Pharmaceutical Technology

AUGUST 17, 2022

These molecules synthesised newly are packaged densely into custom lipid nanoparticles (LNPs), which Orna has made to act on the body’s crucial tissues. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

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RNA Vaccination Vaccine In-Vivo 147

Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. Products are stored and packaged in a dedicated suite by a dedicated team,” explains MacNeir.

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Gene Therapy Gene Packaging Packaging 130

XTalks

SEPTEMBER 16, 2024

The compelling data included in our NDA package show that sebetralstat has the potential to significantly alter the way people treat and manage their disease,” he said.

Protein 52

Protein Genetics Trials Allergies 52

The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. Genetic diseases are vastly more common and more complex than we used to think.”. Patient dosing is expected to begin later this year. “No

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

The Pharma Data

DECEMBER 8, 2020

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Most read today on BioSpace: Source link.

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

AUGUST 3, 2021

While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

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Clinical Trials Clinical Trials Gene Therapy Gene 52