Category Archives: Transhuman News

Gene therapy is still in its infancy and has yet to achieve its full potential. First-generation gene therapies have been challenged by safety issues due to their inability to target cells and organs without also penetrating non-targeted cells and organs, especially the liver. Capsida's proprietary, targeted, non-invasive gene therapy technology allows more selective targeting of specific tissues and cells, overcoming many of the problems associated with first-generation gene therapies, specifically off-target cell and organ activity. In addition, it allows the gene therapy to be delivered non-invasively through intravenous (IV) administration. The company's already strong leadership team is poised to actualize the promise of gene therapy with the addition of Mr. Anastasiou and the promotions of Drs. Flytzanis and Goeden.

"I can't imagine a more exciting time to join this organization," said Mr. Anastasiou. "Capsida is enabling gene therapy to become what the industry, physicians, and patients have been dreaming it will be. Our patent-protected technology allows the targeting of cells and organs while limiting the negative impact on non-targeted areas, and can be applied across multiple therapeutic areas. Another important benefit of our technology is that we are able to deliver the gene therapy non-invasively through IV administration. I'm honored to lead this talented team to achieve Capsida's potential and to improve and even save patients' lives."

Mr. Anastasiou joins Capsida from Lundbeck, where he was an executive vice president and a member of the executive committee, reporting to the CEO. As the president of Lundbeck's U.S. and Canadian business operations, Mr. Anastasiou has built organizations from the ground up. He brings significant leadership experience managing diverse organizations and bringing them together to achieve common goals. He led as many as 1,200 employees and achieved net revenues of $1.5 billion. During his 12-year tenure at Lundbeck, Mr. Anastasiou held several progressive leadership positions, playing a pivotal role in developing and launching multiple products and building the company's cross-functional capabilities. Mr. Anastasiou serves on the Board of PhRMA and the global advisory board for the Healthcare Businesswomen's Association. Mr. Anastasiou begins his new role with Capsida on January 3, 2022.

"We're thrilled to welcome Peter as Capsida's new CEO," said Beth Seidenberg, M.D., founding managing director at Westlake Village BioPartners, one of the company's lead investors, and Capsida board member. "Peter has deep industry expertise, a broad network, and significant public company experience, which will be valuable as Capsida grows. In addition, his strong track record of success demonstrates he is a visionary leader who will be able to deliver on the promise of targeted non-invasive gene therapy to help underserved patients and achieve business success."

"During his tenure at Lundbeck, Peter has created significant shareholder value, creating and leading organizations and successful blockbuster product launches," said Clare Ozawa, Ph.D., managing director at Versant Ventures, one of Capsida's lead investors, and Capsida board member."Under Peter's leadership, we will continue to build Capsida as the industry's leading targeted, non-invasive gene therapy company with the ability to transform the lives of patients with life-threatening genetic disorders."

Prior to Lundbeck, Mr. Anastasiou held management roles at Neuronetics, Inc., Bristol-Meyers Squibb Company, and Eli Lilly and Company. He holds an MBA from the Kelley School of Business at Indiana University, and a B.A. in economics and management from Albion College.

Capsida co-founders Nicholas Flytzanis, Ph.D., promoted to CSO and Nick Goeden, Ph.D., promoted to CTO

In addition to Mr. Anastasiou's appointment, Capsida announced that Dr. Flytzanis has been promoted toCSO and Dr. Goeden has been promoted to CTO.

"The promotions of Drs. Flytzanis and Goeden are in recognition of the significant contributions they have made since co-foundingCapsida in 2019," said Mr. Anastasiou. "Their steadfast commitment to delivering on the promise of Capsida's differentiated, non-invasive gene therapy platform has been a key driver behind many of the company's early achievements."

"Drs. Flytzanis' and Goeden's strong scientific and technical expertise and know-how have already delivered results in the startup of Capsida based on Caltech'sbasic research on targeted non-invasive gene delivery to the brain," said Capsida co-founder Viviana Gradinaru, Ph.D. "Their promotions are timely as Capsida enters the phase of delivering from the lab and for the patients."

Prior to co-founding Capsida, Dr. Flytzanis served as scientific director of the CLOVER research center at the California Institute of Technology (Caltech), leading an interdisciplinary team to develop and disseminate emerging technologies focused on the cross-section of neurological research and gene therapy. His research spans the fields of tissue clearing and imaging, optogenetics and rodent behavior, and adeno-associated virus (AAV) engineering and gene therapy, with collaborations across multiple institutions. During his Ph.D., Dr. Flytzanis applied protein engineering and directed evolution across biological modalities, with a focus on developing AAVs as therapeutic tools for neurological disease.

Dr. Flytzanis holds a Ph.D. in biology from Caltech and a B.S. in biology from the Massachusetts Institute of Technology.

Prior to co-founding Capsida, Dr. Goeden led a team developing the novel adeno-associated virus (AAV) engineering technology underlying Capsida's biologically driven gene therapy platform. During his tenure as a postdoctoral fellow in Dr. Gradinaru's lab at Caltech, he developed high-throughput methods for screening combinatorial libraries to explore the AAV fitness landscape and engineered novel AAVs with high efficiency and specificity for the rodent and primate brain. During his Ph.D., Dr. Goeden developed a novel organ bioreactor to study real-time metabolomics in diseased states, exploring the relationship between gene expression and the pathophysiology of neurodevelopmental disorders.

Dr. Goeden holds a Ph.D. in neuroscience from The University of Southern California and a B.S. in biology from Caltech.

About Capsida Biotherapeutics

Capsida Biotherapeutics Inc. is an industry-leading gene therapy platform company creating a new class of targeted, non-invasive gene therapies for patients with debilitating and life-threatening genetic disorders. Capsida's technology allows for the targeted penetration of cells and organs, while limiting collateral impact on non-targeted cells and organs, especially the liver. This technology allows for the delivery of the gene therapy in a non-invasive way through intravenous administration. Capsida's technology is protected by a growing intellectual property portfolio which includes more than 30 patent applications and one issued U.S. patent 11,149,256. The company is exploring using the technology across a broad range of life-threatening genetic disorders. Its initial pipeline consists of multiple neurologic disease programs. The company has strategic collaborations with AbbVie and CRISPR, which provide independent validation of Capsida's technology and capabilities. Capsida is a multi-functional and fully integrated biotechnology company with proprietary adeno-associated virus (AAV) engineering, multi-modality cargo development and optimization, translational biology, process development and state-of-the-art manufacturing, and broad clinical development experience. Capsida's biologically driven, high-throughput AAV engineering and cargo optimization platform originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at the California Institute of Technology. Visit us at http://www.capsida.com to learn more.

SOURCE Capsida Biotherapeutics

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Capsida Biotherapeutics Poised to Capitalize on Industry-leading Gene Therapy Technology With New CEO, CSO, and CTO - PRNewswire

EMERYVILLE, Calif.--(BUSINESS WIRE)--Metagenomi, a genetic medicines company with a versatile portfolio of next-generation gene editing tools, today announced that the company will share data related to their novel, compact, and hypo-immune gene editing systems at the 63rd Annual Meeting and Exposition of the American Society of Hematology (ASH), which is taking place in Atlanta, GA and virtually, December 1114.

The development of CAR T therapies and other genetically engineered cell therapies in recent years has resulted in significant benefits for patients, yet there remains a large unmet need for gene editing systems that can be used to develop novel immunotherapy approaches to treat blood cancers, said Brian C. Thomas, PhD, CEO and Co-Founder of Metagenomi. At ASH, we are presenting data on our novel nucleases that display highly efficient and specific gene editing both in vivo and ex vivo and hold significant potential to drive the development of new and efficacious therapies for patients.

In a poster titled A Novel Type V CRISPR System with Potential for Genome Editing in the Liver, it is shown that Metagenomis novel Type V CRISPR-associated nuclease was highly active in the liver of mice when systemically administered via lipid nanoparticles (LNP). The nuclease was derived from a unique natural environment and is phylogenetically distinct from previously identified Type V systems. Moreover, no antibodies to the nuclease were detected in serum from 50 healthy human donors, while between one third and half of the same serum samples contained antibodies that bind to spCas9, which is derived from a Streptococcus bacteria that commonly infects humans. In summary, this novel Type V CRISPR-associated nuclease is a promising new gene editing system for in vivo editing of the liver.

In a separate poster titled Novel CRISPR-Associated Gene Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genomic Engineering for Cell Therapy Development, three novel gene editing systems were used to make reproducible and efficient edits to human immune cells, demonstrating utility for the next generation of cell therapy development for blood cancers. Metagenomis novel gene editing systems were used to disrupt the T cell receptor alpha-chain constant region and the T cell receptor beta-chain constant region in approximately 90 percent of cells. Beta-2 microglobulin was edited in 95 percent of T cells. A chimeric antigen receptor (CAR) construct was also shown to be integrated in up to 60 percent of T cells. Novel gene editing systems were deployed in NK cells to disrupt CD38 a cell surface immune modulator that can be targeted in the development of cancer immunotherapy and to integrate a CAR construct that led to robust CAR-directed cellular cytotoxicity. B cell editing occurred in approximately 80% of target cells with successful transgene integration. Whats more, as these gene editing systems are taken from environmental samples as opposed to human pathogens, pre-existing immunity is expected to be rare. In summary, these novel systems were shown to result in highly efficient and specific gene edits in human immune cells and display the potential for use in cell therapy development.

Details of the presentations are below:

Presentation Title: A Novel Type V CRISPR System with Potential for Genome Editing in the LiverSession Title: 801. Gene Therapies: Poster IPresenting Author: Morayma Temoche-Diaz, PhDPublication Number: 1862 Session Time: Saturday, December 11, 5:30 p.m. ET

Presentation Title: Novel CRISPR-Associated Gene-Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering for Cell Therapy DevelopmentSession Title: 801. Gene Therapies: Poster IIIPresenting Author: Gregory Cost, PhD, Vice President of Biology, MetagenomiPublication Number: 3984 Session Time: Monday, December 13, 6:00 8:00 p.m. ET

About Metagenomi

Metagenomi is a gene editing company committed to developing potentially curative therapeutics by leveraging a proprietary toolbox of next-generation gene editing systems to accurately edit DNA where current technologies cannot. Our metagenomics-powered discovery platform and analytical expertise reveal novel cellular machinery sourced from otherwise unknown organisms. We adapt and forge these naturally evolved systems into powerful gene editing systems that are ultra-small, extremely efficient, highly specific and have a decreased risk of immune response. These systems fuel our pipeline of novel medicines and can be leveraged by partners. Our goal is to revolutionize gene editing for the benefit of patients around the world. For more information, please visit https://metagenomi.co/.

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Metagenomi to Present Preclinical In Vivo and Ex Vivo Gene-Editing Data at the 63rd American Society of Hematology (ASH) Annual Meeting - Business...

Erika Moore studies lupus using a unique biomaterial that she developed.

Erika Moore, PhD

Long before she took the helm of her engineering lab as an assistant professor of materials science and engineering at the University of Florida, Erika Moore was fascinated by the careful dance between immune cells and biomaterials engineered to interact with living systems. Now, her curiosity has led her to design biomaterials to help diverse lupus patients receive personalized care.

How did you first realize that biomaterials could be useful for studying the immune system?

I had to start wearing contacts when I was 11 years old, and as I put something into my eye every day that was foreign to my body, it really piqued my interest in understanding biomaterials. While doing my undergraduate degree at Johns Hopkins University, I started to realize that immune cells are probably going to be an important part of how our bodies respond to biomaterials.

My area of expertise is at the meeting point of immunology, biomaterials science, and translational medicine. I was classically trained as a biomedical engineer, so the combination of engineering and medicine is just ingrained into me. Then I was drawn to creating new materials and using those materials to study immunology, but I'm not a classically trained immunologist. Biomedical engineers have been designing materials and putting them into rodents to validate them, but we haven't always considered how the immune system responds to these materials. During my PhD studies at Duke University, I focused on macrophages and understanding their contribution to biomaterials.

How did you connect this to studying autoimmunity and lupus specifically?

My personal connection to systemic lupus erythematosus (SLE), the autoimmune disease that I study, is that I identify as a woman of color, as a Black woman. SLE is a hugely sex-differential disease. About 90% of the people with SLE are women. If you break down that 90%, about 70% of that 90% are women of color. I actually have many friends with lupus; there are women in my life who have died from lupus, and I never realized how much of a health inequity and health disparity it was. When I realized that there was this autoimmune disease that disproportionately affects communities that I identify with, I wanted to use my privilege and education to try to improve outcomes for that disease.

Erika Moore developed a jello-like tissue system to study how immune cells stimulate tissues and blood vessel growth.

Erika Moore, PhD

That brings us back to the science. If we want to study something in the clinic, we have to enroll patients. But that takes a lot of time, effort, and energy. So, during graduate school, I wanted to create a tissue model outside the body that would recreate or mimic some of the interactions that occur in the body. The 3-D model system that I created then is a little bit squishylike jellomimicking some soft tissues in the body. You can take images of it, destroy it, and create it again. I used this system in graduate school to understand how macrophages help us build new tissues and blood vessels.

If we introduce other variables, we can study tissue responses in specific human contexts. For example, women with lupus usually have super inflammatory immune cells, including a lot of macrophage activation. If we take immune cells from healthy controls or patients with lupus and study them in our biomaterial models, what differences do we observe in those cells? And what does that tell us about what might be happening clinically?

The promise of our model system is that we can have personalized medicine approaches in our mini-jello tissues that we create in the lab. Maybe in the future, we can take some patient cells, put them in our system, and then introduce a drug to see how they respond. Does it get better? Does it get worse? That's better than just putting volunteers on a drug without any prior knowledge or background.

How do you consider the variation in lupus between populations?

I want to understand the contribution of ancestry to disease pathology and disease progression. We use markers of genetic ancestry to understand how different patients respond. We also use socio-cultural, educational, and financial markers because we understand that ancestry doesn't always capture lived experience. We know that environment and geneticsnature and nurturecombine to inform biology and cell function. We can declare those variables and use them to determine which groups we encapsulate into our jello biomaterial tissue models, and then understand how their cells respond differently. For example, does the number of discrimination events you have seen inform how your cells respond?

What are the challenges for this kind of interdisciplinary work?

The hardest part so far has been knowledge. I am personally driven to study this autoimmune disease because of its disproportionate impact, so I really want to learn, and I'm still learning. Because my work is interdisciplinary, I have to master multiple fields. I'm really grateful for my collaborators who have been helpful in educating me to ask the right questions, because not all questions are worth answering immediately. You have to prioritize. I know a lot of people with lupus, and I ask, what's helpful? What's not working? It's a shared community effort. I am really sharing the voices of many people, not just myself.

This interview has been edited and condensed for clarity.

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An Engineers Perspective on Autoimmunity - The Scientist