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. They can be developed and adapted quickly.

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pharmaphorum

OCTOBER 16, 2020

With large datasets crucial to gaining an understanding of rare diseases and opening the door to drug development, digital technology is proving transformative. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise.

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Clinical Trials Clinical Trials Trials Life Science 105

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost.

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RNA Antibody Life Science Protein 52

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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Clinical Trials Clinical Trials Genetics Trials 98

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

NOVEMBER 10, 2023

Acute manifestations include thrombocytopenia, microangiopathic hemolytic anemia, bleeding episodes, headache and abdominal pain, while chronic ones include stroke, cardiovascular disease and damage to organs. As a rare disease, cTTP affects less than 1,000 individuals in the US.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. 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

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. The marketing rights to Thiola were acquired by Retrophin in 2014 from Mission Pharmacol.

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Trials Gene Hormones Clinical Trials 98

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic. vaccine, mRNA-1273.815.

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Genetics Vaccination Vaccine DNA 111

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 We believe it can now do the same for rare diseases.” “We

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RNA Protein Vaccination Vaccine 52

Drug Discovery World

MARCH 6, 2024

The prevalence of these diseases has grown substantially in recent decades due to an increasing global population, longer life expectancies and increasing morbidity with predisposing risk factors. This now represents one of the most challenging and costly burdens to health and social care systems and societies worldwide.

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Genome Genomics Clinical Trials Clinical Trials 64

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. The case for new approaches and thought processes is clear, so in which drug discovery areas is it being embraced and developed? Where is the innovation?

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Drugs Life Science Genome Genomics 59

The Pharma Data

OCTOBER 15, 2020

The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. The committee is tasked with supporting BeyondSpring’s business development activities related to its lead asset, Plinabulin, and other pipeline assets. marketing and diagnostics.

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