By Pat Furlong and Sheila Moeschen Consider this: At 90 years old, my mother still spoke about the Nun from her grammar school who tapped her on the shoulder and said, “You are nothing like your sisters and brothers.” The sentence formed a toxin, a slow drip into the bloodstream…

Cell therapy biotech Immusoft will partner Takeda to develop new cell therapies to target rare inherited metabolic disorders with central nervous system (CNS) manifestations and complications in a deal that could be worth more than $900M if all targets are met. Immusoft was founded in 2009 and is headquartered in…

Researchers based at Harvard and the Broad Institute have adapted currently used gene therapy vectors to make them more suitable for targeting muscular conditions such as Duchenne muscular dystrophy and also to make them less toxic to patients. One of the most common methods for delivering gene therapy today is…

There is a still a lot of ground to make up in encouraging young girls and women to follow their hearts into STEM careers where women continue to be underrepresented in industry positions. But that doesn’t mean women aren’t making their presence felt in jobs that can greatly influence the…

The announcement of the failure of two Huntington’s disease trials run by Roche and Wave Life Sciences earlier this year was a great disappointment to clinicians and patients alike, but what do the results mean for the field? Researchers have been working to develop more effective treatments or even cures…

Molecular methods of diagnosing diseases promise fast and accurate approaches for an array of conditions. While many molecular diagnostics look for mutations in DNA, an increasing number analyze a sample’s RNA,  and those are explored here. Manufacturers offer a variety of RNA-based diagnostics, and academic scientists continue to explore new…

In genome sequencing, “complete” never really means 100% complete. The genomes that are called complete usually omit repetitive DNA segments, which are usually scanned in short, hard-to-piece-together segments. Not content with with this standard of completions, a large group of genomic researchers recently decided to try something different. The researchers,…

Researchers at Cedars-Sinai Medical Center are planning a randomized, controlled trial to evaluate a form of allogeneic cell therapy for COVID-19, after four out of six patients critically ill with the disease recovered well enough following infusions of the cells to be discharged from the hospital. The remaining two patients…

Researchers at UPenn have found a way to modify gene expression without modifying genome coding, opening the door for future possibilities of treatment for patients with Duchenne muscular dystrophy (DMD). The team of researchers have identified a group of small molecules that may aid in the  development of new therapies…

Personalized medicine has taken a big step forward with the launch of non-profit n-Lorem Foundation, which will create patient-tailored antisense oligonucleotide (ASO) therapeutics for people with rare diseases at no cost to the patients.  This comes at the same time as custom gene therapies for rare disease patients are being…

A new partnership was just announced between YourDNA and DNA ID to connect rare disease patients to genetic researchers in a safe, secure, and more efficient way. The two companies aim to help patients with rare genetic diseases form communities where they can find support and the chance to further…

CRISPR-Cas9 is best known for its powerful ability to make double-stranded breaks in DNA, allowing scientists to delete and edit genes with relative ease. But switch out Cas9 for another protein, and CRISPR becomes a programmable tool for detecting the presence of certain nucleic acid sequences. This feature has startups…

There is no doubt the age of wearable monitors is upon us. Sales of physical activity monitors have increased dramatically from 15 million in 2014 to 65 million in 2017, according to Statista.com. A 2017 study by Juniper Research estimates that the use of fitness trackers will double by 2021.…

A team of academic and industry researchers in California has developed a new technique that further demonstrates the utility of CRISPR-Cas technology in the diagnostic space. The researchers have developed a novel graphene-based biosensor, named CRISPR-Chip, that enables digital detection of a specific genetic mutation from a patient’s DNA sample,…

After scientists at the Wellcome Sanger Institute last month published findings that called the precision of CRISPR-Cas9 gene editing into question, three public companies focused on the technology saw their share prices fall to their 2018 lows before starting to climb back. Between March 9 and August 20: CRISPR Therapeutics…