Exosomes are small vesicles that are naturally released by almost all cell types in the body and play a key role in intracellular communication. Exosomes transport a diverse cargo of molecules such as proteins, lipids, mRNA, and miRNA between cells. Due to their nanoscale size of 30-150 nm and natural ability to fuse with target cells, scientists view exosomes as promising drug delivery vehicles for therapeutic applications.
Exosome Therapeutics: Developing Vesicle-Mediated Therapies
Founded in 2011, Therapeutics is a biotech company focused on engineering exosomes for targeted drug delivery. The company was one of the earliest to realize the potential of harnessing Exosome Therapeutics as drug delivery vehicles. Therapeutics’ platform allows loading therapeutic molecules into purified exosomes from donor cells. These exosome-based therapies can then be administered directly to patients.
The company's therapeutic focus areas include neurodegenerative diseases, cardiovascular diseases, and oncology. Exosome Therapeutics has ongoing clinical and preclinical pipelines in Alzheimer's disease, Parkinson's disease, myocardial infarction, and cancers. Some of the company's most advanced programs are:
- EXO-AAV-9 for Alzheimer's disease and Parkinson's disease: This program utilizes exosomes to deliver AAV9 gene therapy vectors across the blood-brain barrier. Promising preclinical data shows corrective genes are delivered to the brain and spinal cord cells.
- EXO-Ang1 for myocardial infarction: Angiopoietin-1 protein is encapsulated in exosomes to promote angiogenesis and cardiac repair after a heart attack. In animal models, a single dose showed reduced scarring and improved heart function.
- EXO-PDOX for pancreatic cancer: This program delivers tumor-suppressing miRNA-loaded exosomes to inhibit pancreatic tumor growth and metastasis. In mouse models, a significant decrease in tumor progression and longer survival times were observed.
Partnering For Clinical Advances
While pursuing its own pipeline, Therapeutics has also formed strategic partnerships to accelerate clinical development. In 2016, the company partnered with Celgene to develop exosome targeting multiple myeloma and other hematologic tumors. Under the multi-year collaboration, Celgene gained options to license certain exosome-based pipeline assets in oncology.
In 2018, Exosome partnered with Neurotrack Technologies to advance the EXO-AAV-9 program in Alzheimer's disease. This partnership combined Neurotrack's advanced digital biomarkers with Exosome's ongoing clinical trial. The collaboration aims to improve patient selection criteria and outcome measures for future AD trials.
Fueling Pipeline Growth With New Financings
To sustain its progress, Exosome has raised significant capital through private financing over the years. In 2015, the company raised a $70 million series C round led by life sciences investors OrbiMed Advisors and Novo A/S. This strengthened the company’s financial position and supported a growing pipeline then consisting of five clinical and preclinical programs.
The company completed its largest fundraising round of $100 million series D in 2017 led by RA Capital Management and OrbiMed Advisors. RA Capital’s chairman Peter Kolchinsky joined Exosome’ board of directors. The funds were intended for the company’s clinical and manufacturing activities towards transitioning into a commercial-stage organization.
Securing Assets Through Mergers
In 2018, Exosome Therapeutics merged with Chicago-based startup Materialise to form the new company Ascendia Pharmaceuticals. Materialise contributed proprietary nanoprinting technologies capable of industrial-scale production of individualized exosome-therapies.
Through this merger, Ascendia Pharmaceuticals acquired complementary drug development and manufacturing capabilities from both parent companies. With combined resources and expertise, the merged company aims to accelerate clinical testing and commercialization of its engineered exosome therapies across therapeutic areas. The merger positioned Ascendia to become a leader in developing and commercializing therapeutics at industrial scales.
With an experienced management team, growing research collaborations, and strong funding support, Ascendia Pharmaceuticals is well positioned at the forefront of exosome therapeutic development. The company's pioneering work in harnessing nature's nanoparticles for drug delivery holds promise to transform treatment for diseases with high unmet needs. If clinical trials continue producing safety and efficacy data, some of Ascendia’s lead programs may reach s in the next 5 years. This can establish engineered exosome therapies as a viable therapeutic modality and generate appealing commercial opportunities.
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*Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
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