Pfizer, Novartis lead $2 billion spending spree on gene therapy production
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 with the company’s gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.
ProQR: Developing RNA Therapies for Rare Genetic Disorders
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 and is expanding its toolbox of RNA approaches to develop life-changing medicines for rare genetic disorders that are currently untreatable.
Arrowhead Hits the Mark
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 results.
“Squeezing” Cells Could Transform Cell Therapy Manufacturing
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 with a virus is the most expensive and time-consuming step. That’s according to Armon Sharei, PhD, the 32-year-old CEO of SQZ Biotech, whose cell engineering platform was named as a top 10 world-changing technology by Scientific American in 2014.
The Eyes Behind Surgical Robots
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 for laparoscopic surgery, in which a flexible optical instrument was inserted into the body and used to guide surgeons through hard-to-reach areas, from the pelvis to the chest cavity.
Silencing Novel Target Genes: A New Strategy for Lipid Lowering
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 for cardiovascular medicine.
Plano-based drug developer Reata raises $505 million, expects to double to 400 employees by end of 2020
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 genetic disease that can cause kidney disease and failure, and the other for Friedreich’s ataxia, a genetic disease that often begins in childhood and leads to brain and nervous system damage.
Novel RNA interference agents improve lipid profiles in healthy volunteers
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 other lipids, while another agent targeting ANGPTL3 (ARO-ANG3, Arrowhead Pharmaceuticals) was associated with reductions in ANGPTL3 and improvements in other lipids, researchers reported.