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 60

Gene Silencing Gene RNA Contract Manufacturing 60

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

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

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

JUNE 8, 2022

The passport means that LentiGlobin will be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA) via the new innovative licensing and access pathway (ILAP) introduced in 2020, which reduces the review time for a new medicine to 150 days.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52

pharmaphorum

OCTOBER 29, 2020

The Swiss pharma group already sells one gene therapy for inherited retinal diseases (IRDs) – Luxturna (voretigene neparvovec) – for which it licensed ex-US rights from Spark Therapeutics (now part of Roche) in 2018. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40