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2021-02-12| FundingStartups

Ensoma Enters Gene-Editing Market with $70 Million Funding and Takeda Partnership

by Ruchi Jhonsa
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The ability to manipulate the genetic makeup of a human cell has come off age with various gene-editing technologies, including the Nobel Prize-winning CRISPR technology. So far, two FDA-approved therapies, including Roche’s Luxturna and Novartis’s Zolgensma, have received the FDA’s blessings, and a few more genetic medicines for a range of diseases, including hemophilia, sickle cell disease, and several muscular dystrophies are in the offing. But as more and more gene therapies are entering clinical testing, the limitations of current viral vectors that transport the therapies inside the cell are becoming more apparent.

In that wake, Ensoma Therapeutics, co-founded by a world-renowned oncologist and pioneer in gene-editing technologies, Dr. Hans-Peter Kiem, is developing immune resistant and spacious vectors that can deliver a diverse range of genomic modification therapies, including the bulky ones directly to hematopoietic stem cells or various cell types that arise from stem cells including, T cells, B cells and myeloid cells in vectors.

Looking at the potential of these vectors dubbed as Engenious, Ensoma has been granted a series A funding of $70 million and a deal with Takeda potentially worth $100 million on February 11th. The financing round was led by co-founder and seed investor 5AM Ventures along with F-Prime Capital, Takeda Ventures, Viking Global investors, RIT Capital Partners, Symbiosis II, LLC, and Alexandria Venture Investments.

Ensoma plans to use Engenious vectors to develop therapies that are “accessible to all.” Paula Soteropoulus, Executive Chair of Ensoma, explains how. “Because our in vivo therapies do not require prior conditioning or stem cell donors, we hope to deliver them as ‘off-the-shelf’ treatments to address diseases – both rare and common – dramatically simplifying the logistics of scaling production and reducing patient and healthcare-system burden. Every person, no matter where they are in the world, should have access to the innovative technologies that are changing the way we treat disease,” Paula Soteropoulos, Executive Chair of Ensoma, said in a statement.

 

Ensoma’s ENGENIOUS Platform

Current gene-editing technologies rely heavily on adenovirus-derived vectors or AAVs to shuttle genetic modifiers into the body’s cells. AAVs can be easily engineered and administered in a human cell and have proven safe in clinical testing. But they have limited space and can carry only a small genetic cargo inside them.

Ensoma is trying to overcome some of these challenges by stripping AAV vectors of its genetic material-a modification with several advantages. Modifying the AAV vector this way frees up space within the vectors to accommodate large and varied genetic payloads. Moreover, getting rid of viral RNA or DNA reduces the chance of immunogenicity that AAV vectors are known to induce in the host system.

Engenious vectors can preferentially target HSCs inside the body. But if the targeting is inefficient, then Ensoma has an in vivo selection system to increase the levels of modified HSCs. This method increases the effect of the genetic modifiers without the need of re-dosing the patients- something that has immunogenic challenges associated with it.

These vectors have already been tested in preclinical studies. A study published by the company showed their approach to treat beta-thalassemia- a condition where blood cells have low hemoglobin levels- using Engenious vectors. In the mice study, hematopoietic stem cells were first moved out of the bone marrow in the blood, where they were modified using the vector. The cells were then returned to the bone marrow, where they persisted, proliferated, and produced correct red blood cells.

“There have been tremendous advancements in technologies to precisely target, genetically edit and modify human disease. However, many of these tools pose delivery challenges; some lack the ability to reach the right cells within the body, while others lack the ability to broadly reach significant numbers of patients due to complex procedures and supply chain challenges,” said Kush M. Parmar, M.D., Ph.D., founding chief executive officer of Ensoma. “Ensoma’s scientific approach allows us to do what hasn’t been done before—to make the curative power of genomic medicine and stem cell technology portable so they may be administered in low-resource and outpatient settings for the very first time.”

 

New Entrants in the Vector Space

A number of companies are trying to solve the problems of existing vectors. Last year, Harvard spinout Dyno therapeutics bagged a $1.8 billion deal with Roche to develop novel AAV capsids with improved properties such as larger cargo space and stronger immune resistance.

4D Molecular Therapeutics is also working on evolved vectors that are derived by combining the power of directed evolution with billions of synthetic capsid sequences. The company bagged $222 million in an initial public offering to advance its vector platform. Besides the smaller biotechs, the larger companies are also invested in making better vectors. For example, Sarepta and Novartis have partnered with Dyno to advance their gene therapies.

Related Article: Proteomics Company Nautilus Bio to List on NASDAQ via SPAC

References

  1. https://www.businesswire.com/news/home/20210211005185/en/Ensoma-Launches-to-Pioneer-Next-Generation-In-Vivo-Approach-to-Deliver-First-%E2%80%9COff-the-shelf%E2%80%9D-Genomic-Medicines

 

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