uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1  ~ License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~ ~ APB-102 and uniQure’s c9orf72-ALS...

The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS Publication includes the Phase 3 VALOR SOD1-ALS trial and its open-label-extension study, underscoring the importance of these longer-term data12-month data show earlier initiation of tofersen slowed...

Biogen to Present New Interim Data from Its Phase 1/2 Clinical Study of Tofersen (BIIB067) for the Potential Treatment of a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) Tofersen, an antisense oligonucleotide (ASO) that selectively targets the genetic driver of disease, is being studied for the potential treatment of ALS in adults with a confirmed superoxide dismutase...

Biogen Exercises Option with Ionis to Develop and Commercialize Investigational Treatment BIIB067 for a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) Based on Positive Phase 1 Data Positive interim analysis demonstrated proof-of-biology and proof-of-concept for BIIB067Biogen plans to advance BIIB067 to a pivotal clinical studyBiogen paid Ionis Pharmaceuticals a $35 million...