Browsing Tag
rare disease
23 posts
Breakthrough hope: Benitec Biopharma’s BB-301 delivers promising results in rare muscular dystrophy trial
Find out how Benitec Biopharma’s BB-301 gene therapy is showing early success in restoring swallowing function for OPMD patients.
Belite Bio wins UK MHRA nod to file conditional marketing application for tinlarebant in Stargardt disease
Find out how Belite Bio gained UK MHRA backing to file a conditional marketing application for tinlarebant, advancing the first potential therapy for Stargardt disease.
Savara’s $130m stock offering strengthens cash position ahead of MOLBREEVI launch
Savara raises $130 million through stock and warrant sale to fund MOLBREEVI’s rare-disease launch—find out how this deal reshapes its commercialization path.
FDA grants priority review for BioMarin’s PALYNZIQ to treat adolescents with phenylketonuria (PKU)
Find out how BioMarin’s PALYNZIQ priority review could transform PKU treatment for adolescents and reshape the company’s rare-disease strategy.
Kiniksa’s KPL-387 earns FDA orphan drug nod, advancing next-gen therapy for recurrent pericarditis
Find out how Kiniksa’s KPL-387 won FDA orphan drug status for pericarditis — and what it means for the company’s expanding rare-disease franchise.
Rani Therapeutics seals $1.085bn Chugai deal and $60m oversubscribed financing to fuel oral biologics push
Rani Therapeutics secured a $1.085 B Chugai collaboration and an oversubscribed $60.3 M raise. Find out how this dual deal could redefine its biotech trajectory.
Quoin Pharmaceuticals raises up to $104.5M in private placement to fuel its rare disease pipeline
Find out how Quoin Pharmaceuticals secured up to $104.5 million in a milestone-linked private placement to advance its rare disease therapy QRX003 and strengthen investor confidence.
DXB rockets on Japan orphan drug win — can Dimerix finally deliver an FSGS breakthrough?
Dimerix (ASX: DXB) secures orphan drug designation in Japan for DMX-200, strengthening its global Phase 3 kidney trial program. Find out what’s next.
Sarepta Therapeutics awards 25 Route 79 scholarships in 2025–2026, but what makes this Duchenne program unique?
Sarepta Therapeutics awarded 25 Route 79 scholarships for 2025–2026 to support students impacted by Duchenne. See how this initiative strengthens community ties.
Takeda’s oveporexton Phase 3 trials show broad success in narcolepsy type 1, paving way for 2025 FDA submission
Takeda announces pivotal Phase 3 trial results for oveporexton (TAK-861), showing statistically significant improvements in narcolepsy type 1 symptoms. Regulatory filings planned.