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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.
The exclusive supply agreement is for the Company’s proprietary topical products that have been commercialized in the Canadian market under the RHO Phyto brand, including AVCN-583601, Ultra-CBD cream, a CBD localized cream developed to target dermatology conditions.
Under the agreement, XOMA has acquired the royalty and milestone rights associated with two assets from LadRx Corporation, BRX-345 (arimoclomol), an oral therapeutic for Niemann-Pick disease type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.
Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.
The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.
The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.
The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.
The partnership will provide Mandos Health with real-world evidence that will directly inform the development of its Niemann-Pick type C1 (NPC1) investigational product, VTS-270 (adrabetadex).
The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.
The proceeds will fund research and clinical development of Disc's candidates including, DISC-1459 (bitopertin), a first-in-class, oral small molecule inhibitor of glycine transporter 1, to treat erythropoietic porphyrias, a rare, genetically-driven hematologic diseases.