Browsing Tag
Rare diseases
36 posts
Rocket Pharmaceuticals receives FDA clearance to begin human trials for RP-A701 in treating BAG3-associated dilated cardiomyopathy
Rocket Pharmaceuticals earns FDA IND clearance for RP-A701, advancing gene therapy for rare BAG3-associated dilated cardiomyopathy. Trial begins in U.S.
Rallybio Phase 2 trial of RLYB212 begins to tackle FNAIT in high-risk pregnancies
Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company known for advancing innovative therapies for rare diseases, has commenced…
Avanzanite Bioscience secures EC approval for AKANTIOR as first authorised treatment for acanthamoeba keratitis
The European Commission (EC) has granted marketing authorisation for AKANTIOR (polihexanide), the first and only approved treatment for…
AstraZeneca’s Alexion to boost rare disease commitment with Pfizer’s gene therapy programs
Alexion, a subdivision of AstraZeneca focusing on rare diseases, has announced its definitive purchase and licensing agreement for…
Alexion to acquire genetic medicine company LogicBio Therapeutics
Alexion, AstraZeneca Rare Disease has agreed to acquire LogicBio Therapeutics, an American clinical-stage genetic medicine company in a…
Idera Pharmaceuticals acquires rare orphan disease company Aceragen
Idera Pharmaceuticals, a US-based biopharma company, has acquired Aceragen, a biotechnology company focused on developing drugs for rare,…
CANbridge Pharmaceuticals begins CAN103 phase 1/2 trial in Gaucher disease
CANbridge Pharmaceuticals said that it has dosed the first patient in a phase 1/2 trial of CAN103 in…
Horizon Therapeutics announces $3.05bn acquisition of Viela Bio to expand rare disease pipeline
In a major move to strengthen its leadership in rare diseases, Horizon Therapeutics, an Irish biopharmaceutical company, has…
AstraZeneca signs $39bn deal to acquire Alexion Pharmaceuticals
In a landmark move that’s poised to shake up the world of immunology and rare disease treatments, AstraZeneca…
Protalix BioTherapeutics receives FDA extension for pegunigalsidase alfa review: What this means for Fabry disease treatment
Protalix BioTherapeutics, a leader in the development of innovative therapies for rare diseases, has announced a significant update…