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biotech

New gene therapy helps patients with rare disease. One mother wouldn't have it any other way.

A biotech in Cambridge, Mass., is targeting a rare disease called cystinosis with gene therapy. It's been effective for five patients in a clinical trial that's still underway.

Cherqui Lab

Three years ago, Jordan Janz of Consort, Alberta, knew his gene therapy treatment for cystinosis was working when his hair started to darken. Pigmentation or melanin production is just one part of the body damaged by cystinosis.

“When you have cystinosis, you’re either a redhead or a blonde, and you are very pale,” attests Janz, 23, who was diagnosed with the disease just eight months after he was born. “After I got my new stem cells, my hair came back dark, dirty blonde, then it lightened a little bit, but before it was white blonde, almost bleach blonde.”

According to Cystinosis United, about 500 to 600 people have the rare genetic disease in the U.S.; an estimated 20 new cases are diagnosed each year.

Located in Cambridge, Mass., AVROBIO is a gene therapy company that targets cystinosis and other lysosomal storage disorders, in which toxic materials build up in the cells. Janz is one of five patients in AVROBIO’s ongoing Phase 1/2 clinical trial of a gene therapy for cystinosis called AVR-RD-04.

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Christopher Johnston
Christopher Johnston has published more than 3,500 articles in publications including American Theatre, Christian Science Monitor, History Magazine, and Scientific American. His book, Shattering Silences: Strategies to Prevent Sexual Assault, Heal Survivors, and Bring Assailants to Justice (Skyhorse) was published in May 2018. He is a member of the Board of the American Society of Journalists and Authors.
A new approach to kidney transplants would reprogram the immune system

If approved by the FDA, a new procedure for kidney transplants that doesn't require anti-rejection medication could soon become the standard of care.

Talaris Therapeutics, Inc., a biotech company based in Louisville, Ky., is edging closer to eradicating the need for immunosuppressive drugs for kidney transplant patients.

In a series of research trials, Talaris is infusing patients with immune system stem cells from their kidney donor to create a donor-derived immune system that accepts the organ without the need for anti-rejection medications. That newly generated system does not attack other parts of the recipient’s body and also fights off infections and diseases as a healthy immune system would.

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Christopher Johnston
Christopher Johnston has published more than 3,500 articles in publications including American Theatre, Christian Science Monitor, History Magazine, and Scientific American. His book, Shattering Silences: Strategies to Prevent Sexual Assault, Heal Survivors, and Bring Assailants to Justice (Skyhorse) was published in May 2018. He is a member of the Board of the American Society of Journalists and Authors.
New Cell Therapies Give Hope to Diabetes Patients

Researchers are developing new cell therapies that could cure Type 1 diabetes and make constant management of the disease a way of the past.

For nearly four decades, George Huntley has thought constantly about his diabetes. Diagnosed in 1983 with Type 1 (insulin-dependent) diabetes, Huntley began managing his condition with daily finger sticks to check his blood glucose levels and doses of insulin that he injected into his abdomen. Even now, with an insulin pump and a device that continuously monitors his glucose, he must consider how every meal will affect his blood sugar, checking his monitor multiple times each hour.

Like many of those who depend on insulin injections, Huntley is simultaneously grateful for the technology that makes his condition easier to manage and tired of thinking about diabetes. If he could wave a magic wand, he says, he would make his diabetes disappear. So when he read about biotechs like ViaCyte and Vertex Pharmaceuticals developing new cell therapies that have the potential to cure Type 1 diabetes, Huntley was excited.

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Carrie Arnold
Carrie Arnold is an independent public health journalist from Virginia.
People With This Rare Disease Can Barely Eat Protein.  Biotechnology May Change That.

The Brown family at the Grand Tetons (2019). Clockwise from left, Christine, Kevin, Keagan, Connor, and Kellen.

Courtesy Brown family

Imagine that the protein in bread, eggs, steak, even beans is not the foundation for a healthy diet, but a poison to your brain. That is the reality for people living with Phenylketonuria, or PKU. This cluster of rare genetic variations affects the ability to digest phenylalanine (Phe), one of the chemical building blocks of protein. The toxins can build up in the brain causing severe mental retardation.

Can a probiotic help digest the troublesome proteins before they can enter the bloodstream and travel to the brain? A Boston area biotech start up, Synlogic, believes it can. Their starting point is an E. coli bacterium that has been used as a probiotic for more than a century. The company then screened thousands of gene variants to identify ones that produced enzymes most efficient at slicing and dicing the target proteins and optimized them further through directed evolution. The results have been encouraging.

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Bob Roehr
Bob Roehr is a biomedical journalist based in Washington, DC. Over the last twenty-five years he has written extensively for The BMJ, Scientific American, PNAS, Proto, and myriad other publications. He is primarily interested in HIV, infectious disease, immunology, and how growing knowledge of the microbiome is changing our understanding of health and disease. He is working on a book about the ways the body can at least partially control HIV and how that has influenced (or not) the search for a treatment and cure.