XTalks

MAY 17, 2023

In the past several years, gene therapies have finally been hitting the market after decades of setbacks and uncertainties. The announcement of its clinical trial portfolio is an important milestone, bringing people with rare genetic diseases one step closer to approved therapies.”

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pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. The post Acadia closer to bringing first Rett syndrome drug to market appeared first on.

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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

pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. The post Rare diseases, repurposing and the role of AI appeared first on.

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XTalks

FEBRUARY 27, 2023

Related: Interview with the Leaders at Amylyx, Maker of New FDA Approved ALS Drug Relyvrio – Xtalks Life Science Podcast Ep. 90 Accessing Alpha-Mannosidosis Treatment The Chiesi Group previously received marketing authorization from the European Commission for Lamzede in 2018.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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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

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. Cornering the untapped Chinese CNS market, SciNeuro launched with $100 million in their pocket.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Regulatory requirements.

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XTalks

NOVEMBER 27, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio Price of Pombiliti and Opfolda Therapy Amicus Therapeutics is set to introduce Pombiliti and Opfolda to the US market without delay, with an annual price of $650,000 for patients with a median weight.

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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

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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XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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XTalks

NOVEMBER 10, 2023

Approvals for rare disease treatments have been on the rise in a growing rare disease market that is currently valued at more than $195 billion in 2022. The company received an FDA nod for its VEGFR inhibitor Fruzaqla (fruquitinib) as a third-line treatment for adults with metastatic colorectal cancer.

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XTalks

SEPTEMBER 17, 2020

Thiola (tiopronin) is produced by Retrophin, the rare disease biopharmaceutical company founded by Martin Shkreli. The marketing rights to Thiola were acquired by Retrophin in 2014 from Mission Pharmacol.

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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. I would also specifically highlight the importance of considering the patient perspective in defining drug discovery strategies and delivering medicines to market.

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Drugs Life Science Genome Genomics 59

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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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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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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The Pharma Data

OCTOBER 15, 2020

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. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

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