Codiak’s Versatile engEx™ Platform for Engineered Exosomes Published in the Journal Molecular Therapy

Rhea-AI Summary

Codiak BioSciences (CDAK) announces the advance online publication of a manuscript detailing the identification of two novel exosome-associated proteins, PTGFRN and BASP1, essential to their engEx Platform. This platform is designed to harness exosomes for therapeutic applications across various diseases. The study shows that these proteins allow high-density incorporation of biologically active molecules, enhancing targeting and potency in preclinical models. Codiak is pursuing engineered exosomes with promising clinical data anticipated for exoIL-12 and exoSTING in mid-2021.

Positive

• Advance publication of PTGFRN and BASP1 proteins enhances the utility of engineered exosomes.
• Demonstrated ability to deliver biologically active molecules effectively, suggesting improved therapeutic outcomes.
• Initial clinical results show tolerability for exoIL-12; safety and efficacy results expected in mid-2021.

Negative

• None.

– New publication details the characterization of novel scaffold proteins that can enable engineered exosomes with defined therapeutic properties –

– engEx Platform designed to harness exosomes as a new class of biologic medicines for a wide spectrum of diseases –

CAMBRIDGE, Mass., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Codiak BioSciences, Inc. (NASDAQ: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, today announced the advance online publication of a new manuscript. The paper, entitled “A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties,” by Dooley et al, will appear in an upcoming print issue of Molecular Therapy, the journal of the American Society of Gene and Cell Therapy. The publication describes the identification and characterization of two novel exosome-associated proteins, PTGFRN and BASP1, that enable Codiak’s proprietary engEx Platform and facilitate precise design and engineering of exosomes for potential therapeutic purposes.

PTGFRN and BASP1 are highly abundant, naturally-occurring proteins that, through Codiak’s research, have been found to enable high-density exosome surface display and luminal loading of a wide range of macromolecules that can be directed to specific target cells of interest. In preclinical models, the use of PTGFRN and BASP1 to display biologically active molecules on the surface of or inside exosomes, respectively, resulted in uniform incorporation and increased potency, suggesting the potential utility of these engineered exosomes in a wide variety of therapeutic settings. These two proteins can also be used in combination, which further broadens the utility and modular approach to designing engineered exosomes for targeted delivery of numerous drug payloads to specific cells.

“The identification and characterization of PTGFRN and BASP1 by our scientific team provided us with versatile scaffolds to precisely and reliably design exosome therapeutic candidates with intentionally chosen properties, thus forming the backbone of our engineering platform,” said Douglas E. Williams, Ph.D., President and Chief Executive Officer of Codiak. “The ability to functionalize exosomes with diverse macromolecules at a high density represents a significant advance toward unlocking the therapeutic potential of these extracellular vesicles.”

To identify potential scaffold proteins for engineering exosomes, Codiak scientists first developed a methodology to stringently purify a population of exosomes. Detailed characterization of these high-purity exosomes enabled the identification of exosome-specific proteins, PTGFRN and BASP1. When used as scaffold proteins to incorporate biologically active payloads either on or in exosomes, PTGFRN and BASP1 were shown to be superior to previously reported exosome scaffolds with respect to copy number and uniformity. These proteins enabled functional display of cytokines, antibodies/fragments, TNF superfamily members, enzymes, RNA binding proteins and vaccine antigens, all of which retained pharmacodynamic activity in vivo.

Codiak is advancing a broad pipeline of product candidates generated using its engEx Platform. Codiak’s most advanced programs use PTGFRN to display biologically active molecules and/or direct tropism to specific cellular targets for precise delivery of drug payloads. Codiak’s two clinical programs, exoIL-12™ and exoSTING™, are the first engineered exosomes to be evaluated in humans. Initial results from the Phase 1 exoIL-12 program demonstrated tolerability and absence of systemic IL-12 exposure in healthy volunteers. Safety, biomarker and preliminary anti-tumor efficacy results for both clinical programs are anticipated in mid-2021.  

About the engEx™ Platform
Codiak’s proprietary engEx Platform is designed to enable the development of engineered exosome therapeutics for a wide spectrum of diseases and to manufacture them reproducibly and at scale to pharmaceutical standards. By leveraging the inherent biology, function and tolerability profile of exosomes, Codiak is developing engEx exosomes designed to carry and protect potent drug molecules, provide selective delivery and elicit the desired pharmacology at the desired tissue and cellular sites. Through its engEx Platform, Codiak seeks to direct tropism and distribution by engineering exosomes to carry on their surface specific targeting drug moieties, such as proteins, antibodies/fragments, and peptides, individually or in combination. Codiak scientists have identified two exosomal proteins that serve as surface and luminal scaffolds. By engineering the exosome surface or lumen and optimizing the route of administration, Codiak aims to deliver engEx exosomes to the desired cell and tissue to more selectively engage the drug target, potentially enhancing the therapeutic index by improving potency and reducing toxicity.

About Codiak BioSciences
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of diseases, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease. For more information, visit http://www.codiakbio.com and follow @CodiakBio.

Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, among other things, statements regarding the capabilities and potential of Codiak’s engEx Platform and engineered exosomes generally and statements concerning the development and therapeutic potential of exoSTING and exoIL-12, including timing of release of data. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. For a discussion of these risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Codiak’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and in subsequent filings with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors in Codiak’s subsequent filings with the Securities and Exchange Commission. All information in this press release is current as of the date of this report, and Codiak undertakes no duty to update this information unless required by law. 

Investor Contact
Christopher Taylor
VP, Investor Relations and Corporate Communications
T: 617-949-4220
E: investor@codiakbio.com

Media Contact
Lindy Devereux
Scient PR
T: 646-515-5730
E: media@codiakbio.com

FAQ

What is the significance of PTGFRN and BASP1 for CDAK?

PTGFRN and BASP1 are crucial in Codiak's engEx Platform, allowing precise engineering of exosomes for targeted therapies.

When will clinical results for exoIL-12 be released?

Safety, biomarker, and preliminary anti-tumor efficacy results for exoIL-12 are anticipated in mid-2021.

What does the engEx Platform do?

It enables the development of engineered exosome therapeutics aimed at various diseases.

What are the therapeutic applications of Codiak's engineered exosomes?

They are designed to treat a wide range of diseases, including oncology and neurology.

What is the potential of Codiak's exosome-based therapies?

Codiak's therapies may transform treatment across several diseases with high unmet medical needs.