The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

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In-Vivo Marketing Engineer Genetics 52

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

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XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production.

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FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

FEBRUARY 27, 2023

Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues).

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

XTalks

MAY 12, 2023

Caused by mutations in the MECP2 gene located on the X chromosome, Rett syndrome is primarily an X-linked disorder; however, there have been rare cases of boys being affected, usually with more severe symptoms due to the absence of a second X chromosome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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FDA Approval Genetics Trials Drugs 97

XTalks

SEPTEMBER 17, 2020

Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Thiola (tiopronin) is produced by Retrophin, the rare disease biopharmaceutical company founded by Martin Shkreli. Individuals with this disease are deficient in ?-galactosidase

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Trials Gene Hormones Clinical Trials 98

Drug Discovery World

JULY 8, 2024

For decades, the life sciences community held the belief that RNA was merely the intermediary messenger (mRNA) between DNA and protein, a dogma that has been the foundation of biology. RNA interference: silencing gene expression The second category of RNA therapeutics is a class that has also yielded marketable drugs in recent years.

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RNA Protein Gene Expression Vaccination 59

Drug Discovery World

OCTOBER 12, 2023

ELRIG’s Drug Discovery 2023 will be taking place at ACC Liverpool, UK from 18-19 October 2023, focusing on the importance of a diverse life science ecosystem. Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity.

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Drugs Life Science Genome Genomics 59

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

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Genome Genomics Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

Moreover, it has submitted global marketing authorization applications for mRNA-1345, a vaccine designed to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD), supported by favorable data from a predefined interim analysis. vaccine, mRNA-1273.815.

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Genetics Vaccination Vaccine DNA 111

Drug Discovery World

APRIL 30, 2024

The biggest growth driver for mRNA therapeutics came from the Covid-19 pandemic, when mRNA-based vaccines proved effective in treating SARS-CoV-2, and pharmaceutical companies such as Pfizer/BioNTech and Moderna were successful in bringing these treatments to market. We believe it can now do the same for rare diseases.” “We

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RNA Protein Vaccination Vaccine 52

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. marketing and diagnostics. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization.

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In-Vivo Business Development Manufacturing Development 52