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New avenues for rare disease treatment

Drug Target Review

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

Disease 113
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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111
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Ascentage Pharma’s Licensee UNITY Biotechnology Announces Milestone Reached in Clinical Development of Treatments for Age-Related Disease, Leading to Milestone Payment

The Pharma Data

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. This progress in clinical development qualifies Ascentage Pharma for a milestone payment according to the terms of the licensing agreement. SUZHOU, China and ROCKVILLE, Md. ,

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Human neuronal cells: possibilities in drug safety testing

Drug Target Review

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. 2018 Mar;17(3):167–81. 2018 Dec 11;94(10):390–411.

Drugs 111
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Meet the Patient Advocate Panelists in Our Upcoming Webinar: Rare Disease Day 2023 Wrapped, What You Need to Take Away

Conversations in Drug Development Trends

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

Disease 52
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Women in Stem with Dr Christine Schuberth-Wagner

Drug Target Review

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Everything started in school with an experiment on isolating DNA from bananas. in North America) in late 2017.

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Hummingbird Bioscience Announces Collaboration with Tempus to Harness AI-driven Precision Medicine to Accelerate Clinical Development of HMBD-001 In HER3 Driven Cancers

The Pharma Data

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.