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Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation.

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The ins and outs of the life sciences: top hires for October 2022 

pharmaphorum

This month, we highlight new appointments from Lumeris, Lineage Cell Therapeutics, 1910 Genetics, and ADC Therapeutics. . ” Patrick Genestin, 1910 Genetics . “I am excited to join 1910 Genetics at such a pivotal time for the company,” Genestin said. Ben Grabski, Lumeris.

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Eli Lilly’s Olumiant FDA Approved for Alopecia + Alnylam’s RNAi Drug Amvuttra Approved for Rare Protein Disorder – Xtalks Life Science Podcast Ep. 66

XTalks

Ayesha also discussed the FDA approval of Alnylam’s RNAi therapeutic Amvuttra for the treatment of polyneuropathy associated with a rare protein disorder called hereditary transthyretin-mediated (ATTR) amyloidosis. Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder.

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How cells repair DNA's protective barrier: Addressing a rare genetic disorder characterized by rapid aging in children

Medical Xpress

Researchers at Nano Life Science Institute (WPI-NanoLSI), Kanazawa University, have discovered how a protein called lamin A helps repair the protective barrier around a cell's DNA.

DNA 52
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Solid Biosciences acquires AavantiBio, conducts $75 million private placement

pharmaphorum

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal which is expected to result in a combined sum of $215 million in cash and investments – projected to be enough for funding into 2025. This is due to close concurrently with the merger.

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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

Elsewhere, Albireo Pharma is aiming to file for supplemental NDA to the FDA for its drug Bylvay (odevixibat), which could be the second treatment for Alagille syndrome, a rare genetic disorder caused by a JAG1 gene mutation. The condition is characterised by blockage of the flow of bile from the liver, and pruritus or itching.

Drugs 317
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Mapping Networks of Immune Genes Behind Autoimmune Diseases

XTalks

For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. The proteins are interconnected and changes in one protein level can impact that of another.

Gene 98