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 myelin-based disorders of the central nervous system for which few, if any, treatment options exist.

Discover Our Technology

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

Canavan Phase 1/2 Trial

Our clinical trial to treat Canavan disease is underway, utilizing the first clinical technology to target oligodendrocytes. Encouraging results to date pave the way for further development.

See Our Current Trial

Novel AAV

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 oligodendrocytes.

Extensive Patent Estate

Our intellectual patent portfolio includes technologies from an exclusive worldwide licensing agreement with Pfizer Inc. We have an ongoing global procurement of multiple patent families across the composition of matter, unique vector and gene sequences, routes of administration, and method of use claims.

Robust Discovery Pipeline

Myrtelle’s proprietary technologies target global patient populations with unmet medical needs. Our enabling technologies allow for a therapeutic pipeline with hundreds of disease candidates, from prevalent indications like MS and MSA to rare diseases like Canavan, PMD and H-ABC.

See Full Pipeline

Patient-Centered Approach

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

Experienced Management

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.

Meet the Team