Gene therapy: Making Rare Disease Therapies Less Rare
…therapies for patients with rare diseases. More than 70% of rare diseases are known to be caused by a single mutation, which makes them good candidates for gene therapy. Several…
Search Results for: metabolic diseases
News and Trends 29 Aug 2022
Marketing authorization granted for treatment of serious retinal diseases
The European Commission has granted marketing authorization to Ranivisio, a treatment for several serious retinal diseases. Polpharma Biologics Group BV, Formycon AG and their Swiss joint venture, Bioeq AG, announced…
News and Trends 21 Oct 2016
New Partnership comes to the Rescue in Neurological Diseases
Developing effective treatments for neurological diseases is a big challenge in medicine. But here come UCB and Evotec with a new partnership that could help those suffering from epilepsy and…
Podcast 6 Oct 2023
Beyond Biotech podcast 66 – treating rare diseases with tRNA
…are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic mutation. This year, Alltrna presented…
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News and Trends 16 Oct 2017
Interview: On the Future of iPS Cells and why Partnering is so Important in this Field
…own defective cells. The second is in the broader field of drug discovery. Here, iPS cells are being used to explore the underlying causes of human diseases at a molecular…
News and Trends 23 Nov 2022
Khondrion’s mitochondrial diseases treatment producing positive results
Clinical-stage pharmaceutical company, Khondrion, discovering and developing therapies targeting primary mitochondrial diseases announced yesterday (November 22) its wholly-owned lead asset sonlicromanol for MELAS spectrum disorder has demonstrated positive treatment results….
News and Trends 6 Jul 2023
£100M LifeArc program aims to help people living with rare diseases
…rare disease, with approximately 3.5 million people in the U.K. affected. With more than 7,000 rare diseases identified, there is a need for new treatments and technologies to speed up…
News and Trends 4 Jul 2019
Dutch Antibodies for Autoimmune Diseases Boosted by €15M Series A
The Dutch biotech Citryll has raised €15M to develop antibodies that could fight autoimmune and inflammatory diseases more effectively than current treatments by inhibiting the innate immune system. The Series…
News and Trends 24 Apr 2023
Life Biosciences gene therapy data shows visual function restored in primates
…development of our scientific platform to address diseases of aging and restore human health.” Life Biosciences tackling range of aging diseases Life Biosciences is advancing its cellular rejuvenation capabilities across…
In Depth 9 Oct 2018
How to Capitalize on Opportunities in Underserved Disease Areas
…biotech companies and investors. Traditionally diseases are ‘underserved’ because they are complex and we don’t understand them well enough, such as some of the more common autoimmune diseases, or because…
News and Trends 4 Dec 2019
€48M Series B Fuels Immunotherapies for Cancer and Autoimmune Diseases
ImCheck Therapeutics has raised a Series B round worth €48M to fund the development of first-in-class immunotherapies that treat cancer and autoimmune diseases by targeting a rare type of immune…
News and Trends 26 Oct 2016
Proteins can now Sing about their Anomalies to help cure Diseases
…its features. This project could become a powerful new tool to detect anomalies that cause all kinds of diseases. Protein fold assignment is a notoriously tricky area of research in…
