Home/Pipeline/GMAC for Fabry Disease

GMAC for Fabry Disease

Fabry Disease

PreclinicalJoint development with Kyorin Pharmaceutical

Key Facts

Indication

Fabry Disease

Phase

Preclinical

Status

Joint development with Kyorin Pharmaceutical

Company

Japanese biotech developing one-time ex-vivo gene therapies using genetically modified human adipocytes for rare genetic diseases.

Drug	Company	Phase	Status
Corrector Program	Octant Biotech	Hit to Lead	Active
JR-171 (lepunafusp alfa)	JCR Pharmaceuticals	Phase 3	Active
Undisclosed Gene Therapy	uniQure	Preclinical	Active
Lucerastat	Idorsia	Phase 3	Development status post-prioritization
PRX–115 (pegunigalsidase alfa)	Protalix BioTherapeutics	Submitted	BLA Submitted
Isaralgagene civaparvovec (ST-920)	Sangamo Therapeutics	Phase 1/2	Active, BLA Rolling Submission