Genetic Disease | IND Enabling Drugs | UNITED STATES

HemoShear Therapeutics, LLC

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HemoShear Therapeutics Receives FDA Clearance of IND for Phase 2 Study of its Investigational Drug HST5040

Lead Product(s): HST5040

Therapeutic Area: Genetic Disease Product Name: HST5040

Highest Development Status: IND Enabling Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 24, 2020

Details:

The U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to conduct a phase 2 clinical trial of HST5040, an oral small molecule drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA).

4D Molecular Therapeutics

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4D Molecular Therapeutics Raises $75 Million in Series C Financing

Lead Product(s): 4D-310

Therapeutic Area: Genetic Disease Product Name: 4D-310

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Viking Global Investors

Deal Size: $75.0 million Upfront Cash: Undisclosed

Deal Type: Series C Financing June 16, 2020

Details:

Funds to be used to advance 3 precision-guided AAV gene therapy candidates through initial clinical proof-of-concept, to advance pre-clinical product pipeline, proprietary next-generation Therapeutic Vector Evolution platform, and to expand GMP manufacturing capabilities.

Beam Therapeutics Inc

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Magenta's Technology Attracts Another Gene Therapy Developer Beam Therapeutics

Lead Product(s): MGTA-117

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Large molecule

Partner/Sponsor/Collaborator: Magenta Therapeutics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration June 15, 2020

Details:

This non-exclusive research and clinical collaboration agreement aims to evaluate the potential utility of MGTA-117, Magenta’s novel targeted ADC for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beam’s base editing therapies.

Editas Medicine

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Editas Medicine Presents Pre-Clinical Data from a Study of EDIT-301 with Sickle Patient Cells for Potential Treatment of Sickle Cell Disease

Lead Product(s): EDIT-301

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 12, 2020

Details:

EDIT-301 contains CD34+ hematopoietic stem cells from sickle patients that are edited at the HBG1/2 promoter in the beta-globin locus using Cas12a (also known as Cpf1) to induce fetal hemoglobin (HbF) where HbF-inducing mutations occur naturally.

Passage Bio

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Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis

Lead Product(s): PBGM01

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 21, 2020

Details:

USFDA has granted Rare Pediatric Disease designation to PBGM01 broadly for the treatment of GM1 gangliosidosis. PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis.

Inozyme Pharma

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Inozyme and GACI Global Initiate Study to Understand Severity and Impact of Disease in Patients with ENPP1 Deficiency and ABCC6 Deficiency

Lead Product(s): INZ-701

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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 May 14, 2020

Details:

Inozyme is preparing to seek regulatory approval to initiate a clinical trial in patients with ENPP1 deficiency and a separate clinical trial in patients with ABCC6 deficiency.

LogicBio Therapeutics

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LogicBio Therapeutics to Present New Data on GeneRide™ Platform and Next Generation Capsid Development Program at ASGCT 2020

Lead Product(s): LB-001

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 11, 2020

Details:

LogicBio's presentation will highlight results of treatment of Juvenile Mice with methylmalonic Acidemia (MMA) by targeted integration of MMUT into Albumin using a Promoterless AAV Vector.

Taysha Gene Therapies

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Taysha Gene Therapies Announce $30 Million Seed Financing and Strategic Partnership with the UT Southwestern Gene Therapy Program

Lead Product(s): TGTX-101

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: PBM Capital

Deal Size: $30.0 million Upfront Cash: Undisclosed

Deal Type: Financing April 29, 2020

Details:

Financing would assist in meeting Taysha's goal of advancing its 15 AAV gene therapy programs pipeline with options for another four programs targeting central nervous system diseases.

4D Molecular Therapeutics

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4D Molecular Therapeutics Announces Upcoming Oral and Poster Presentations at the American Society of Gene and Cell Therapy Meeting

Lead Product(s): 4D-310

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 29, 2020

Details:

Oral presentations to include data on 4D-310 Product Candidate for Fabry Disease and 4D-A101 Aerosol Vector for Cystic Fibrosis and other Lung Diseases.

AmideBio

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U.S. FDA Grants Orphan Drug Designation to Amidebio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism

Lead Product(s): AB-G023

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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 April 23, 2020

Details:

AB-G023 is a solution stable, soluble glucagon analog designed to overcome the limitations of glucagon. Glucagon is an effective treatment for congenital hyperinsulinism, but rendered impractical for long term administration given its instability in solution.

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

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HemoShear Therapeutics, LLC

HemoShear Therapeutics Receives FDA Clearance of IND for Phase 2 Study of its Investigational Drug HST5040

4D Molecular Therapeutics

4D Molecular Therapeutics Raises $75 Million in Series C Financing

Beam Therapeutics Inc

Magenta's Technology Attracts Another Gene Therapy Developer Beam Therapeutics

Editas Medicine

Editas Medicine Presents Pre-Clinical Data from a Study of EDIT-301 with Sickle Patient Cells for Potential Treatment of Sickle Cell Disease

Passage Bio

Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis

Inozyme Pharma

Inozyme and GACI Global Initiate Study to Understand Severity and Impact of Disease in Patients with ENPP1 Deficiency and ABCC6 Deficiency

LogicBio Therapeutics

LogicBio Therapeutics to Present New Data on GeneRide™ Platform and Next Generation Capsid Development Program at ASGCT 2020

Taysha Gene Therapies

Taysha Gene Therapies Announce $30 Million Seed Financing and Strategic Partnership with the UT Southwestern Gene Therapy Program

4D Molecular Therapeutics

4D Molecular Therapeutics Announces Upcoming Oral and Poster Presentations at the American Society of Gene and Cell Therapy Meeting

AmideBio

U.S. FDA Grants Orphan Drug Designation to Amidebio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism