The biotech industry boomed in 2020 due to development of COVID-19 medicines/vaccines as well as positive developments in other therapeutic areas too. The industry is expected to grow further due to increased financial support, following promising outcomes from studies on new innovative medicines for a variety of diseases.
Drug/biotech companies constantly strive to bring differentiated and high-quality medicines, vaccines and consumer healthcare products to as many people as possible.
To bring a successful drug on the market, these companies spend millions of dollars and several years in research and development (R&D) activities. However, many candidates fail in studies and never reach patients. Biotech, medical research and drug discovery involve significant expenditure. It is thus very important for the research landscape to change.
Major advances in science and technology are transforming the way scientists conduct research on diseases and are likely to improve diagnosis and treatment in the future.
Here we discuss three booming technological developments in the biotech sector, which will remain on investor radar in 2021. These are poised to transform the sector as they can save both time and cost.
Genomic editing using CRISPR technology to repair a defective genetic material that causes diseases is probably one of the most promising and exciting healthcare innovations seen in decades.
CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. The technology can selectively delete, modify or correct a disease-causing abnormality in a specific deoxyribonucleic acid segment. The technology has the potential to change how diseases, especially those caused by genetic mutations, are treated.
There are only a handful of companies making medicines using this revolutionary technology. Editas Medicine EDIT, Intellia Therapeutics NTLA and CRISPR Therapeutics CRSP are the three main companies making medicines employing CRISPR gene editing.
Editas’ lead pipeline candidate is EDIT-101 that employs CRISPR gene editing to treat LCA10 — a rare genetic illness that causes blindness. Editas completed dosing in the first cohort of the phase I/II BRILLIANCE study, which is evaluating EDIT-101 for LCA10. Editas is also pursuing the development of CRISPR candidates for eye diseases other than LCA10 including Usher Syndrome type 2A and the recurrent ocular Herpes Simplex Virus type 1.
Intellia is also developing therapeutics using the CRISPR system targeting genetic diseases, including transthyretin amyloidosis, and hereditary angioedema, various cancers and autoimmune diseases.
CRISPR Therapeutics’ lead pipeline candidate CTX001, a CRISPR gene-edited therapy, is being developed for treating sickle cell disease and transfusion-dependent beta thalassemia in collaboration with Vertex VRTX.
Other than CRISPR, zinc-finger nuclease (“ZFN”) and transcription activator-like effector nuclease are two primary gene-editing tools that are used today. Sangamo Therapeutics is a biotech that uses ZFN gene editing.
Monoclonal Antibodies/Antibody-Drug Conjugates
Monoclonal antibodies or mAbs are laboratory-produced antibodies that can bind to substances in the body including cancer cells. Once attached, they can force other parts of the immune system to destroy the cancer cells. Many copies of that antibody can be made in the lab. Monoclonal antibodies are used to treat many diseases, mainly cancer.
While monoclonal antibodies have become established as a new drug class for use in targeted cancer therapy, they are now being modified in new ways. They are now being used to make antibody-drug conjugates or ADCs, which are highly potent biological drugs. ADCs are mAbs, which are connected by a linker to antitumor cytotoxic molecules. The antitumor cytotoxic molecule is chemically linked to a mAb that recognizes a specific tumor-associated antigen, making the drug combination very specific.
ADCs are still in early stages of clinical application. However, with improved technology, the clinical application of ADC is rapidly accelerating and hold the potential to bring forth a new generation of cancer therapeutics.
At present, nine ADCs have received marketing approval, all for cancer indications including Pfizer’s PFE Mylotarg and Besponsa, Roche’s RHHBY Kadcyla and AstraZeneca/Daiichi Sankyo’s Enhertu. Several investigational ADCs are in pre-clinical and clinical studies and most of them are for cancer treatments. The first immunology ADC, ABBV-3373 is being evaluated by AbbVie ABBV in mid-stage development for rheumatoid arthritis.
Personalized or Precision Medicines
Precision medicine is the new mantra for healthcare companies, which allows tailoring of medical treatment to individual characteristics of each patient based on a patient’s molecular profile
The technological development improves the ability to diagnose and treat disease and also offers the potential to detect disease at an earlier stage, when it is easier to treat effectively. It can also reduce costs by doing away with a “trial-and-error” approach to disease treatment and also minimize side-effects.
Personalized medicine is benefiting patient care in mainly oncology indications like breast cancer (with trastuzumab) and melanoma (with vemurafenib). In 2021, precision medicine will likely continue to be focused on finding different treatments for oncological diseases. However, it is expected that use of precision medicine may increase in non-oncology areas like infectious diseases, central nervous system diseases, cardiovascular diseases, Alzheimer’s and Parkinson’s.
While larger companies such as Roche, Pfizer and Novartis are among the key pharma players in this space, some smaller private players are also entering the market.
Biggest Tech Breakthrough in a Generation
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