Gene Editing, Gene Silencing and Trials - Clinical Research Informer

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

In-Vivo

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

JULY 13, 2021

The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system. All told, the deal could be worth up to $1.2

Gene Silencing

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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

Genetics

Genetics Medicine Gene Therapy Marketing

Clinical Research Informer

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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