BioMarin to acquire Alesta Therapeutics for $275M upfront, gaining ALE1 — a potential first oral therapy for hypophosphatasia (HPP)....
Vaderis raises an oversubscribed $152M Series B to fund engasertib through FDA approval, as Phase 3 HEROIC begins for rare disease HHT....
Jazz Pharmaceuticals to acquire Actio Biosciences for $820M, adding ABS-1230, a first-in-class therapy for rare KCNT1+ epilepsy....
Unnatural Products Series B financing secures $45M after a Novartis pact to advance macrocycle drugs toward the clinic....
Vico Therapeutics begins twice-annual dosing of VO659 in HD, SCA3 & SCA1 patients. FDA IND cleared. First ASO targeting CAG repeat expansion across polyglutamine diseases....
“The preclinical progress of ALE1 and ALE2 underscores the company’s potential to deliver much-needed treatment options, and we are excited to support the continued advancement of these programs in 2025 and beyond.”...
“The preclinical progress of ALE1 and ALE2 underscores the company’s potential to deliver much-needed treatment options, and we are excited to support the continued advancement of these programs in 2025 and beyond.”...
“The preclinical progress of ALE1 and ALE2 underscores the company’s potential to deliver much-needed treatment options, and we are excited to support the continued advancement of these programs in 2025 and beyond.”...
The FDA's IND clearance is a pivotal moment for Accent Therapeutics, propelling our lead program into the clinical arena. ATX-559 has shown remarkable promise in preclinical studies, and we're excited to now evaluate its potential to meaningfully improve outcomes for patients facing these challenging malignancies....
Judo Bio has discovered a novel approach to create oligonucleotide medicines targeted to the kidney, an organ that has been challenging to access for genetic medicines. The company has completed preclinical studies successfully demonstrating receptor-mediated delivery of oligonucleotides to the kidney with proprietary ligand-siRNA conjugates...