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Driving Drug Discovery Using Patients’ Immune Response

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.

IPO Window, Recession, Trade War & More — 5 Biopharma Finance Experts Share Their Perspectives For 2020

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 environment will remain open to healthy deals at the right valuations.

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.