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[{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Z Factor","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Z Factor Doses First Subject with ZF874, a Potential Disease-Modifying Treatment for Alpha-1-Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Z Factor"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase I Trial for Novel Cystic Fibrosis Therapy ETD001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Mission Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Syncona","pharmaFlowCategory":"D","amount":"$28.3 million","upfrontCash":"$28.3 million","newsHeadline":"Syncona to Acquire Freeline Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Panakes Partners","pharmaFlowCategory":"D","amount":"$33.1 million","upfrontCash":"Undisclosed","newsHeadline":"Enterprise Therapeutics Closes \u00a326 million ($33.1 million) Series B Follow-on Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"}]
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Details:
The financing will fund the Phase 2a clinical trial of the Company’s lead programme, ETD001, a novel first in class ENaC blocker, to deliver clinical proof of concept to treat cystic fibrosis.
Lead Product(s):
ETD001
Therapeutic Area: Genetic Disease
Product Name: ETD001
Highest Development Status: Phase I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Panakes Partners
Deal Size: $33.1 million
Upfront Cash: Undisclosed
Deal Type: Series B Financing
January 30, 2024
Details:
Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in Phase 1/2 clinical trial in people with Gaucher disease type 1.
Lead Product(s):
FLT201
Therapeutic Area: Genetic Disease
Product Name: FLT201
Highest Development Status: Phase I
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Syncona
Deal Size: $28.3 million
Upfront Cash: $28.3 million
Deal Type: Acquisition
November 22, 2023
Details:
FLT201 is an adeno-associated virus (AAV) gene therapy enhacer of GCase level that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.
Lead Product(s):
FLT201
Therapeutic Area: Genetic Disease
Product Name: FLT201
Highest Development Status: Phase I
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
May 22, 2023
Details:
MTX652 potently and specifically inhibits USP30 with the aim of enabling appropriate degradation of dysfunctional mitochondria to preserve overall mitochondrial quality and improve cellular health.
Lead Product(s):
MTX652
Therapeutic Area: Genetic Disease
Product Name: MTX652
Highest Development Status: Phase I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
January 05, 2023
Details:
By increasing the amount of airway fluid available to hydrate mucus, ETD001 addresses the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life.
Lead Product(s):
ETD001
Therapeutic Area: Genetic Disease
Product Name: ETD001
Highest Development Status: Phase I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 14, 2021
Details:
First human volunteer was dosed with ZF874, company's novel treatment for alpha-1-antitrypsin deficiency (AATD). ZF874 is a novel compound that acts as a molecular ‘patch’ for the faulty protein, allowing it to fold correctly.
Lead Product(s):
ZF874
Therapeutic Area: Genetic Disease
Product Name: ZF874
Highest Development Status: Phase I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 19, 2020
Details:
ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).
Lead Product(s):
ETD002
Therapeutic Area: Genetic Disease
Product Name: ETD002
Highest Development Status: Phase I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 17, 2020