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[{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"$882.0 million","upfrontCash":"$44.0 million","newsHeadline":"Evox Therapeutics and Takeda Sign Multi-target Rare Disease Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Evox Therapeutics"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Redmile Group","pharmaFlowCategory":"D","amount":"$95.4 million","upfrontCash":"Undisclosed","newsHeadline":"Evox Therapeutics Completes \u00a369.2 Million Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Evox Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","amount":"$211.5 million","upfrontCash":"$17.5 million","newsHeadline":"Orchard Therapeutics and Pharming Group Collaborate to Develop and Commercialize Ex Vivo Autologous HSC Gene Therapy for Genetic Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Oxford Biomedica Initiates New Project With Orchard Therapeutics Utilising LentiStable\u2122 Technology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Oxford Biomedica"},{"orgOrder":0,"company":"AlveoGene","sponsor":"Oxford Science Enterprises","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AlveoGene Launches to Develop Unique Inhaled Gene Therapies for Rare Respiratory Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"AlveoGene"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"ALSA Ventures","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"ALSA Ventures Launches Novel Gene Therapy Portfolio Company Axovia Therapeutics to Treat Ciliopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Axovia Therapeutics"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Preclinical Data Showing AMO-02 Improves Muscle Function, Glucose Handling and CNS Function in Mouse Models of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"Harness Therapeutics","sponsor":"Epidarex Capital","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Harness Therapeutics Secures Additional \u00a34 Million Funding to Pursue Physiological Upregulation Approach to Treat Neurodegenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Harness Therapeutics"},{"orgOrder":0,"company":"MitoRx Therapeutics","sponsor":"Wren Capital","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"MitoRx Therapeutics Announces Close of Seed Extension Financing Round to Advance its Mitochondrial-Protective Therapeutics to Treat Degenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"MitoRx Therapeutics"}]
Find Drugs for Genetic Disease in Preclinical Development in UNITED KINGDOM
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Details:
The company will use the net proceeds to develop its first-in-class therapeutic pipeline which targets mitochondria dysfunction linked to the progression of rare diseases such as the neuromuscular disorder Duchenne muscular dystrophy, Huntington’s disease.
Lead Product(s):
Undisclosed
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Wren Capital
Deal Size: $5.0 million
Upfront Cash: Undisclosed
Deal Type: Financing
December 13, 2023
Details:
The net proceeds will be used to advance Harness’ lead Huntington’s Disease (HD) programme targeting FAN1 nuclease and pursue target discovery and validation activities in other neurodegenerative disease pathways.
Lead Product(s):
Undisclosed
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Epidarex Capital
Deal Size: $5.0 million
Upfront Cash: Undisclosed
Deal Type: Financing
November 28, 2023
Details:
Axovia is developing the first gene therapies for ciliopathies and has a pipeline of products, including AXV101, an AAV9-based gene therapy targeting retinal dystrophy associated with BBS in patients carrying biallelic mutations in the BBS1 gene.
Lead Product(s):
AXV101
Therapeutic Area: Genetic Disease
Product Name: AXV101
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
ALSA Ventures
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Acquisition
September 19, 2023
Details:
AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.
Lead Product(s):
Tideglusib
Therapeutic Area: Genetic Disease
Product Name: AMO-02
Highest Development Status: Preclinical
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 19, 2023
Details:
The financing will enable AlveoGene to fast-track the development of its first candidate AVG-001, a novel, inhaled gene therapy designed to promote localised production of alpha-1 antitrypsin to treat patients with Alpha-1 Antitrypsin Deficiency (AATD).
Lead Product(s):
AVG-001
Therapeutic Area: Genetic Disease
Product Name: AVG-001
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Oxford Science Enterprises
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Financing
September 14, 2023
Details:
OTL-203, an investigational hemopoietic stem cell (HSC) gene therapy in development for the potential treatment of mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH).
Lead Product(s):
OTL-203
Therapeutic Area: Genetic Disease
Product Name: OTL-203
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Orchard Therapeutics
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
July 26, 2022
Details:
OTL-105 is an investigational HSC gene therapy designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent hereditary angioedema attacks.
Lead Product(s):
Autologous Hematopoietic Stem Cell Gene Therapy
Therapeutic Area: Genetic Disease
Product Name: OTL-105
Highest Development Status: Preclinical
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Pharming
Deal Size: $211.5 million
Upfront Cash: $17.5 million
Deal Type: Collaboration
July 01, 2021
Details:
Proceeds from this financing will support the advancement of Evox’s exosome-based therapeutics pipeline, including progression of several rare disease assets into the clinic, and continued development of its world-leading DeliverEX exosome drug platform.
Lead Product(s):
EVX-101
Therapeutic Area: Genetic Disease
Product Name: EVX-101
Highest Development Status: Preclinical
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Redmile Group
Deal Size: $95.4 million
Upfront Cash: Undisclosed
Deal Type: Series C Financing
February 18, 2021
Details:
Deal allows Evox to continue advancing its exosome-based targeting and delivery technology, while also leveraging Takeda’s development and clinical expertise to advance these partnered programmes.
Lead Product(s):
Protein therapeutics
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Takeda Pharmaceutical
Deal Size: $882.0 million
Upfront Cash: $44.0 million
Deal Type: Collaboration
March 26, 2020
Details:
Paper demonstrates first in class TMEM16A chloride channel potentiators, to accelerate mucociliary clearance.
Lead Product(s):
ETX001
Therapeutic Area: Genetic Disease
Product Name: Undisclosed
Highest Development Status: Preclinical
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 08, 2020