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 its Phase 2 clinical trial for RLYB212. ... Read More
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 acanthamoeba keratitis (AK) globally, announced Avanzanite Bioscience ... Read More
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 a portfolio of preclinical gene therapy programmes ... Read More
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 deal worth $68 million. As per the ... Read More
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, orphan pulmonary, and rheumatic diseases. The acquisition ... Read More
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 treatment-naïve patients in China having Gaucher disease ... Read More
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 agreed to acquire Viela Bio, a US-based ... Read More
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 has sealed a $39 billion deal to ... Read More
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 regarding its treatment for Fabry disease—pegunigalsidase alfa ... Read More
F2G’s olorofim gets FDA ODD for mold infections and Valley Fever
F2G has bagged orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for olorofim (formerly F901318) for the treatments of invasive mold ... Read More