Genetic Disease | IND Enabling Drugs | UNITED STATES

Taysha Gene Therapies

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Taysha Gene Therapies Receives Orphan Drug Designation and Rare Pediatric Disease Designation for TSHA-101 for GM2 Gangliosidosis

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.

Editas Medicine

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Editas Medicine Receives Rare Pediatric Disease Designation For EDIT-301 For The Treatment Of Sickle Cell Disease

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.

AmideBio

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

U.S. FDA Grants Rare Pediatric Disease Designation to Amide Bio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism

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).

Abeona Therapeutics

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202

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.

4D Molecular Therapeutics

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease

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.

Benitec Biopharma

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

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.

Forge Biologics

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Forge Debuts with $40M and Tim Miller at the Helm

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.

Alexion Pharmaceuticals

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Inozyme Pharma Acquires ENPP1 Deficiency Program Assets from Alexion Pharmaceuticals

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.

Neurogene

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Neurogene Appoints Kenneth Huttner, M.D., Ph.D., as Senior Vice President of Clinical Development

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.

Azzur Group

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

United States

Post An Enquiry

Virtual Booth

ESMO 2020 Congress

Participation Not Confirmed

Update your events

Editas Medicine and Azzur Group Announce a Multi-year Agreement for EDIT-301 and EDIT-201 Manufacturing

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.

Find

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

United States

Product Type

Companies

Taysha Gene Therapies

Taysha Gene Therapies Receives Orphan Drug Designation and Rare Pediatric Disease Designation for TSHA-101 for GM2 Gangliosidosis

Editas Medicine

Editas Medicine Receives Rare Pediatric Disease Designation For EDIT-301 For The Treatment Of Sickle Cell Disease

AmideBio

U.S. FDA Grants Rare Pediatric Disease Designation to Amide Bio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism

Abeona Therapeutics

Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202

4D Molecular Therapeutics

4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease

Benitec Biopharma

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

Forge Biologics

Forge Debuts with $40M and Tim Miller at the Helm

Alexion Pharmaceuticals

Inozyme Pharma Acquires ENPP1 Deficiency Program Assets from Alexion Pharmaceuticals

Neurogene

Neurogene Appoints Kenneth Huttner, M.D., Ph.D., as Senior Vice President of Clinical Development

Azzur Group

Editas Medicine and Azzur Group Announce a Multi-year Agreement for EDIT-301 and EDIT-201 Manufacturing