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

Loading...

All Data

Filters Filter refresh
×
FILTER DATA :
News Type filter
    Company filter
      Product Type filter
        Deal Size filter
          Upfront Payment filter
            refresh

            Product Type Full Screen

            Companies Full Screen

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): TSHA-101

            Therapeutic Area: Genetic Disease Product Name: TSHA-101

            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 August 27, 2020

            Details:

            Taysha Gene Therapies has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA for TSHA-101, an AAV9-based gene therapy in development for GM2 Gangliosidosis.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): EDIT-301

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            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 August 24, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for sickle cell disease.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): AB-G023

            Therapeutic Area: Genetic Disease Product Name: AB-G023

            Highest Development Status: IND Enabling Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 20, 2020

            Details:

            US Food and Drug Administration Offices of Pediatric Therapeutics and Orphan Products Development granted a rare pediatric disease designation to AmideBio’s glucagon analog (AB‐G023) for the treatment of congenital hyperinsulinism (CHI).

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): ABO-202

            Therapeutic Area: Genetic Disease Product Name: ABO-202

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

            Partner/Sponsor/Collaborator: Taysha Gene Therapies

            Deal Size: $63.0 million Upfront Cash: $3.0 million

            Deal Type: Licensing Agreement August 17, 2020

            Details:

            The agreements grant Taysha worldwide exclusive rights to intellectual property developed by scientists at the University of North Carolina at Chapel Hill (UNC) and Abeona, and Abeona know-how relating to the research, development, and manufacture of ABO-202.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            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: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 13, 2020

            Details:

            FDA has granted Fast Track designation to 4D-310 for Fabry disease. 4D-310 is a gene medicine engineered with a proprietary optimized AAV capsid discovered by 4DMT through its proprietary Therapeutic Vector Evolution platform.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): BB-301

            Therapeutic Area: Genetic Disease Product Name: BB-301

            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 July 23, 2020

            Details:

            The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall goal of providing data to submit an investigational new drug application to the U.S. Food and Drug Administration, a mandatory step to conduct clinical trials.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): FBX-101

            Therapeutic Area: Genetic Disease Product Name: FBX-101

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

            Partner/Sponsor/Collaborator: Perceptive Xontogeny Venture Fund

            Deal Size: $40.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing July 21, 2020

            Details:

            Proceeds to expand Contract Development and Manufacturing Organization (CDMO) capacity to deliver GMP adeno-associated viruses (AAV) product for clients by mid-2021 and to develop a proprietary pipeline of novel gene therapies.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): INZ-701

            Therapeutic Area: Genetic Disease Product Name: INZ-701

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Inozyme Pharma

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition July 17, 2020

            Details:

            The acquisition complements the ongoing development of Inozyme’s lead product candidate, INZ-701, which Inozyme is investigating for the treatment of ENPP1 and ABCC6 deficiencies, which are rare diseases of abnormal mineralization.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): AAV-based gene therapy

            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 July 15, 2020

            Details:

            Dr. Huttner to lead clinical development as Neurogene advances towards first Investigational New Drug (IND) filing.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): EDIT-301

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

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

            Partner/Sponsor/Collaborator: Editas Medicine

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement July 08, 2020

            Details:

            Editas Medicine will utilize the space and Azzur’s services to execute pre-clinical and early-phase clinical manufacturing activities for its cell medicines, including EDIT-301 in development for the treatment of sickle cell disease and beta thalassemia and EDIT-201.

            PharmaCompass