Drugs in Dev. Genetic Disease
Preclinical
Spain 
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Columbus Venture Partners
Deal Size : $46.4 million
Deal Type : Series B Financing
Details : The Series B proceeds will be used to advance company's lead compound, ATX-01 (amitriptyline hydrochloride), an antimiR designed to target microRNA 23b (miR-23b), to a Phase I/IIa clinical trial for the treatment of Myotonic Dystrophy Type 1 (DM1).
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
May 03, 2023
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Columbus Venture Partners
Deal Size : $46.4 million
Deal Type : Series B Financing
Lead Product(s) : ORY-4001
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ORYZON Announces Clinical Candidate Nomination of ORY-4001 for the Treatment of CNS Disorders
Details : ORY-4001 is a selective histone deacetylase 6 (HDAC-6) inhibitor, as clinical development candidate for the treatment of certain neurological diseases as Charcot-Marie-Tooth (CMT), Amyotrophic Lateral Sclerosis (ALS).
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 13, 2023
Lead Product(s) : ORY-4001
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : EIC Accelerator
Deal Size : $13.7 million
Deal Type : Funding
Arthex Biotech Selected for EIC Accelerator Award of up to 14 Million Euros
Details : Proceeds will be used to fund the FIGHT-DM 1 project to initiate clinical trial of Arthex' lead candidate, ATX-01, in DM1 in 2023.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
October 25, 2022
Lead Product(s) : ATX-01
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : EIC Accelerator
Deal Size : $13.7 million
Deal Type : Funding
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : CMT Research Foundation
Deal Size : Undisclosed
Deal Type : Collaboration
ORYZON Collaborates with the CMT Research Foundation in the US
Details : Under this collaboration, CMTRF will be financing a series of in vivo tests with Oryzon’s HDAC6 candidates in a murine model of CMT1A, which reliably recapitulates many of the symptoms of this condition in humans.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
July 26, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : CMT Research Foundation
Deal Size : Undisclosed
Deal Type : Collaboration
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Study Phase : Preclinical
Sponsor : University of Minnesota
Deal Size : Undisclosed
Deal Type : Licensing Agreement
SOM Biotech Signs Licensing Agreement With the University of Minnesota
Details : This agreement follows the completion of research agreement, in which the University of Minnesota conducted a series of in vitro studies on potential pre-clinical product candidates for Duchenne and Becker muscular dystrophy identified using SOM Biotechâ...
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
May 17, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Genetic Disease
Highest Development Status : Preclinical
Sponsor : University of Minnesota
Deal Size : Undisclosed
Deal Type : Licensing Agreement
Reset All
