Engineering Accuracy in CRISPR

Technologynetworks | April 16, 2019

Biomedical engineers at Duke University have developed a method for improving the accuracy of the CRISPR genome editing technology by an average of 50-fold. They believe it can be easily translated to any of the editing technology's continually expanding formats. The approach adds a short tail to the guide RNA which is used to identify a sequence of DNA for editing. This added tail folds back and binds onto itself, creating a "lock" that can only be undone by the targeted DNA sequence. The study appears online on April 15 in the journal Nature Biotechnology.  "CRISPR is generally incredibly accurate, but there are examples that have shown off-target activity, so there's been broad interest across the field in increasing specificity," said Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke. "But the solutions proposed thus far cannot be easily translated between different CRISPR systems." CRISPR/Cas9 is a defense system that bacteria use to target and cleave the DNA of invading viruses. While the first version of CRISPR technology engineered to work in human cells originated from a bacteria called Streptococcus pyogenes, many more bacteria species carry other versions. Scientists in the field have spent years looking for new CRISPR systems with desirable properties and are constantly adding to the CRISPR arsenal. For example, some systems are smaller and better able to fit inside of a viral vector to deliver to human cells for gene therapy. But no matter their individual abilities, all have produced unwanted genetic edits at times.

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Twenty-one high school students are assuming the roles of scientists as participants in the Environmental Biotechnology STEM Academy at Pontotoc Technology Center in Ada, Oklahoma. The first-of-its-kind science, math and biotechnology course was established in 2014 through a grant from Oklahoma NSF EPSCoR. Designed as a two-year, half-day program for eleventh and twelfth-grade students, the STEM Academy has been highly successful, with 95% of juniors reenrolling for their senior year in 2015.

Spotlight

Twenty-one high school students are assuming the roles of scientists as participants in the Environmental Biotechnology STEM Academy at Pontotoc Technology Center in Ada, Oklahoma. The first-of-its-kind science, math and biotechnology course was established in 2014 through a grant from Oklahoma NSF EPSCoR. Designed as a two-year, half-day program for eleventh and twelfth-grade students, the STEM Academy has been highly successful, with 95% of juniors reenrolling for their senior year in 2015.

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CELL AND GENE THERAPY

Tune Therapeutics Launches with Pioneering Epigenomic Control Platform to Master Gene Networks, Treat Broad Range of Diseases

Tune Therapeutics | December 03, 2021

Tune Therapeutics, a biotechnology company pioneering the creation of epi-therapeutic medicines, launched today with its powerful and precise genetic tuning platform, TEMPO. This cutting-edge technology dials gene expression up or down to desired levels – with the potential to reverse pathways of cancer, genetic disease, and aging by changing cell fate and function at will. "Genetic medicine is at a tipping point. We now understand that the driving force of human health and disease is not our genes, but the epigenomic elements that shape and control them. Until now, scientists and bioengineers lacked the combined understanding, clinical expertise, and technology needed to make epigenomic therapies a practical reality. Now, we have all three." Matt Kane, CEO of Tune Therapeutics TEMPO Platform Tune's proprietary TEMPO platform can rapidly target and adjust the epigenomic machinery of the cell, which shapes DNA and controls gene expression. By varying specific control modules in an iterative process, TEMPO can fine-tune expression toward healthy levels – even in diseases involving multiplex or polygenic interactions. Unlike genome editing, the tuning process does not generate double- or single-strand breaks in DNA and makes no permanent changes to the DNA sequence. This de-risks the precise targeting of entire gene networks, allowing Tune to simultaneously turn silenced genes on and dial over-expressed genes down, in a practical, therapeutic context. Tune has already shown that TEMPO can locate epigenomic elements involved in several intractable genetic conditions – revealing targets and networks that would be invisible or inaccessible to gene editing approaches. Moreover, Tune can optimize TEMPO to command expression of individual genes or networks with remarkable specificity and precision. This opens the door to an entirely new class of epi-therapeutics. "The exciting challenge in front of us is taking these transformative advances in technology and extending their potential for our greater society," said Charlie Gersbach, PhD, Acting Chief Scientific Officer, Tune Therapeutics. "From proof of concept in rare, single-gene disorders to common conditions that aren't linked to a single gene mutation – but are treatable through epigenomic control and constitute the vast majority of human diseases." Veteran Genomic Medicine Leadership Team Tune is launching with a veteran leadership team, endowed with deep expertise in gene and cell therapy, genome editing, and epigenetics. Matt Kane, Chief Executive Officer Akira Matsuno, Co-Founder, President and Chief Financial Officer Charlie Gersbach, Ph.D., Co-Founder, Acting Chief Scientific Officer Fyodor Urnov, Ph.D., Co-Founder, Scientific Advisory Board Heidi Zhang, Ph.D., Executive Vice President, Head of Technical Operations Blythe Sather, Ph.D., Vice President, Head of Research In addition, Tune's Board of Directors includes Mr. Kane, Dr. Gersbach, Ali Behbahani, M.D., (New Enterprise Associates), and co-founder Dan McHugh Drawing upon deep, local talent pools in Durham and Seattle, Tune has assembled two highly seasoned discovery and development teams, secured foundational intellectual property from Duke University, and raised $40 million from top-tier investors – including co-leads New Enterprise Associates and Emerson Collective, with Hatteras Venture Partners, Mission BioCapital, and others joining the round. This financing will enable Tune to rapidly advance its preclinical research, attract top-tier talent, and further develop its therapeutic platform. "Tune is effectively pioneering a brand-new therapeutic modality," said Dr. Behbahani. "With the unbound potential of this approach, and their collective successes in the field, Tune is primed to become a transformative presence in modern biomedicine." About Tune Therapeutics Tune Therapeutics, Inc. is orchestrating the power of the epigenome to treat complex, pervasive diseases that have long eluded treatment. With its versatile and powerful TEMPO epigenomic control platform, Tune's experienced team is pioneering a new therapeutic modality that can fine-tune any gene network. In breaking free from the limitations of traditional gene and cell therapies, Tune is developing solutions for even the most challenging and intractable diseases – and building the capacity to transform human health and medicine.

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Stemcyte Submitted its Application for A Biologics License (BLA) For Its Product "HPC-Cord Blood" To The US FDA

Stemcyte | March 23, 2022

On January 7th, 2022, the FDA received the BLA (Biologics License Application) for the "HPC-Cord Blood" product. On March 8th, 2022, StemCyte was formally informed that the submission was acceptable to join the biologics license quality review process. The "HPC-Cord Blood" is an umbilical cord blood hematopoietic stem cell product intended for unrelated donor hematopoietic progenitor cell transplantation procedures in patients with hematopoietic system disorders an appropriate preparative regimen for hematopoietic and immunologic reconstitution. There have been over 40,000 successful umbilical cord blood transplants globally to treat disorders of the hematological and immunological systems and genetic metabolic diseases since the first successful use of umbilical cord blood to treat Fanconi Anemia patients in 1988. StemCyte has donated more than 2,200 cord blood units for transplantation to 1 in every 20 patients with umbilical cord blood transplants globally during the last 20 years. In addition, at least 350 transplant centers around the world, including well-known medical centers such as UCLA Medical Center, Duke University Hospital, Taiwan Chang Gung Memorial Hospital, and National Taiwan University Hospital, consistently meet the quality standards of international accrediting bodies and are recognized and trusted as safe and effective by StemCyte's products.

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DIAGNOSTICS

Avantor® to Collaborate with GeminiBio to Bring Custom Hydrated Solutions and Cell Culture Media to Biopharma Customers

Avantor | June 24, 2022

Avantor, Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries has entered into an agreement with GeminiBio to deliver custom hydrated solutions and cell culture media to the biopharma industry. Custom hydrated solutions and cell culture media are critical components of the bioproduction workflow, especially for emerging modalities such as gene therapy. This collaboration will complement Avantor's proven ability to serve manufacturing platforms with GeminiBio's small-to-medium scale offering, enabling customers to utilize custom cGMP products through the full development lifecycle – including early-stage research, scale-up and commercialization. This new capability along with Avantor's recent acquisitions and site investments, support a growing portfolio of proprietary offerings, demonstrating Avantor's commitment to supporting customer workflows in both upstream and downstream processing for the biopharma industry. With the imperative to meet patient needs quickly, our customers require custom solutions to accelerate their bioproduction goals, Customized media is critical in optimizing cell line productivity, and hydrated solutions offer efficiency to biomanufacturers as they optimize their downstream processes. Our collaboration with GeminiBio will drive increases in product yields, helping bring efficiency and speed to our customers as they scale. Together, we can drive science forward to bring therapies to the patients who need them most." Dr. Ger Brophy, Executive Vice President, Biopharma Production for Avantor. Enabling customers to optimize their upstream and downstream liquid manufacturing workflows by offering custom solutions, built around a strong quality foundation, has been an essential part of GeminiBio's success. Collaborating with Avantor, who shares our focus on quality, will allow us to expand our ability to address customer needs for both small and large volume process liquids." Brian Parker, CEO of GeminiBio. About Avantor Avantor®, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries. We set science in motion to create a better world.

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