Epigenomic editing: A promising new frontier for biotech investors in 2023 and beyond

Epigenomic gene editing is an emerging field that has the potential to revolutionize the treatment of a wide range of diseases. Unlike traditional gene editing techniques that focus on modifying the DNA sequence of genes, epigenomic gene editing targets the epigenetic marks that regulate gene expression. These marks include DNA methylation, histone modifications, and non-coding RNAs. The epigenome is a complex system of chemical modifications that act as a switch to turn genes on or off. By modifying these marks, scientists can control gene expression without changing the DNA sequence itself. This makes epigenomic gene editing a potentially safer and more precise tool for treating genetic diseases.

One of the most promising applications of epigenomic gene editing is in the treatment of cancer. Cancer is caused by the accumulation of genetic mutations that disrupt the normal regulation of cell growth and division. Epigenomic gene editing can target the epigenetic marks that contribute to the development and progression of cancer, such as the hypermethylation of tumor suppressor genes. By modifying these marks, scientists can reactivate tumor suppressor genes and restore normal cell growth and division. This approach has shown promising results in preclinical studies and is being developed as a potential treatment for several types of cancer.

The advent of commercial epigenetic editing may not be too distant, as a handful of companies have emerged with this technology.

One such company is Navega Therapeutics, situated in San Diego, where researchers are investigating how to treat chronic pain by decreasing the expression of a gene known as SCN9A. This gene sends out numerous pain signals when it is highly expressed, but erasing it would be imprudent since some degree of pain is beneficial. It signals when something is amiss within the body. In rare cases, individuals with an inactive SCN9A mutation are unable to feel pain, rendering them vulnerable to injuries. Navega's experiments using epigenetic editing in mice seemed to suppress pain for a number of months.
Tune Therapeutics, led by Urnov, intends to employ epigenetic editing to address a wide range of conditions, such as cancer and genetic disorders. While Urnov does not view epigenetic editing as a solution to binge drinking, he believes that this study's proof-of-concept suggests it may be possible to modify gene experiences to reverse some of the harm caused by early alcohol abuse.

Another potential application of epigenomic gene editing is in the treatment of neurological disorders. Many neurological disorders, such as Alzheimer's disease and Parkinson's disease, are caused by the dysregulation of genes that are critical for brain function. Epigenomic gene editing can be used to modify these genes and restore their normal function, potentially offering a cure for these debilitating diseases.

The business Epic Bio was founded in July 2022 with an amazing $55 million Series A funding to overcome the barriers to CRISPR-Cas9 gene editing. The company, which is situated in San Francisco, California, is creating gene therapies based on altering the epigenome, a biological mechanism used by cells to regulate which genes are translated into proteins.

The treatments offered by Epic Bio combine a DNA-binding protein with a so-called modulator protein, which has the ability to alter the DNA molecule's epigenetic state. Using a unique guide RNA molecule, this construct is guided to a specific spot in the genome. The Gene Expression Modulation System, or GEMS for short, is the name of Epic Bio's technology.

A variety of rare disorders, including facioscapulohumeral muscular dystrophy, heterozygous familial hypercholesterolemia, and several types of retinitis pigmentosa, are being treated with Epic Bio's epigenome-editing medicines. Each time, the therapy is intended to undo damaging epigenetic alterations to the genes connected to the disease.

With $135 million in fresh funding, Chroma Medicine says it will use this year to advance its pipeline of single-dose medicines identified using the platform, present encouraging proof-of-concept evidence for the platform, and expand its workforce.

Chroma concluded its $135 million Series B funding earlier this month, boosting the company's total capital received to $260 million despite a frigid financing environment for early-stage biotechs.

The new funding granted recently in 2023, according to Chroma, will enable them to develop a platform for programmable epigenetic editors that can simultaneously change the expression of several genes or precisely turn genes on or off.

Modalis Therapeutics Corporation developed innovative products for the treatment of rare genetic diseases with its proprietary CRISPR-GNDM ® epigenetic editing technology and recently announced that the abstract has been accepted for presentation in the late-breaking session at The American Society of Gene & Cell Therapy (ASGCT)'s 26th Annual Meeting, which will be held in Los Angeles, California, on May 16-20, 2023. The abstracts provide preclinical data from the Company's LAMA2-CMD (LAMA2-Congenital Muscular Dystrophy) research.

BioIntel360 projects that epigenomic gene editing is an exciting and rapidly developing field that has the potential to transform the treatment of genetic diseases. While there are significant challenges and ethical considerations that must be addressed, the potential benefits of this technology are enormous. With careful development and responsible use, epigenomic gene editing could offer new hope for patients with a wide range of genetic diseases. Despite the potential benefits of epigenomic gene editing, there are also significant challenges and ethical considerations that must be addressed. One concern is the off-target effects of epigenomic gene editing, where unintended changes in gene expression could lead to unintended consequences, such as the development of new diseases.

Another source of concern is the possibility of using epigenomic gene editing for non-medical goals, such as improving cognitive or physical capacities. This raises ethical concerns concerning the employment of this technology, as well as the possibility of unexpected repercussions. To address these challenges, effective regulatory frameworks and guidelines for the development and use of epigenomic gene editing are required. Close coordination between scientists, regulators, and ethicists is required to guarantee that this technology is created and used responsibly and safely.