BIO 2015 #2 Gene Therapy and Antimicrobial Resistance (AMR) 6/26

This is a continuation of our previous update for BIO 2015 #1. Keep in mind these are notes not complete articles.

Gene Editing, CRISPR, CAR-T

Gene therapy is potentially the first major new drug discovery platform for Next Generation therapies since monoclonal antibodies. 70 cellular therapies are in the clinic and 10 are approved. We introduced this topic and some leading companies last October from the BIO Investor Forum-T-cell therapies. The lay press has covered this topic recently so brush-up your background with some research. Since October we have posted news on some of the gene therapy companies such as Bluebird Bio (BLUE), Kite Pharma (KITE) and Juno Therapeutics (JUNO). We attended two sessions from BIO 2015: “The Promise of Gene Editing” and “Roundup on Next Generation Biotherapeutics”. The basic concept of “gene editing” is that you can intercept disease at an early stage by re-programming the DNA of the cell. Up until now gene editing has been hard to use but methods have been recently invented known by the acronym Crispr-Cas9 which can bond to specific RNA molecules and insert a DNA sequence in the genome to prime the bodies defense system or repair broken genes. The result of the CRISPR technique is a “knock-out”gene which replaces a defective gene with a normal one. Editas Medicine is a private company focused in genome editing to develop a novel class of human therapeutics that enable precise and corrective molecular modification to treat disease at the genetic level. Editas is funded in part by the prolific Cambridge, MA Third Rock Ventures. Recently Editas did a partnership with Juno Therapeutics combining their technologies for treatment of cancer.

Poseida Therapeutics is another pre-clinical stage genome engineering company with two gene therapy platforms for cancer and liver diseases. They are partnered with Janssen (JLABS/Poseida) for CAR-T.

The gene editing and cellular therapy sessions were too complex and detailed to report further. The summary session was both cautious and optimistic about these innovative platforms comparing them to monoclonal antibodies which took a long time to mature and create blockbuster drugs. Paraphrasing-although there is no end to where the science can take us but it is important to have transparency and a science based regulatory framework.

Investment Incentives to Combat Antimicrobial Resistance (AMR)

From intro: “Globally, antimicrobial resistance has become one of the most significant public health issues, affecting all countries and causing increased hospitalizations, extended illness, and escalating healthcare costs.” For example in the MS Delta region there is ~4X the prescription rate of antibiotics compared to CA!

Despite the magnitude of the problem and dwindling therapeutic options there is limited investment interest in this area.There appears to be few economic incentives for drug companies to develop pipeline in this sector. Cubist Pharmaceuticals (CBST), previously on our focus list, was one of the few high-profile mid-caps in the sector was recently bought by Merck (MRK) for close to $10B. Better diagnostics are needed such as accurate and simple Dx test for Strep now on the market to help differentiate a serious infection from a sore throat and curtail antibiotic use.

There are limited medical options to deal with this problem but communication tools, real-time data and better surveillance are needed. One potential solution to stimulate investment in antibiotic research is the “Orphan Drug Model” which has been successful in rare diseases. Also legislative action and new reimbursement models need to be considered.