Drugs in Dev.
Genetic Disease
Phase I
United Kingdom 
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Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 14, 2025
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 11, 2025
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Florbetaben
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : pharmtrace
Deal Size : Inapplicable
Deal Type : Inapplicable
Test-retest Study With [18F]FBB in Cardiac Amyloidosis
Details : Florbetaben is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Amyloidosis, Hereditary, Transthyretin-Related.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
January 24, 2025
Lead Product(s) : Florbetaben
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : pharmtrace
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : HLX-1502
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : R42 Group
Deal Size : $47.0 million
Deal Type : Series C Financing
Healx Announces $47M Series C and FDA Clearance for Neurofibromatosis Type 1 Trial
Details : The net proceeds will be used to advance the company's lead program HLX-1502 through a mid-stage clinical trial studyies for the treatment of patients with neurofibromatosis Type 1.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Undisclosed
August 01, 2024
Lead Product(s) : HLX-1502
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : R42 Group
Deal Size : $47.0 million
Deal Type : Series C Financing

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Lead Product(s) : Eplontersen
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
Details : Eplontersen is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Amyloidosis, Hereditary, Transthyretin-Related.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
July 30, 2024
Lead Product(s) : Eplontersen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
July 25, 2024
Lead Product(s) : KER-0193
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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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 : Undisclosed
Product Type : Miscellaneous
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

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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 : Undisclosed
Product Type : Cell & 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

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Lead Product(s) : AUT00201
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK
Details : AUT00201 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Myoclonic Epilepsies, Progressive.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 24, 2023
Lead Product(s) : AUT00201
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 22, 2023
Lead Product(s) : FLT201
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
