Chiesi Group and Arbor Biotechnologies Secure the EC’s Orphan Drug Designation for ABO-101 to Treat Primary Hyperoxaluria

Shots: The EC has granted ODD to ABO-101 for the treatment of primary hyperoxaluria ABO-101 is being evaluated in an open-label global P-I/II (redePHine) study assessing safety, tolerability, PK/PD, & preliminary efficacy of a single dose of ABO-101 in pts with PH type 1 ABO-101 is a one-time, liver-directed gene-editing therapy that uses an Acuitas-licensed…

ByRidhi Rastogi5 hours ago

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Chiesi Group Enters a ~$2.1B Deal with Arbor Biotechnologies to Develop Novel Rare Disease Gene Editing Programs

Shots: Chiesi Group has entered into an exclusive global collaboration & license deal with Arbor for ABO-101 in primary hyperoxaluria type 1 (PH1) & a multitarget option to use Arbor’s gene editing platform for developing liver-targeted therapies for rare diseases As per the deal, Chiesi will obtain exclusive rights to develop & commercialize ABO-101 for PH1…

ByRidhi RastogiOctober 7, 2025

Chiesi Group and Arbor Biotechnologies Secure the EC’s Orphan Drug Designation for ABO-101 to Treat Primary Hyperoxaluria

Chiesi Group Enters a ~$2.1B Deal with Arbor Biotechnologies to Develop Novel Rare Disease Gene Editing Programs

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