Drugs in Dev. Genetic Disease
Phase I
Australia 
Lead Product(s) : PYC-003
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
Details : PYC-003 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
December 03, 2024
Lead Product(s) : PYC-003
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : PYC-001
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
Details : PYC-001 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Optic Atrophy, Autosomal Dominant.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 14, 2024
Lead Product(s) : PYC-001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : VP-001
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
Details : VP-001 is a Oligonucleotide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Retinitis Pigmentosa.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 12, 2024
Lead Product(s) : VP-001
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : KIO-301
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Kiora Pharmaceuticals
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : KIO-301 acts as a reversible 'photoswitch', specifically designed to restore the eyes' ability to perceive and interpret light in visually impaired patients with retinitis pigmentos.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
February 14, 2023
Lead Product(s) : KIO-301
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Kiora Pharmaceuticals
Deal Size : Inapplicable
Deal Type : Inapplicable
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