A man who lives in Surprise is helping researchers come up with a treatment for a rare disease that he was diagnosed with 20 years ago. Jerry DeVries, now 61, is possibly the oldest living person with Proteus Syndrome.
Rather than picking targets based on conventional approaches, Immunome is letting a patient‘s ability to fight the disease guide target selection and therapy identification.
We are always acutely aware of investors’ appetite to participate in financing deals, and to what degree. There has been a lot of chatter about the “window closing” in 2020, but from what we can see, financing deals for good science and novel ideas are still getting done, and we’re cautiously optimistic that the [...]
Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world’s priciest medicines. The full scope of Novartis’ (NOVN.S) $500 million plan, revealed to Reuters in an interview [...]
There is a great need for transformative treatments that increase life expectancy and improve the quality of life of patients and families living with rare inherited diseases. Current therapeutic approaches, including small molecules and enzyme replacement therapies, have limitations and cannot be applied to every disease. ProQR specializes in the development of RNA therapies [...]
As of Nov. 21, Arrowhead stock is trading at a record high of $49 a share. It has a new slate of drugs that use slightly different molecular technology to silence disease-causing genes and is targeting a range of different diseases. Several of those drugs are now going through clinical trials with promising early [...]
Manufacturing today’s cell therapies remains expensive and complex due to the need for viruses or electric shocks to engineer patient cells. Yescarta®, for example, one of the first CAR-T therapies approved for sale, takes 3-4 weeks to reach patients and has a price tag of $373,000.00. With CAR-T, expanding out a patient’s T-cells and transducing them [...]
The first robotic surgery took place in 1985 when the PUMA 560 was used in a stereotaxic operation in which computed tomography (or x-ray) was used intraoperatively to guide a robot as it inserted a needle into the brain for biopsy. In the late 1980s and early 1990s, robotic systems began to be used [...]
Two more novel lipid-lowering therapies based on silencing specific genes involved in lipid metabolism have shown promising preliminary results in initial clinical trials. The idea of using small pieces of RNA, known as small interfering RNA (siRNA) specific for a certain gene to switch off the activity of that gene is a new area [...]
Plano-based drug developer Reata Pharmaceuticals Inc. raised more than $505 million in a stock sale Monday to speed its transition from a clinical-stage biotech startup to a company that makes treatments for life-threatening diseases. The publicly-traded company will use the money to register and commercialize its first two treatments: one for Alport syndrome, a [...]
Two novel RNA interference agents, one targeting apolipoprotein C-III and one targeting angiopoietin-like protein 3, improved various lipid parameters in early studies with healthy volunteers, according to new data presented at the American Heart Association Scientific Sessions. One agent targeting apolipoprotein C-III (ARO-APOC3, Arrowhead) was associated with reductions in serum APOC3 and improvements in [...]
Valneva chikungunya vaccine candidate VLA1553 was generally safe in all dose groups and the local tolerability profile was excellent.
The FDA’s decision to approve Talicia could have far-reaching impact, as this bug infects millions of American adults a year and is one of the strongest known causes of stomach cancer. Talicia is a three-drug combination of omeprazole, a proton pump inhibitor, amoxicillin, a penicillin-class antibacterial, and rifabutin, a rifamycin antibacterial. It is indicated [...]
Family and friends gathered at the University of Miami Life Science and Technology Park Wednesday morning to remember Tuly Gonzalez on what would have been her 32nd birthday. She died 11 years ago but the gathering was not just to remember her, but to also celebrate the lives she has impacted though her donation. [...]
In this exclusive video from The Liver Meeting 2019, Tarek Hassanein, MD, FAASLD, professor of medicine at UC San Diego Health and medical director of Southern California GI and Liver Centers, discusses results of a phase 2a study, which demonstrated that DUR-928 was well-tolerated and effective in patients with acute alcoholic hepatitis.
Hepatitis used to be the talk of the town at The Liver Meeting, the annual gathering of the American Association for the Study of Liver Diseases. These days, the conversation is turning to nonalcoholic steatohepatitis (NASH), but its cousin alcoholic hepatitis (AH) isn’t getting much attention. Durect is trying to change that. [...]
The interim results come from an ongoing phase I trial in patients with colorectal cancer. Of the 12 patients given Celyad’s CAR T-cell immunotherapy so far, six have seen their tumors shrink. The therapy has also shown no evidence of causing graft-versus-host disease, a condition where foreign immune cells attack the host body. Celyad’s CAR T-cell immunotherapy [...]
In the OPTIC trial, patients with wet age-related macular degeneration who were treated with ADVM-022, an intravitreal gene therapy, did not require additional injections following treatment, according to a presenter at the American Academy of Ophthalmology annual meeting.
The 28-patient study showed that Angion's drug improved kidney function, based on patients’ estimated glomerular filtration rate (eGFR), a key renal function metric.
Arrowhead Pharmaceuticals (ARWR) and Johnson & Johnson (JNJ) said Friday they eradicated nearly all traces of hepatitis B virus in a Phase 2 study — sending Arrowhead stock careening toward a record high. On the stock market today, Arrowhead stock jumped 6.3% to 42.10. On Monday, shares hit a record high in intraday action of 42.29. Arrowhead stock broke out on [...]
ProQR Therapeutics is a Netherlands-based company currently researching RNA therapies to inhibit or counteract the harmful effects of the defective genes associated with the above retinal diseases. How does that work? As you may recall from biology class, genes are the building blocks for the proteins that build and maintain our bodies. [...]
There is amazing new technology that’s helping surgeons perform the most delicate of all operations — brain surgery. It saved the life of a Westchester County teenager whose headaches were from a brain tumor, CBS2’s Dr. Max Gomez reported Tuesday.
Neurosurgery is often thought of as one of the most difficult medical disciplines to pursue. Surgeons are tasked with breaching the multiple protective layers of the brain then navigating through an intricate web of fibers, tissues, blood vessels and fluid to extract a tumor or implant a device — all this without harming the [...]
Product-release testing is required for all drug products to ensure their quality and safety. For most conventional small-molecule and biologic drugs, batches are held until the release testing results are received and confirm the product is within specifications. Cell and gene therapies and some of their components (e.g., viral vectors), however, often must be [...]
Arrowhead Pharmaceuticals (ARWR) is using a technology called RNAi to tackle two colossal hurdles in treating genetic diseases. And it's helping Arrowhead stock to finally realize profits after the biotech company operated for years in the red. RNAi, or RNA interference, is a method of silencing genes. Some genetic diseases cause the overproduction of [...]
Biopharmaceutical executives, especially those who have founded their own companies, never cease to amaze me. Why? Well for starters, the process of starting with nothing but an idea and then pulling together all the necessary resources to build a company capable of developing and delivering a product to patients is an amazing logistical challenge. [...]
In the early 2000s, chimeric antigen receptor T (CAR-T) cells emerged on the scene with promise as a revolutionary cancer treatment. However, several key challenges and, in particular, downsides related to manufacturing, continue to hamper broad adoption of this therapeutic approach. In addition, existing CAR-T cell therapies are based on autologous approaches (i.e. using the [...]
Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics. Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene. With the designation, the drug will receive priority review by the FDA, and it [...]
At a medical mycology meeting here earlier this month, scientists presented promising phase II clinical data for rezafungin, a new member of an existing class called the echinocandins developed by Cidara Therapeutics in San Diego.
