myrtelle is a patient-centered, innovation-driven gene therapy company transforming the treatment of neurological diseases. Our mission is to develop and deliver novel treatments for devastating disorders of the central nervous system for which few, if any, treatment options exist.

We envision a world in which patients with rare
CNS diseases can access treatments that improve
their quality of life & longevity

Our clinical trial to treat Canavan disease is underway, utilizing the first clinical technology to target oligodendrocytes. The team has completed manufacturing cGMP to initiate the clinical trial, and has established contract manufacturing for additional product.

We intend to become the world leader in gene therapies for white matter diseases. We are committed to advancing our unique insights and capabilities in disorders involving myelin, building on our understanding of unique AAV vectors that for the first time enable robust targeting of oligodendroyctes. We also are investing in cutting edge approaches for bio-energic rescue in a broad range of CNS diseases.

Myrtelle holds broad intellectual property, with patented technologies from an exclusive worldwide licensing agreement with Pfizer Inc. We have an ongoing global procurement of multiple patent families across composition of matter, unique vector and gene sequences, routes of administration, and method of use claims.

Myrtelle’s proprietary technologies target global patient populations with unmet medical needs. The enabling technologies allow for a therapeutic pipeline with hundreds of disease candidates, from large indications like MS, to orphan diseases like Canavan. Primary discovery candidates have been identified.

Myrtelle is a patient-centered, innovation-driven, gene therapy company transforming the treatment of central nervous system disorders to benefit the livelihood of sufferers globally.

Myrtelle’s team brings decades of deep experience in drug development, rare diseases and CNS gene therapy to advance our mission. Our scientific advisors are from MIT, Harvard, Eli Lilly, GlaxoSmithKline, and Rowan University.