Lead Product(s) : Afamelanotide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Clinuvel Trial Results Show Drug Reduces DNA Damage
Details : Scenesse® (afamelanotide) belongs to the family of melanocortins, hormones, which activates human pigmentation, reduce oxidative damage, inflammation and swelling, and optimise the response of skin cells to UV-induced damage. It is being investigated fo...
Product Name : Scenesse
Product Type : Peptide
Upfront Cash : Inapplicable
January 16, 2023
Lead Product(s) : Afamelanotide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Pentosan Polysulfate Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Three of the four subjects included in Paradigm's open-label phase 2 trial of pentosan polysulfate sodium (PPS) in MPS-I have completed the 48-week study, with a 6-month treatment extension available.
Product Name : Zilosul
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
December 08, 2022
Lead Product(s) : Pentosan Polysulfate Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Antisense Therapeutics to Present Poster at World’s Largest NMD Conference
Details : ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4. Antisense inhibition of VLA-4 expression has demonstrated activity in animal models of inflammatory disease. ATL1102 has also shown to be very effective in reducing inflammatory brain lesion...
Product Name : ATL1102
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
September 03, 2022
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Vamifeport
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
First Patient Enrolled in Phase-IIa Study of Vamifeport in Patients with Sickle Cell Disease
Details : VIT-27639 (Vamifeport), is the first oral ferroportin inhibitor investigated for treatment of diseases with ineffective production of red blood cells and iron overload such as sickle cell disease.
Product Name : VIT-2763
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
October 12, 2021
Lead Product(s) : Vamifeport
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The poster presentation includes the recently reported data from the Phase II clinical trial of the Company’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) where ATL1102 met the primary endpoint.
Product Name : ATL1102
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
November 06, 2020
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Primary endpoint met with confirmation of drug’s safety and tolerability. Strong effects on secondary endpoints on activity markers and disease progression along with improvement or stabilisation across different measures of motor function & strength w...
Product Name : ATL1102
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
May 21, 2020
Lead Product(s) : ATL1102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable