ACGENETIC Therapeutics is developing two distinct, variant-directed approaches to address the underlying genetic causes of LGMD2B/R2. Our ex vivo strategy generates gene-corrected muscle satellite cells from iPSCs for intramuscular (IM) administration, while our in vivo strategy delivers gene-editing components intravenously (IV) to directly correct endogenous muscle satellite cells. These approaches may provide a foundation for future expansion into other inherited disorders.
We remain committed to advancing research toward new therapeutic approaches for inherited muscle disorders
ACGENETIC was founded by a patient with LGMD2B/R2 to pursue new therapeutic approaches for inherited muscle disorders. His family’s shared experience with the disease further strengthens the company’s long-term dedication to this research.
Our ex vivo strategy focuses on differentiating gene-corrected iPSCs into muscle satellite cells for intramuscular (IM) administration
We evaluate whether muscle satellite cells derived from gene-corrected iPSCs can engraft and contribute to the formation of dysferlin-expressing myofibers
Our in vivo strategy delivers LNP-encapsulated gene-editing components intravenously (IV), aiming to achieve transient gene-editor expression and directly edit endogenous muscle satellite cells
We evaluate transient gene-editor expression following intravenous LNP delivery, along with biodistribution, immunogenicity, redosing potential, and genomic integrity