Drug intelligence / Profile preview

GA-002

Development stage
Preclinical
Lead developer
Genosera
Modality
Gene Therapies
01

Overview

**GA-002** is a preclinical bicistronic adeno-associated virus gene therapy originally described by Genosera and Solve GNE for **GNE myopathy**, also known as hereditary inclusion body myopathy. The vector is intended to deliver a functional copy of the **GNE** gene together with an unspecified muscle growth or regenerative-factor gene. This dual-gene strategy is designed both to correct the underlying GNE deficiency and to promote restoration of muscle mass and strength in already affected muscle. The program originated from dual-gene AAV research at Nationwide Children’s Hospital. ([biospace.com](https://www.biospace.com/solve-gne-and-genosera-sign-agreement-to-develop-a-novel-bicistronic-aav-gene-therapy-targeting-hibm))

Other names
GA 002GA002GA-002
02

Targets

LATS1LATS2 (Large tumor suppressor kinase 2)MSK1 (Mitogen- and stress-activated protein kinase 1)PKA (Cyclic amp-dependent protein kinase)PRKACB (cAMP-dependent protein kinase catalytic subunit beta)SGK2 (Serum/glucocorticoid regulated kinase 2)SGK3 (Serum/glucocorticoid regulated kinase family member 3)RPS6KB1 (Ribosomal protein S6 kinase beta-1)GNE (UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase)

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