Find FDA Investigational New Drug (IND) Submissions for Genetic Disease in UNITED STATES

Lead Product(s): GTX-102

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 15, 2020

Details:

GeneTx’s Phase 1/2 study, named KIK-AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020.

Lead Product(s): BMN 307

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 13, 2020

Details:

BMN 307 represents a potential third PKU treatment option from BioMarin and its second gene therapy clinical program and actively preparing regulatory submissions.

Lead Product(s): L-Homocysteine

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 13, 2020

Details:

Aeglea BioTherapeutics expects to initiate a Phase 1/2 trial in the second quarter of 2020.

Lead Product(s): LB-001

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 10, 2020

Details:

LogicBio Therapeutics has submitted an IND application with the U.S. FDA to initiate a Phase 1/2 trial of LB-001, for the treatment of methylmalonic acidemia (MMA).

Lead Product(s): PR001

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 09, 2020

Details:

Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020.