Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

Gene Therapy 277

Gene Therapy Licensing Licensing Gene 277

pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. In 2019 the Danish company also licensed a preclinical-stage, small-molecule candidate for NASH from Japan’s UBE.

Gene Silencing 66

Gene Silencing Gene RNA Contract Manufacturing 66

pharmaphorum

MAY 11, 2022

The pharma group will provide funding for the research in return for exclusive license options on the programmes. billion takeover of gene-silencing specialist Dicerna Pharma last year, another deal aimed at extending its R&D capabilities by bolting on a new platform technology – in this case RNA interference (RNAi) drugs.

Gene Silencing 126

Gene Silencing Medicine RNA Licensing 126

pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7 It also gets two preclinical-stage development projects.

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

pharmaphorum

FEBRUARY 9, 2021

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells. Regulus meanwhile remains a microRNA player, but has suffered a number of setbacks.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52