Drugs in Dev. Genetic Disease
Phase I
United Kingdom 
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
KER-0193 Gets Orphan and Pediatric Drug Designation for Fragile X by FDA
Details : KER-0193, a novel BK channel modulator, small molecule drug candidate, which is being evaluated for the treatment of Fragile X syndrome.
Product Name : KER-0193
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
May 14, 2025
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Kaerus' KER-0193 Shows Proof of Mechanism in Phase 1 for Fragile X Syndrome
Details : KER-0193, a novel BK channel modulator, small molecule drug candidate, which is being evaluated for the treatment of Fragile X syndrome.
Product Name : KER-0193
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
March 11, 2025
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Kaerus Bioscience Initiates Phase 1 Trial for KER-0193 in Fragile X Syndrome
Details : KER-0193 is a novel, proprietary, and orally-bioavailable small molecule modulator of BK channels, being developed for patients with Fragile X Syndrome.
Product Name : KER-0193
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
July 25, 2024
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ETD001
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Panakes Partners
Deal Size : $33.1 million
Deal Type : Series B Financing
Enterprise Therapeutics Closes £26 million ($33.1 million) Series B Follow-on Financing
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.
Product Name : ETD001
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
January 30, 2024
Lead Product(s) : ETD001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Panakes Partners
Deal Size : $33.1 million
Deal Type : Series B Financing
Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Syncona Limited
Deal Size : $28.3 million
Deal Type : Acquisition
Syncona to Acquire Freeline Therapeutics
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.
Product Name : FLT201
Product Type : Cell and Gene therapy
Upfront Cash : $28.3 million
November 22, 2023
Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Syncona Limited
Deal Size : $28.3 million
Deal Type : Acquisition
Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : FLT201
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
May 22, 2023
Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : MTX652
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652
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.
Product Name : MTX652
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
May 01, 2023
Lead Product(s) : MTX652
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ETD001
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : ETD001
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 14, 2021
Lead Product(s) : ETD001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ZF874
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : ZF874
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
August 19, 2020
Lead Product(s) : ZF874
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ETD002
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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).
Product Name : ETD002
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
August 17, 2020
Lead Product(s) : ETD002
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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