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Medicines"},{"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Verve Therapeutics Highlights Preclinical Data Supporting Nomination of VERVE-201 ANGPTL3 Product Candidate at the European Society of Cardiology 2022 Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Verve Therapeutics"},{"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BrainVectis, a subsidiary of AskBio, Receives Clearance to Conduct Phase I\/II clinical trial in France for Its Novel Gene Therapy for Early-stage Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"AskBio"},{"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus to Present New Data on Its Amyloidosis Portfolio at the 2022 International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"iECURE Receives FDA Rare Pediatric Disease Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"iECURE"},{"company":"VectorY","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"VectorY to Present Poster on Huntington\u2019s Preclinical Data at the Hereditary Disease Foundation, August 24-27 in Cambridge, MA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"VectorY"},{"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Castle Creek Biosciences Announces Publication of Preclinical Study for Potential In Vivo Gene Therapy in Nature Communications","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Castle Creek Biosciences"},{"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Charles River Laboratories, Inc"},{"company":"iECURE","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"iECURE Receives FDA Orphan Drug Designation for GTP-506, an Investigational Gene Editing Product Candidate for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"iECURE"},{"company":"Attralus","sponsor":"University of Tennessee Graduate School of Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus Announces New Preclinical Data Demonstrating Novel Human Chimeric Antigen Receptor-Macrophages (CAR-M) for Potential Amyloid Removal at 18th International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus Presents In Vivo Preclinical Data for AT-02 Demonstrating Significant Amyloid Reduction at 18th International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Moderna Reviews Clinical Trial Programs Across Portfolio at 2022 R&D Day","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Vaccine","graph2":"Moderna Therapeutics"}]
Details:
Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe.
Lead Product(s):
AAV-based gene therapy
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
AavantiBio
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Partnership
April 27, 2021
Details:
A new development candidate, mRNA-3139, for ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder, uses the same lipid nanoparticles (LNP) as in Moderna's GSD1a program and is the Company's sixth rare disease candidate.
Lead Product(s):
mRNA-3139
Therapeutic Area: Genetic Disease
Product Name: mRNA-3139
Highest Development Status: Preclinical
Product Type: Vaccine
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 08, 2022
Details:
AT-04 is a fusion of our pan-amyloid removal (PAR) peptide technology with Fc component of an IgG1 antibody. The PAR-peptide mediates binding to all types of amyloid and the Fc stimulates immune system to remove amyloid.
Lead Product(s):
AT-04
Therapeutic Area: Genetic Disease
Product Name: AT-04
Highest Development Status: Preclinical
Product Type: Large molecule
Partner/Sponsor/Collaborator:
University of Tennessee Graduate School of Medicine
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 07, 2022
Details:
The collaboration will leverage Charles River’s market leading expertise in plasmid DNA production, specifically HQ plasmid, which combines key features of GMP manufacture with rapid turnaround times to accelerate the timeline to clinic
Lead Product(s):
Undisclosed
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Cure AP-4
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Collaboration
September 06, 2022
Details:
GTP-506 comprises two vectors, an ARCUS® nuclease vector (GTP-506A) targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector that inserts the OTC gene.
Lead Product(s):
GTP-506A,GTP-506D
Therapeutic Area: Genetic Disease
Product Name: GTP-506
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 06, 2022
Details:
BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein.
Lead Product(s):
BV-101
Therapeutic Area: Genetic Disease
Product Name: BV-101
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Bayer AG
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 23, 2022
Details:
GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC deficiency, a rare urea cycle disorder.
Lead Product(s):
GTP-506A,GTP-506D
Therapeutic Area: Genetic Disease
Product Name: GTP-506
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
August 23, 2022
Details:
VecTabs, which are secreted- or intracellular-antibody fragments that are AAV-vectorized and delivered to neurons and/or astrocytes, have previously been shown to efficiently target such misfolded proteins (TDP-43) and toxic lipids (oxPL).
Lead Product(s):
AAV Vector
Therapeutic Area: Genetic Disease
Product Name: VecTabs
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
August 23, 2022
Details:
VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride metabolism, with a precise A-to-G base pair DNA change.
Lead Product(s):
VERVE-201
Therapeutic Area: Genetic Disease
Product Name: VERVE-201
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
August 22, 2022
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