Drugs in Dev.
Genetic Disease
Phase II
Canada 
Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : SAT-3247
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study
Details : SAT-3247 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Muscular Dystrophy, Duchenne.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 10, 2025
Lead Product(s) : SAT-3247
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : TTI-0102
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
Details : TTI-0102 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of MELAS Syndrome.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
October 16, 2024
Lead Product(s) : TTI-0102
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Psilocybine
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : KGK Science
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Psilocybin is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Fragile X Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 27, 2023
Lead Product(s) : Psilocybine
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : KGK Science
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : XRx-008 (oxypurinol) inhibits the enzyme xanthine oxidase, blocking the conversion of the oxypurines hypoxanthine and xanthine to uric acid, which results in a decrease in the concentrations of uric acid in the blood and urine.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 21, 2023
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
XORTX Announces Completion of Dosing in XRX-OXY-101 Clinical Study
Details : XORTX’s lead program XRx-008 is designed to deliver the xanthine oxidase inhibitor (“XOI”) oxypurinol at concentrations sufficient to substantially inhibit aberrant purine metabolism in the kidneys of individuals with progressing kidney disease due...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 19, 2022
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
XORTX Announces New Proof of Concept Data in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Details : XORTX’s XRx-008 therapeutic development program for ADPKD, is advancing a proprietary composition of xanthine oxidase inhibitor, to manage aberrant purine metabolism and chronically high serum uric acid concentration.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 08, 2022
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
XORTX Announces Completion of Screening and Enrollment in Bridging Pharmacokinetics Study
Details : XRx-008 is designed to deliver xanthine oxidase inhibitor oxypurinol at concentrations sufficient to substantially inhibit aberrant purine metabolism in kidney of individuals with progressing kidney disease due to ADPKD inhibit production of uric acid in...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
November 28, 2022
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Fenretinide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : LAU-7b (oral fenretinide) is a novel candidate that acts on cell membrane lipids to modulate inflammation signaling and protein trafficking. Fenretinide was shown to correct the levels of certain membrane phospholipids and sphingolipids in multiple in vi...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
November 03, 2022
Lead Product(s) : Fenretinide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : QBKPN SSI
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : National Research Council of Canada Industrial Research Assistance Program
Deal Size : $6.6 million
Deal Type : Funding
Details : QBKPN SSI is designed to reprogram the functional competence of immune cells to fix the immune dysregulation that typically develops with aging, providing broad immune protection, not only against COVID-19.
Product Name : QBKPN SSI
Product Type : Vaccine
Upfront Cash : Undisclosed
October 17, 2022
Lead Product(s) : QBKPN SSI
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : National Research Council of Canada Industrial Research Assistance Program
Deal Size : $6.6 million
Deal Type : Funding

Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass.
Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Alliance Global Partners
Deal Size : $5.0 million
Deal Type : Public Offering
XORTX Announces US$5 Million Public Offering
Details : The offering will support to increase the liquidity and raise capital to further develop and advance the pipeline of product candidates, including the conduct of clinical studies and initiation of a registration trial (Phase 3) for lead program XRx-008 h...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Undisclosed
October 04, 2022
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Alliance Global Partners
Deal Size : $5.0 million
Deal Type : Public Offering
