Gene therapy is making huge strides in tackling genetic neuromuscular diseases. These diseases, which include conditions like spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), are caused by faulty genes. Recently, a type of virus called adeno-associated virus (AAV) has been used to deliver healthy genes to patients. This method has shown promising ...
Bluebird Bio, once a shining star in the biotech world, is now a shadow of its former self. The company, known for its groundbreaking gene therapies, has been sold to private equity firms Carlyle and SK Capital for a mere $30 million. This sale marks the end of a long and tumultuous journey for Bluebird, which once had a market cap of around $9 billion. The company'...
Rare gene problem called Aromatic L-amino acid decarboxylase deficiency (AADCd) makes it tough for kids to move and learn. It gets in the way of their coordination, balance, and brain development. AADCd affects a small number of people but can cause big problems. Think of it as a roadblock on the path to normal growth and movement for children. There's a new treatme...
Diving right in, choroideremia is a rare disease that affects a person's vision and is a big deal for patients. It's like a storm in a small town. This gene-related condition can severely impact a person's quality of life. Gene therapy, a fancy word for fixing genetic issues, has been a big star in treating choroideremia. Traditional methods, like replacing ...
Imagine if the tiny, and usually nasty, viruses could be used to target and eliminate cancer cells. Scientists have been exploring this idea for a while. They are using oncolytic viruses. One type of virus, the recombinant Adeno-associated virus, has been programmed to carry a gene called TRAIL. This gene is programmed to destroy cancer cells, but the process is no...
In May 2024, San Antonio, Texas, played host to the 18th Workshop on Recent Issues in Bioanalysis (18th WRIB). This annual gathering brings together over 1, 100 experts from pharmaceutical and biotech companies, contract research organizations (CROs), and regulatory agencies. The week-long event is dedicated to discussing the latest challenges and solutions in bioana...
You might have heard about cell and gene therapies (CGTs), which can treat rare and sometimes deadly diseases. The catch? They're expensive. People are worried that insurance companies might not want to pay for them. If you look at the big picture, the yearly cost of covering these therapies for everyone in the US from 2023 to 2035 would be less than $20 per person. ...
Hospital pharmacists are increasingly being acknowledged for their work with cell and gene therapies, as well as advanced therapy medicinal products (ATMPs). However, there's a lack of systematic evidence showing how effective their role really is. Let's break down what they do and why it's important. First, pharmacists help ensure these complex treatments are used ...
Scientists are finding an unexpected hero in the fight against cancer and inflammation: plants. They've discovered tiny bubbles made by plants, called PDNVs, that can safely carry important genetic messages to the right cells. These messages can help cells fix broken genes or fight diseases, making them more stable and effective than other methods. This plant-powered...
Imagine trying to solve a puzzle where you need to match the right pieces together. In the world of genetics, this is called phasing—figuring out which genes on chromosome pairs go together. This process is crucial for diagnosing and treating certain skin diseases, like recessive dystrophic epidermolysis bullosa. Advances in long-read sequencing have revolutionized...