Relay Therapeutics & Crispr Therapeutics
Spotlight on two promising companies
BB Biotech anticipates that 2021 will bring significant technology progress and new products that address important unmet medical needs. In addition to established treatment areas such as oncology, orphan diseases and neurological indications, attention will increasingly center on rapidly developing promising technologies that offer tremendous economic value thanks to their excellent therapeutic profile. Bearing that in mind, we cast a spotlight on two companies that investors should be following.
Relay Therapeutics: On-screen molecular design
An innovative treatment approach emerging from biotech laboratories is called molecular design. Relay Therapeutics, which was floated on the stock exchange in July 2020, is a leader in rational drug design. The process involves studying the movement of protein molecules to understand the role of protein motion in causing disease. Relay uses a computational platform instead of conventional three-dimensional crystal structures. Machine learning is used for digital simulation and visualization of chemical and biophysical processes that occur on a timescale of milliseconds. This is a faster and cheaper way to identify molecules with potential to be developed as drugs. What’s more, molecule design allows more customized mechanisms of action because properties like pharmacodynamics, effective dose, bioavailability and toxicity can all be tweaked much more precisely.
«Relay Therapeutics will present topline data
for two products in 2021»
Relay Therapeutics is using this technology to develop oral medications to treat cancer. The most advanced candidate, RLY-1971, is an SHP2 inhibitor mainly being tested for use in combination with other drugs. RLY-1971 is currently in the Phase I clinical trial stage. Relay will present topline data for two products in 2021.
BB Biotech added Relay Therapeutics to its portfolio in the third quarter of 2020 by participating in its successful IPO and bought more of the company's shares on the open market immediately after they were listed.
Crispr Therapeutics: Crispr fixes genetic defects for good
The awarding of the Nobel Prize in Chemistry to Emmanuelle Charpentier and Jennifer A. Doudna in October 2020 underlines the importance of genome editing as a disruptive technology for future drug development. This molecular biological tool is used to make targeted changes in the human genome to cure diseases permanently. Genome editing is already being used with success in cell-based cancer therapies. In contrast, Crispr Therapeutics is a leading exponent of ex-vivo applications of the Crispr/Cas9 approach. This involves taking cells from a patient, modifying them in a lab and re-administering them to the patient. The novelty of the approach is that fragments of human DNA identified as genetic triggers of disease are cut out and repaired using genetic replacement parts. The Crispr/Cas9 enzyme activates the genetic repair mechanism which every somatic cell possesses.
«The genetically modified stem cells that were administered back to the patients led to a complete cure»
BB Biotech has been invested in Crispr Therapeutics since the first quarter of 2019. The company is pursuing two clinical trials in beta thalassemia and sickle cell anemia in cooperation with Vertex Pharma, a core position in BB Biotech's portfolio. Both diseases are inherited genetic disorders that result in the defective formation of blood cells with severe progression. The genetically modified stem cells that were administered back to the patients led to a complete cure. In addition to these two clinical trials, Crispr Therapeutics is pursuing three cell-based projects in immuno-oncology on its own.