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

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).

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Gene Therapy Gene FDA Approval Dermatology 97

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. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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Gene Therapy Gene Development Production 52

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases. By Cytiva Thematic.

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RNA Gene Therapy Genetics Genetic Disease 130

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Bone marrow transplantation is also used in some infantile-onset cases to help delay disease progression.

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Gene Therapy Drugs Gene Gene Silencing 98

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. “They called him ‘The Boy.’ Who wasn’t supposed to sit up.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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DNA Gene Gene Silencing Genome 98

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP.

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FDA Approval Gene Therapy Gene Clinical Trials 59

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.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

DNA 52

DNA Gene Gene Silencing Genome 52

pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

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Clinical Trials Clinical Trials Trials Development 110

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.

Drugs 59

Drugs Life Science Genome Genomics 59

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. However, these therapies can cause severe adverse effects. We believe it can now do the same for rare diseases.” “We Wang YS, Kumari M, Chen GH, et al.

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The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. 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. Additionally, Parag V.

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In-Vivo Business Development Manufacturing Development 52