Lifeboat Foundation

Following strong results for its flagship CTX001 drug, should you add CRISPR to your portfolio?

Merck now holds 22 CRISPR-related patents worldwide across nine different geographies.

- Patents cover Paired Cas9 Nickase CRISPR genome-editing technology to advance gene therapy and research.

DARMSTADT, Germany, Nov. 26, 2019 /PRNewswire/ — Merck, a leading science and technology company, today announced that the Japan Patent Office and the Intellectual Property Office of Singapore have each allowed the company’s patent application for the use of paired CRISPR nickases, bringing Merck’s number of patents to 22 worldwide.

NOTE FROM TED: Please do not look to this talk for medical advice. This talk, which was filmed at a TEDx event, contains strong assertions about multiple sclerosis and lifestyle medicine that lack sufficient scientific evidence for general prescription. TEDx events are independently organized by volunteers. The guidelines we give TEDx organizers are described in more detail here: http://storage.ted.com/tedx/manuals/tedx_content_guidelines.pdf

After a shocking diagnosis that would begin stripping Bob Cafaro of his ability to perform, sheer willpower and changes to his daily life allow him to beat all odds.

Continue reading “The Psychology of Beating an Incurable Illness | Bob Cafaro | TEDxCharlottesville” »

Akash Manoj started reading medical books written by global writers only for fun when he was in the 8th class. And just after two years, this child has taken the medical scientists by surprise as he has developed a device to predict ‘silent’ heart attacks. This disease kills thousands of people every year in India alone.

Just after the complition of his 7^th class exams, Akash began to visit fairly well-known libraries in Bangalore and he gradually fell in love with medical novels.

“Journal articles are expensive, so visiting the libraries was the only way I could do it. Otherwise, it would have cost more than a crore (of rupees) for the amount I read. I was always interested in medical science and I liked reading the journals…cardiology is my favourite,” said Manoj.

It turns out the gut is full of surprises. And one of those surprises may have offered up a key for unlocking a new way of treating multiple sclerosis (MS). Investigators from Brigham and Women’s Hospital have discovered a microRNA—a small RNA molecule—that increases during peak disease in a mouse model of MS and in untreated MS patients. When a synthetic version of the microRNA was orally given to the mice, it prevented disease. While several steps remain before these insights can be translated into therapy for patients, the researchers describe their results as both exciting and unexpected. Their findings are published in Cell Host & Microbe.

“We’ve discovered a new mechanism to regulate the microbiome and treat human disease that hadn’t been known before,” said senior author Howard Weiner, MD, co-director of the Ann Romney Center for Neurologic Diseases at the Brigham. “The gut microbiome is known to play an important role in MS and other diseases. Our findings, which show that a microRNA can be used to target and influence the microbiome with precision, may have applicability for MS and many other diseases, including diabetes, ALS, obesity and cancer.”

Weiner, lead author Shirong Liu, MD, Ph.D., an instructor in the Weiner laboratory, and their colleagues investigated how the altered gut microbiome affects the course of MS. To do so, they studied the microbiome and microRNAs found in the experimental autoimmune encephalomyelitis (EAE) model of MS. Unexpectedly, they found that when they transferred fecal matter from EAE mice at peak disease, it protected the mice who received the transfer. The team found that a specific microRNA, known as miR-30d, rather than live bacteria, was responsible for preventing disease. The investigators found that miR-30d is enriched in untreated, relapsing-remitting MS patients as well.

Recently, Libella Gene Therapeutics has announced that it will be running a patient-paid trial in Colombia with an eye-watering $1 million USD price tag on enrollment.

Patient-paid trial likely to cause backlash

The topic of patient-paid trials often stirs up considerable debate among the research community, regulatory authorities, and the general public, with many people suggesting that it is unethical to expect patients to pay to participate in clinical trials. While this is a controversial issue, these trials are a legitimate way to test therapies that would otherwise struggle to reach the clinic due to cost constraints, and the data gathered by such trials can still be valuable.

The study saw the deployment of a multiple target gene therapy focused on 3 known longevity genes delivered via an adeno-associated virus. The focus was on mitigating T2 diabetes, heart failure, and kidney failure in mouse models with very positive results observed. Join us on Tuesday, 26th November, 1pm EDT on our Facebook page for the livestream show.

In Technion, Bar-Ilan trials, nanochip ferries ‘neural growth factor’ — which combats neurodegenerative diseases — and releases it where needed, overcoming blood-brain barrier.

Testing on the equipment and testing procedure will begin next year.