Myrtelle is advancing a robust pipeline of gene therapy programs using proprietary technologies, vectors, constructs, and capabilities. Our core area of focus is targeting oligodendrocytes to address underlying causes of disorders involving myelin.
Myrtelle’s clinical program for Canavan disease is the first gene therapy to specifically target oligodendrocytes. rAAV-Olig001-ASPA has been granted Orphan Drug, Fast-Track and Rare Pediatric Disease Designations for the treatment of Canavan disease in the US, with similar designations in Europe.
Learn More About Canavan DiseaseMyrtelle is investigating potential gene therapies for MSA, a neurological condition in which some parts of the brain gradually shrink and deteriorate. MSA typically affects adults, causing difficulty with movement, balance, coordination, speech, and maintaining normal blood pressure. MSA shares many symptoms with Parkinson’s disease. The cause is unknown, however, in MSA, a protein called alpha-synuclein typically builds up and forms clumps in certain brain cells (called oligodendrocytes). These are the brain cells that make myelin, the material that wraps around parts of other brain cells (called neurons) and helps them to communicate with each other.
Learn More About MSAMyrtelle is investigating potential gene therapies for H-ABC Syndrome. H-ABC is a leukodystrophy, a disease caused by a gene mutation that affects brain white matter. White matter is brain tissue that has a lot of myelin, the material that wraps around parts of the brain cells (called neurons) and helps those cells to communicate with each other. H-ABC syndrome is caused by a mutation in the TUBB4A gene, which is necessary to make the microtubules that move things around inside cells. This mutation disrupts the myelin-making cells of the brain (called oligodendrocytes). When oligodendrocytes don’t make enough myelin (hypomyelination), it is hard for kids to move and speak as they grow. Symptoms of H-ABC usually appear when kids are very young, affecting their ability to walk and talk.
Learn More About H-ABCMyrtelle is investigating potential gene therapies for PMD. PMD is a leukodystrophy, a disease caused by a gene mutation that affects brain white matter. White matter is brain tissue that has a lot of myelin, a material that wraps around parts of the brain cells (called neurons) and helps those cells to communicate with each other. The most common form of PMD is caused by duplication of the PLP1 gene. This causes the myelin-making cells of the brain (called oligodendrocytes) to make too much of a protein called proteolipid protein-1 (PLP1), which disrupts myelin formation. When oligodendrocytes don’t make enough myelin (hypomyelination), it hard for signals to travel properly. Symptoms of PMD usually appear when kids are very young, leading to problems like shaky movements, difficulty walking, and learning challenges.
Learn More About Pelizaeus Merzbacher DiseaseMyrtelle is investigating potential gene therapies for DFNB-8, a specific type of inherited hearing loss. DFNB-8 is caused by a mutation in the TMPRSS3 gene, which the hair cells of the inner ear need to make a protein called TMPRSS3. Hair cells are critical for turning small changes in air pressure (sound) into electrical signals in the brain, and TMPRSS3 protein is necessary for their function and survival. TMPRSS3 gene mutations cause hair cell loss and loss of hearing. Some individuals with mutations in this gene are born with profound deafness (DFNB-10). Others experience a delayed onset of hearing loss (DFNB-8). Both groups will have significant difficulty or are completely unable to hear sounds. As it’s genetic, the condition often runs in families, and affected individuals often require interventions like hearing aids or cochlear implants to aid their hearing.
Learn More About DFNB-8