All Data
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): TSHA-102
Therapeutic Area: Genetic Disease Product Name: TSHA-102
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 14, 2020
Details:
U.S. Food and Drug Administration for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug application for TSHA-102 to the FDA in 2021.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): ET-01
Therapeutic Area: Genetic Disease Product Name: ET-01
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: 3H Health Investment
Deal Size: $67.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing October 13, 2020
Details:
EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. Proceeds from the financing will be used to advance the company’s pipeline into clinics.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Lentiviral gene therapy
Therapeutic Area: Genetic Disease Product Name: AVR-RD-05
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Avrobio
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement October 05, 2020
Details:
Gene therapy licensed from The University of Manchester, will be called as AVR-RD-05. Avrobio will make the therapy by modifying patient’s own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of functional enzymes.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): SPIRO-2101
Therapeutic Area: Genetic Disease Product Name: SPIRO-2101
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 24, 2020
Details:
SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): CRN04777
Therapeutic Area: Genetic Disease Product Name: CRN04777
Highest Development Status: Preclinical Product Type: Undisclosed
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 21, 2020
Details:
U.S. Food and Drug Administration has granted rare pediatric disease designation (“RPD”) for CRN04777, an investigational, orally available, nonpeptide somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital hyperinsulinism.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): AT-02
Therapeutic Area: Genetic Disease Product Name: AT-02
Highest Development Status: Preclinical Product Type: Peptide
Partner/Sponsor/Collaborator: venBio Partners
Deal Size: $25.0 million Upfront Cash: Undisclosed
Deal Type: Series A Financing September 14, 2020
Details:
The financing will allow Attralus to further validate their pan-amyloid imaging agent, AT-01, and advance two therapeutic candidates, AT-02 and AT-03, into clinical development for ATTR (transthyretin), AL (light chain) and potentially ALECT2 systemic amyloidosis.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Undisclosed
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: The Column Group
Deal Size: $50.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing September 10, 2020
Details:
Proceeds will be used to advance Casma’s First-in-Class TRPML1 agonist in muscular dystrophy and enable preclinical proof-of-concept for the Company’s novel autophagy degrader platform.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): INV-101
Therapeutic Area: Genetic Disease Product Name: INV-101
Highest Development Status: Preclinical Product Type: Undisclosed
Partner/Sponsor/Collaborator: Forbion
Deal Size: $35.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing September 08, 2020
Details:
Proceeds will be used to develop Inversago’s best-in-class peripherally-acting CB1 inverse agonist candidate INV-101 to clinical proof-of-concept, and advance the research on other selected compounds.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): AOC 1001
Therapeutic Area: Genetic Disease Product Name: AOC 1001
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: Myotonic Dystrophy Clinical Research Network
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration September 08, 2020
Details:
The collaboration focuses at supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1. END-DM1 is a non-interventional study designed and run by the Myotonic Dystrophy Clinical Research Network.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): LB-100
Therapeutic Area: Genetic Disease Product Name: LB-100
Highest Development Status: Preclinical Product Type: Small molecule
Partner/Sponsor/Collaborator: Lixte Biotechnology
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration August 18, 2020
Details:
Collaboration will support the preclinical studies of the potential benefit of Lixte’s proprietary lead clinical compound, LB-100, in a mouse model of Angelman Syndrome (AS).
Drugs in Development
Market Place
Active Pharmaceutical Ingredients
Finished Drug Formulations
Price
Excipients
Services
Marketplace
