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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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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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

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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.

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Drug Discovery World

JULY 11, 2022

There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. " Critical Considerations For Packaging And Delivery Of Gene Therapy Products." Conclusion. The promise of gene therapy is substantial. Molecular therapy. Methods& clinical development, 21,524–529. link] omtm.2021.04.001.

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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 Trials 52