CRISPR’d Stem Cells Could Provide Enduring Therapy for Muscular Dystrophy

GEN | September 18, 2019

In the past several years, we have seen some major advancements in the study of Duchenne muscular dystrophy (DMD), a rare but devastating genetic disorder that causes muscle loss and physical impairments. Investigators using the gene-editing tool CRISPR have shown in previous studies, in rodent and canine models, that the repair or removal of key disease mutations is possible, as well as improvement of muscle function. Now, in a new study from researchers at the University of Missouri (MU) School of Medicine scientists have shown that employing CRISPR to edit mutations in muscle stem cells (MuSCs) may provide the means for lifelong correction of the genetic mutation responsible for the disorder. “Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model,” explained senior study investigator Dongsheng Duan, PhD, a professor in the department of molecular microbiology and immunology at the MU School of Medicine. “However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.”

Spotlight

Adeno-associated viruses (AAV) are virus particles composed of single-stranded DNA surrounded by a protein shell. Despite their simple structure, recombinant AAVs (rAAV) can perform the important role of delivering nucleic acids into cells during gene therapy.

Spotlight

Adeno-associated viruses (AAV) are virus particles composed of single-stranded DNA surrounded by a protein shell. Despite their simple structure, recombinant AAVs (rAAV) can perform the important role of delivering nucleic acids into cells during gene therapy.

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INDUSTRIAL IMPACT

Psycheceutical Completes Acquisition by Blue Water Ventures International

Blue Water Ventures International | January 20, 2022

Psycheceutical, Inc. a biotechnology company dedicated to the development and commercialization of psychedelic medicines, announced the company has been fully acquired by Blue Water Ventures International, Inc. “We are thrilled that this deal has closed and that Psycheceutical is now a wholly-owned subsidiary of a publicly-listed company and has secured important near-term capital to pursue our growth plans. We will immediately begin the next steps of our growth strategy as we work to bring our revolutionary psychedelic drug delivery technologies to the market, starting with initial clinical trials.” Psycheceutical CEO Chad Harman BWVI acquired Psycheceutical, Inc. by way of a merger, resulting in Psycheceutical becoming a wholly-owned subsidiary of BWVI. Now that the acquisition has occurred, BWVI intends to cease its previous business activities and the company will focus solely on psychedelic drug development. Once regulatory approvals have been received, the company will consummate related corporate actions, including a name and symbol change. About Psycheceutical, Inc. Psycheceutical, Inc. is seeking to develop cutting-edge delivery technologies for safe and effective psychedelic pharmaceutical medicines. Powered by a team with more than 100 years' combined experience in development, regulatory approval processes and commercialization across the pharmaceutical industry, Psycheceutical is on a mission to bring safety and efficacy to psychedelic compounds., today announced the company has been fully acquired by Blue Water Ventures International, Inc.

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CELL AND GENE THERAPY

Curi Bio Closes $10M Series A in Oversubscribed Round

Curi Bio Inc. | December 30, 2021

Curi Bio Inc., a leading developer of human stem cell-based platforms for drug discovery, announced the second closing of a $10 million oversubscribed Series A financing. New investors include UTC Investment and DS Asset Management, joining current Curi Bio investor and Series A lead Dynamk Capital. The investment will be used to scale Curi’s existing business and accelerate the development of its innovative engineered tissue analysis platforms, including its Mantarray™ platform. “Curi Bio is thrilled to partner with the distinguished teams at UTC Investment, DS Asset Management, and Dynamk Capital to fuel our next stage of growth. To discover new therapies requires human-relevant disease models. Curi is working to close the gap between preclinical results and clinical outcomes, not only in small molecule discovery, but also in frontier areas like next-generation genetic medicines and cell therapies.” Michael Cho, JD, CEO of Curi Bio With costs to develop a single new medicine now exceeding $2Bn, the need for more human-relevant disease models to improve translational efficiency in the drug development process has never been greater. Curi’s core platform – the Curi Engine™ – integrates human stem cells, tissue specific biosystems, and A.I./M.L.-enabled data analysis to accelerate the discovery and development of new therapeutics. With this three-pronged strategy — human cells, systems and data — Curi is rapidly becoming a market leader in creating high-fidelity models of human diseases for drug discovery, especially for striated muscle, including cardiac and skeletal muscle, and neuromuscular models. “Curi Bio’s technology platforms create significant value for pharma and biotech companies by accelerating discovery timelines and increasing the chances of success for new therapies in development,” said Dr. Gustavo Mahler, Managing Partner, Dynamk Capital. “We look forward to strong growth in Curi Bio’s customer portfolio.” Curi Bio’s core technologies and products include NanoSurface™ Plates for structural maturation, Cytostretcher™ cell-stretching instruments, and the Mantarray platform for contractility analysis. The Mantarray platform enables researchers to generate and analyze 3D engineered human muscle tissues, providing clinically relevant functional readouts, and reducing reliance on poorly predictive animal models. Curi also offers a suite of customized research services utilizing the Curi Engine, including new assay and model development and phenotypic screening. Curi Bio counts all of the top-ten global pharmaceutical companies among its clients, customers, and partners. About Curi Bio Curi Bio’s preclinical discovery platform combines human stem cells, systems, and data to accelerate the discovery of new medicines. The Curi Engine is a seamless, bioengineered platform that integrates human iPSC-derived cell models, tissue-specific biosystems, and A.I./M.L.-enabled phenotypic screening data. Curi’s suite of human stem cell-based products and services enable scientists to build more mature and predictive human iPSC-derived tissues—with a focus on cardiac, musculoskeletal, and neuromuscular models—for the discovery, safety testing, and efficacy testing of new drugs in development. The company’s proprietary technologies are supported by over 100 publications and 19 patents. By offering drug developers an integrated preclinical platform comprising highly predictive human stem cell models to generate clinically-relevant data, Curi is closing the gap between preclinical data and human results, accelerating the discovery and development of safer, more effective medicines.

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CELL AND GENE THERAPY

Xylyx Bio partners with Inventia Life Science to develop tissue-specific, digitally-printed bioinks for drug discovery

Xylyx Bio, Inc. | September 02, 2021

Xylyx Bio, a New York-based leader in advanced biomaterials, and Inventia Life Science, a Sydney-based biotechnology company, today announced their strategic partnership following promising results developing more realistic, scalable, and reproducible 3D cell cultures for drug discovery and biomedical research. As researchers seek to develop more physiologically relevant in vitro models, 3D bioprinting provides promising tools to quickly and more precisely culture cells to develop more effective drugs. Inventia Life Science differentiates from other bioprinting solutions with their RASTRUM™ 3D cell culture platform, which empowers biologists with easy access to reproducible and tunable tissue models at scale. By incorporating liver-specific extracellular matrix (ECM) components developed by Xylyx Bio, the partners are developing robust 3D bioprinted liver tissue models. Inventia will complete product development and offer these solutions for research and drug discovery applications, with plans to expand offerings to other tissue types. Dr. John O'Neill, Xylyx Bio Co-Founder and Chief Scientific Officer, stated: "Xylyx is collaborating with groundbreaking companies like Inventia to lead the industry transition to physiologically relevant disease modeling and compound testing 'in matrico'." Dr. Julio Ribeiro, Co-Founder and Chief Executive Officer of Inventia Life Science, is very enthusiastic about this partnership. He stated, "At Inventia Life Science we strive to provide an automated 3D cell culture solution to accelerate drug discovery and biomedical research. This partnership with Xylyx is a significant step in the right direction in further expanding our matrix portfolio to enable the generation of complex tissue models in a reproducible and tissue specific manner." About Xylyx Bio Xylyx Bio is a pioneer in advanced biomaterials, serving customers across research, clinical and commercial applications. The Company's products provide the full suite of components from the natural cell microenvironment essential for the most accurate and actionable results for scientists working in pharmaceutical development, cell biology research, and regenerative medicine. About Inventia Life Science Inventia Life Science is a fast-growing biotech start-up based in Sydney, Australia, that is revolutionizing biomedical research, drug discovery and regenerative medicine. The award-winning RASTRUM™ 3D cell culture platform has already been adopted in leading medical research institutes and biotechnology and pharmaceutical companies worldwide, which are creating bioprinted tissue models that mimic real human biology.

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