The patients had a genetic disease called adenosine deaminase–deficient severe combined immune deficiency (ADA-SCID) — also known as bubble boy syndrome. Caused by a mutation on a vital gene, the disease weakens the immune system to the point where patients are very vulnerable to day-to-day infections.
Israel-based Pluristem Therapeutics is hoping to do via a collaboration with NASA. The biotech, which focuses on developing stem cell therapy products, is partnering with the space agency’s Ames Research center to see whether or not its experimental PLX cell therapies could help treat and prevent various medical conditions that may arise during space [...]
When in space, your inner machinery can go awry — ask astronaut Scott Kelly. The assault of protracted radiation and microgravity can culminate in progressive bone and muscle loss and vex the immune system. Placenta-based stem cells may be the answer to limiting that damage, and Israel’s Pluristem is working with NASA to develop such an [...]
It was about ten months ago that Orchard Therapeutics acquired the gene therapy portfolio of GSK, expanding its pipeline and giving it its first approved therapy. We spoke to Orchard CEO Mark Rothera about his company’s approach to gene therapy, its pipeline, and what he sees as the biggest challenges as Orchard moves towards [...]
NEW YORK (NBC News) A peek inside the Friednman family's kitchen leaves one seeing double. There are two microwaves, two toasters and two different pantries. It's how the family separates the family's meals from the gluten-free foods necessary for their 15-year-old son Tyler, who has celiac disease. "He can go in there and get [...]
“The definition of blockbuster is changing,” Fouzia Laghrissi-Thode, MD, chief executive officer of DalCor Pharmaceuticals, tells GEN. “Now it’s not necessarily just a dollar sign [in front of a revenue figure] that determines a blockbuster.” As drug development and regulatory reviews become more efficient, she suggests, lesser revenues can generate blockbuster-scale returns on investment. [...]
Biotech company OncoCyte is using a liquid biopsy to detect lung cancer that is showing promising results. Yahoo Finance’s Alexis Christoforous speaks to William Annett, the CEO of Oncocyte.
It’s the ultimate example of personalized medicine — for some cancer patients who have run out of options, scientists can extract their immune cells and genetically re-engineer them to fight against the tumors.
A set of positive results validating OncoCyte’s liquid biopsy test has bolstered its stock $OCX, up 221% at press time. DetermaVu scored on both sensitivity — capacity to detect true positives — and specificity, the ability to snuff out negatives, in a blinded trial that involved 250 blood samples from lung cancer patients.
Micro-cap diagnostic company OncoCyte Corp tripled intraday after announcing break-through results for its lung cancer test that won praise from Wall Street. The company’s R&D validation study of DetermaVu, a non-invasive blood test, “significantly outperforms any other previous blinded study of its kind and signifies the first blood based test on the market to use strictly biomarkers for [...]
Isofol Medical AB, a small Swedish company founded a decade ago, is seeking to improve the standard of care (SOC) for metastatic colorectal cancer (mCRC), an oncology indication that receives far less attention than metastatic forms of more prevalent breast, prostate and lung cancers, said CEO Anders Rabbe.
Instead of opening up a clinic to perform transfusions of young blood, Alkahest researchers aim to develop drugs for age-related diseases which are inspired by their work with plasma.
Meanwhile, Novartis's crosstown rival Roche Holding AG has enlisted privately held SQZ for up to $1 billion in milestone payments. SQZ created a device that constricts cells to the point where small holes are formed in their outer membranes, allowing genetic material to pass through. The powered-up cells are then able to recognize antigens [...]
Vaccine maker Valneva has some experience pumping up a Big Pharma also-ran, and now it's looking for more.
The company, which is working on drugs based on antisense oligonucleotides to treat genetic conditions, announced a reverse merger with Ohr Pharmaceutical on Jan. 3, which would allow its stock to trade publicly.
Dietrich Stephan, one of the pioneers in the field of precision medicine, was in process of raising venture funds for his gene-silencing company NeuBase Therapeutics when he was introduced to the team at the struggling eye drug developer Ohr Pharmaceutical $OHRP.
CBD oil falls into a gray area that might be legal in some states now because it is not psychoactive.
Leslie Williams, CEO of ImmusanT, discusses the company’s research for developing a disease-modifying therapeutic for patients with celiac disease.
Check-Cap received CE mark for its C-Scan Capsule in January of this year, now the Isfiya, Israel-based company is setting its sights on the U.S. market.
Talicia, a novel drug for the treatment of Helicobacter pylori, achieved positive top-line results in a phase 3 study, according to a press release from RedHill Biopharma, the manufacturer.
Orchard Therapeutics said it will build out a gene therapy manufacturing facility in Fremont, CA, that will employ 100 people and expand its manufacturing capabilities to support plans for an expanded pipeline.
Check-Cap (NSDQ:CHEK) said today it won conditional approval from the FDA for its investigational device exemption application to launch a pilot study of its C-Scan system. The Israel-based company’s C-Scan system is designed as an alternative to standard colon cancer screening methods.
Orchard Therapeutics is expanding its physical footprint in California. The U.K.-based company signed a long-term lease to build out a gene therapy manufacturing facility in Fremont, Calif. The 150,000-square-foot facility will add to the company’s presence in the Bay Area.
Dr. Patrik De Haes, CEO, Oxurion discusses the need for new treatments for diabetic eye disease, a leading cause of blindness in people of working age worldwide.
A single dose of a gene therapy developed by Orchard Therapeutics can restore immune function for at least a year in patients with X-linked chronic granulomatous disease (X-CGD).
Alkahest has dosed the first subject in a Phase II clinical trial of GRF6021 to treat Parkinson’s disease associated with mild cognitive impairment or dementia.
Patrik De Haes, MD, CEO of Oxurion NV, shares the latest updates in clinical studies at the OIS@AAO meeting in Chicago, Oct. 2018.
RedHill Biopharma Ltd. plans to submit an NDA to FDA next half for Talicia (RHB-105) as first-line treatment of Helicobacter pylori infection regardless of ulcer status after reporting that the antibiotic met the primary endpoint in the Phase III ERADICATE Hp2 trial in the indication.
A European/US company collaboration has reported encouraging safety and immunogenicity in early clinical studies with an alum-adjuvanted Zika vaccine based on inactivated whole virus.
