Myrtelle

Website

Myrtelle Inc.

1 Investors
Gene Therapy
NEW YORK, NY

Myrtelle Inc. is a clinical-stage gene therapy company carrying the mission of developing innovative treatments for neurodegenerative diseases that primarily affect myelin and white matter in the brain. With a particular focus on Canavan disease, the company addresses critical gaps in treatment options for rare genetic disorders of the central nervous system, aiming to improve patient outcomes significantly.

Products & Team

rAAV-Olig001-ASPA

Gene TherapySeed

rAAV-Olig001-ASPA is an investigational gene therapy specifically designed to treat Canavan disease, a severe childhood genetic disorder characterized by the degeneration of white matter in the brain.

Value Proposition

This therapy addresses the urgent need for effective treatments for Canavan disease, moving beyond palliative care to potentially halt and reverse neurological decline.

Pain Points

Families face a dire lack of effective treatment options, with existing therapies mainly providing palliative care without addressing the underlying genetic cause.

Utilizes proprietary adeno-associated virus (AAV) vector technologyDelivers a functional copy of the ASPA gene directly to oligodendrocytesAdministered via intracerebroventricular injection for effective brain cell targetingEarly trials showing reductions in disease biomarkers and improvements in white matter
Industry
Biotechnology
Primary business sector
Founded
2025
Year established
Location
NEW YORK, NY
Primary headquarters

Funding History

Total Raised:
$7.5M
E

Debt Financing Offering

March 2025
$15.0M
Target
Use of Proceeds
While no offering proceeds are set aside for payments to the named officers or directors, it is possible that some proceeds to be used as working capital will be used indirectly for paying salaries to employees including executive officers.
Progress
50%
Raised
$7.5M
Target
$15.0M
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