XTalks
SEPTEMBER 13, 2021
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
pharmaphorum
OCTOBER 5, 2022
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.
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pharmaphorum
JANUARY 7, 2022
AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months. The new agreement comes just days after AZ closed a $3.1 billion on the first nine months of 2021, well ahead of Pfizer’s own forecasts.
pharmaphorum
JULY 6, 2021
The small interfering RNA (siRNA) therapy has already been approved in Europe as Leqvio , and the FDA’s rejection was not caused by any issues with its efficacy or safety, according to Novartis. Novartis added inclisiran to its pipeline after buying The Medicines Company, which had licensed the drug from Alnylam, for $9.7
pharmaphorum
DECEMBER 23, 2021
The first-in-class, small interfering RNA (siRNA) therapy for cholesterol lowering inhibiting PCSSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Praluent (alirocumab) – but after a lead-in period is dosed only twice a year rather than every month. It has a long way to go.
pharmaphorum
APRIL 5, 2022
AstraZeneca’s take on the PCSK9 inhibitor – an antisense drug licensed from Ionis six years ago – has cleared a proof-of-concept trial in high-risk patients with high blood cholesterol, but it remains to be seen where it will fit into the increasingly busy category.
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. The major difference between siRNAs and microRNAs is that the former are very specific to one messenger RNA target, while the latter can have multiple targets.
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