In addition to the clinical research underway on MYO-101 (see announcement), and this designation for MYO-102, the other candidates include MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L.
Myonexus Therapeutics' pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L).
Additionally, Myonexus is advancing MYO-102 for LGMD2D, MYO-103 for LGMD2C
, MYO-201 for LGMD2B, and MYO-301 for LGMD2L.
The 361 non-collagen VI-related myopathy cases included 166 cases of dystrophinopathies, 56 cases of inflammatory myopathy, 35 cases of neurogenic muscular atrophy, 33 cases of LGMD2B, 15 cases of congenital myopathy, 15 cases of LGMD2A, 13 cases of metabolic myopathy, 8 cases of LMNA-related myopathy, 7 cases of GNE myopathy, 5 cases of myotonic dystrophy, 3 cases of LGMD2D, 3 cases of LGMD2I, 1 case of LGMD2C
, and 1 case of LGMD2Q.
Using the Motor Chip to investigate a panel of NMD patients with no previous molecular characterization, we identified the first case of limb girdle muscular dystrophy type 2C (LGMD2C) caused by 2 distinct deletions that inactivate both alleles of the SGCG [sarcoglycan, gamma (35kDa dystrophin-associated glycoprotein)] gene.
Analysis with the Motor Chip revealed this individual to be compound heterozygous for 2 different wide deletions involving SGCG, which caused LGMD2C (34).
We also provided the molecular diagnosis of LGMD2C for patient no.
Homozygous contiguous gene deletion of 13q12 causing LGMD2C and ARSACS in the same patient.
The company's pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C
Myonexus' pipeline also includes MYO-102, a gene therapy candidate for LGMD2D (alpha-sarcoglycanopathy) currently completing a Phase 1/2a clinical trial, MYO-201, a gene therapy candidate for LGMD2B (dysferlinopathy) currently in Phase 1, MYO-103, a preclinical gene therapy candidate for LGMD2C (gamma-sarcoglycanopathy), and MYO-301, a preclinical gene therapy candidate for LGMD2L (anoctamin 5).
The company's pipeline includes three clinical stage gene therapy programmes (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programmes (LGMD2C and LGMD2LO).