5 Top Gene Therapy Startups Impacting The Pharma Sector

We analyzed 235 gene therapy startups. StrideBio, Cardior, Generation Bio, Evox Therapeutics, and Mediphage Bioceuticals develop 5 top solutions to watch out for. Learn more in our Global Startup Heat Map!

Our Innovation Analysts recently looked into emerging technologies and up-and-coming startups working on solutions for the pharma sector. As there is a large number of startups working on a wide variety of solutions, we want to share our insights with you. This time, we are taking a look at 5 promising gene therapy startups.

Heat Map: 5 Top Gene Therapy Startups

Using our StartUs Insights Platform, covering 1.116.000+ startups & emerging companies, we looked at innovation in the field of gene therapy. For this research, we identified 235 relevant solutions and picked 5 to showcase below. These companies were chosen based on a data-driven startup scouting approach, taking into account factors such as location, founding year, and technology among others. Depending on your specific criteria, the top picks might look entirely different.

The Global Startup Heat Map below highlights 5 startups & emerging companies developing innovative gene therapy solutions. Moreover, the Heat Map reveals regions that observe a high startup activity and illustrates the geographic distribution of all 235 companies we analyzed for this specific topic.


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Which startups develop the other 230 solutions?


StrideBio – Adeno-Associated Viruses (AAV)-Mediated Gene Therapy

Viral gene therapy takes advantage of viruses’ natural ability to infect cells and deliver genes to cells. Multiple therapies for a variety of medical conditions employ viral vectors, such as adeno-associated viruses, that add, replace, or silence a particular gene to reverse a disease state. However, the host cells often attack viruses as they cause multiple diseases in their natural state. To prevent any adverse response by the patients’ cells, viruses are pseudotyped before application in gene therapies.

US-based startup StrideBio offers AAV-mediated gene therapy solutions. The structure-inspired adeno-associated virus vector engineering (STRIVE) platform improves on naturally occurring AAV serotypes to create unique and differentiated vectors. This enables improvements in tissue targeting and de-targeting, transduction efficiency, and manufacturability at scale. The startup’s pipeline includes drugs for genetic conditions such as Rett syndrome, Dravet Syndrome, and Friedrich’s ataxia.

Cardior – Noncoding RNA Therapeutics

Non-coding ribonucleic acids (ncRNAs) play a crucial role in many biological functions, particularly in gene regulation. Abnormal expression of ncRNAs has been linked with multiple diseases, such as cancers, cardiovascular diseases, and Alzheimer’s. Gene therapies that use synthetic oligonucleotides to restore normal expression levels offer promise in reversing such diseases.

German startup Cardior provides non-coding RNA therapeutics for patients with heart conditions. The startup uses AAV to deliver a synthetic ncRNA inhibitor that reduces aberrant expression and restores cardiac function. In parenteral application, the oligonucleotide therapy selectively blocks specific ncRNA.

Generation Bio – Non-Viral Gene Therapy

The size of the virus chassis is a constraint in viral vectors and results in issues related to delivering large genetic payloads. Moreover, viral gene therapy comes with the risks of inducing cancer or inflammatory reactions in some patients. Non-viral gene therapies overcome these challenges and bring the benefits of gene therapy successfully to more patients.

US-based startup Generation Bio offers non-viral gene therapy solutions. Their platform uses a proprietary closed-ended deoxyribonucleic acid (ceDNA) system that is capable of delivering large or multiple genes to a cell. By allowing larger loads, it also has the potential to offer durable gene expression that lasts many years. The startup’s pipeline includes drugs for liver and retina disorders that are in pre-clinical development and early stages.

Evox Therapeutics – Exosome Gene Therapy

Most eukaryotic cells produce exosomes, membrane-bound extracellular vesicles, that carry nucleic acids and proteins. Startups explore the use of this ability to carry a genetic payload as a method of delivering RNA or DNA for gene therapy. Since encapsulation within exosomes is independent of the nature of the payload, this becomes a universal delivery method and has potential applications in targeted drug delivery as well.

British startup Evox Therapeutics develops naturally inspired exosome gene therapy. The startup’s proprietary technology modifies exosomes to facilitate targeted delivery of nucleic-acid based agents, as well as proteins. The startup’s pipeline includes therapies for rare and inherited genetic diseases, such as lysosomal storage disorders (LSDs).

Mediphage Bioceuticals – Redosable Genetic Medicine

Like many other therapies, gene therapy also often requires multiple and repeat dosing to maintain the effectiveness of the treatment. Interventions like DNA ministrings improve the efficiency of gene delivery into cells and make redosable genetic medicine possible. Also, this allows for the development of scalable and personalized gene therapies that are readily commercialized as well.

Canadian startup Mediphage Bioceuticals specializes in redosable genetic medicine. The startup’s ministring DNA technology enables safe and effective personalized medicine. The startup is developing a curative treatment for Stargardt disease, an inherited ocular condition. In response to the global coronavirus pandemic, it is also developing a DNA vaccine targeting SARS-Cov-2, the causative virus for COVID-19.

What About The Other 230 Solutions?

While we believe data is key to creating insights it can be easy to be overwhelmed by it. Our ambition is to create a comprehensive overview and provide actionable innovation intelligence so you can achieve your goals faster. The 5 gene therapy startups showcased above are promising examples out of 235 we analyzed for this article. To identify the most relevant solutions based on your specific criteria, get in touch.

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