Genetic Disease | IND Enabling Drugs | UNITED STATES

Sigilon Therapeutics

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Sigilon Therapeutics Announces $80.3 Million Series B Financing to Advance

Lead Product(s): SIG-001

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Undisclosed

Partner/Sponsor/Collaborator: Canada Pension Plan Investment Board

Deal Size: $80.3 million Upfront Cash: Undisclosed

Deal Type: Series B Financing March 17, 2020

Details:

The funding will support the first-in-human clinical trial of Sigilon’s novel encapsulated cell therapy for hemophilia A.

LogicBio Therapeutics

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LogicBio Therapeutics Provides Update on FDA Review of LB-001 NDA

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 February 10, 2020

Details:

The Company submitted the IND in January 2020 to support the initiation of a Phase 1/2 clinical trial in patients with MMA.

Passage Bio

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Gene Therapy Developer Passage Bio Files for $125M IPO

Lead Product(s): PBGM01

Therapeutic Area: Genetic Disease

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: J.P. Morgan

Deal Size: $125.0 million Upfront Cash: Undisclosed

Deal Type: Public Offering February 03, 2020

Details:

Passage Bio intend to use the net proceeds to advance its lead products in clinical trials, to advance its discovery and candidate selection stage programs, and for general corporate purposes.

GeneTx Biotherapeutics

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GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

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.

BioMarin Pharm

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BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy

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.

Aeglea BioTherapeutics

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Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)

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.

LogicBio Therapeutics

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LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Hig...

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).

Prevail Therapeutics

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Prevail Therapeutics Provides PR001 Program Update

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.

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Sigilon Therapeutics

Sigilon Therapeutics Announces $80.3 Million Series B Financing to Advance

LogicBio Therapeutics

LogicBio Therapeutics Provides Update on FDA Review of LB-001 NDA

Passage Bio

Gene Therapy Developer Passage Bio Files for $125M IPO

GeneTx Biotherapeutics

GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

BioMarin Pharm

BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy

Aeglea BioTherapeutics

Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)

LogicBio Therapeutics

LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Hig...

Prevail Therapeutics

Prevail Therapeutics Provides PR001 Program Update