Distorted, stretched DNA may increase risk of off-target changes with CRISPR-Cas9

Phys.org | February 26, 2019

Distortions to DNA, which occur routinely during gene expression and other cellular processes, could lead to off-target changes to the genome when using CRISPR-Cas9, a new study suggests. The Medical Research Council scientists behind the research say that their findings may help to pave the way to improve on the accuracy of gene editing for clinical applications. During the expression of genes, DNA is stretched and distorted out of its usual shape. While this is needed for proper function of the cell's own machinery, it may pose a challenge for CRISPR-Cas9 gene editing by increasing the risk of off-target edits, potentially introducing harmful changes. The findings, from scientists at the MRC London Institute of Medical Sciences and AstraZeneca, are published today in the journal Nature Structural and Molecular Biology. CRISPR-Cas9, a gene editing tool that allows researchers to find and edit strands of DNA, has gained worldwide recognition for its multitude of purposes as scientists use the technology across a range of sectors, including medicine, drug discovery and agriculture.

Spotlight

SpaceLife Origin, based in the Netherlands, wants to send a pregnant woman, accompanied by a ‘trained, world-class medical team,’ in a capsule to the space above Earth. The mission would last 24 to 36 hours. Once the woman delivered the child, the capsule would return to the ground.”

Spotlight

SpaceLife Origin, based in the Netherlands, wants to send a pregnant woman, accompanied by a ‘trained, world-class medical team,’ in a capsule to the space above Earth. The mission would last 24 to 36 hours. Once the woman delivered the child, the capsule would return to the ground.”

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Biomica Announces the Advancement to Large-scale Production of BMC128, Its Live Bacterial Product Candidate Consortium

Biomica | October 13, 2020

Biomica, an emerging biopharmaceutical company developing innovative microbiome-based therapeutics, and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), announced today the advancement to large-scale production of BMC128, its Live Bacterial Product (LBP) candidate consortium. The microbes, which will be produced in large-scale are expected to support Biomica's first-in-man proof-of-concept clinical trials for its immuno-oncology program, anticipated to begin in 2021. BMC128 is advancing to the GMP production stage following the successful completion of the initial R&D stage of drug product development and manufacturing, conducted by Biose Industrie (Aurillac, France). Biomica's immuno-oncology program is focused on its leading 4-strain candidate consortium BMC128. Biomica recently announced positive results in the program, demonstrating the efficacy of BMC128 in potentiating the response to immune-checkpoint inhibitors (ICI) in preclinical studies. In these studies, BMC128 was administered to mice bearing cancer tumors prior to and during ICI therapy, and the results showed that treatment with BMC128 significantly improved anti-tumor activity. Biose Industrie is a drug-GMP certified manufacturer of bacteria-based APIs and clinical and commercial products. As previously announced earlier this year, Biomica engaged Biose for the scale-up development and GMP production of a clinical batch of its drug candidates.

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MEDICAL

Targeting Chronic Infections in Cystic Fibrosis, Cystic Fibrosis Foundation Invested in Felix Biotechnology

Felix Biotechnology | March 07, 2022

Felix Biotechnology, a biopharmaceutical business focusing on developing first-in-class, long-lasting antibacterial medicines to treat chronic drug-resistant infections, announced that it has received a Therapeutics Development Award from the Cystic Fibrosis Foundation. The funding will aid Felix's efforts to develop a novel medication to treat chronic Pseudomonas aeruginosa lung infections. Chronic P. aeruginosa infections affect half of the persons with cystic fibrosis, many of whom are multidrug-resistant. Since infections are the leading cause of death in cystic fibrosis patients, innovative treatments are urgently needed. Felix offers a novel strategy by turning bacteria's natural predators, microscopic viruses known as bacteriophage (or phage), into long-lasting, broadly effective medicines. Felix's phage therapies are specifically designed to examine and drive positive evolutionary effects of phage treatment, based on foundational research by Yale University's Drs. Paul Turner, Ben Chan, and Jon Koff. This results in a more long-lasting treatment, which is essential given the requirement to regularly deliver phage to treat chronic infections. This medication is now being tested in a clinical trial at Yale University. More information about the experiment can be found here, and interested patients are encouraged to enroll if they are eligible. We are incredibly honored and excited to receive this support from the CF Foundation, This award will speed Felix in its mission to provide new therapeutic options to patients dealing with life-threatening infections and to develop phage as a broadly applicable antibacterial therapy." Dr. Paul Turner, Ph.D., co-founder of Felix.

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INDUSTRIAL IMPACT

Vaxess Technologies Announces Grand Opening of GMP Manufacturing Facility

Vaxess Technologies | October 27, 2021

Vaxess Technologies, a biotechnology startup developing the MIMIXTM sustained-release intradermal microneedle patch, celebrated the grand opening of their 3,600 square feet pilot GMP (Good Manufacturing Practices) manufacturing facility on Monday. The facility, which is located in Woburn, MA at one of the Cummings Park properties, will feature a state-of-the-art cleanroom where manufacturing of vaccine patches for clinical trials will take place. Using the clinical-grade patches produced in the facility, Vaxess plans to enter the clinic in 2022. In addition to the manufacturing suite, Vaxess has announced plans to expand their Woburn footprint by another 6,300 square feet to provide additional GMP space as well as lab space for a host of other development activities to further the MIMI“ platform. After years of development, the company is thrilled to establish GMP manufacturing capabilities in Massachusetts for both our lead MIMIX-Flu vaccine program as well as a host of other pipeline products to follow,” said Vaxess CEO Michael Schrader. Vaxess has experienced tremendous growth over the past year beyond the opening of the manufacturing facility including technological advancement of the platform and the expansion of the organization from 15 full time employees to 35 with additional future growth planned. All the progress that Vaxess has made this past year would not have been possible without the help of the Massachusetts Life Sciences Center (MLSC), which has supported Vaxess’ efforts since its inception. MLSC President and CEO, Kenn Turner, attended the Grand Opening and shared a few words. “I want to congratulate the Vaxess team on their latest expansion to Woburn Since the Life Science Center’s initial investment in Vaxess, the company has exemplified the type of growth trajectory we aim to accelerate here in the Commonwealth. We will continue to lean in, and support companies seeking to create jobs and expand their footprint regionally, particularly in the manufacturing space.” Massachusetts Life Sciences Center President and CEO Kenn Turner In addition to Turner, numerous other individuals from both the Massachusetts biotechnology and political communities attended the event, including Joe Boncore, President of MassBio, Bill Cummings, Founder of Cummings Properties, Eric Anderson, President of Cummings Properties, Scott Galvin, Mayor of Woburn, Representative Richard Haggerty and Senator Cindy Friedman. “I want to congratulate Vaxess Technologies on their continued job growth and expansion, which offers further proof that opportunities for innovation in life sciences are truly statewide in Massachusetts,” said Massachusetts Housing and Economic Development Secretary Mike Kennealy, who serves as Co-Chair of the MLSC Board of Directors. “The Baker-Polito Administration remains committed to supporting life sciences companies that recognize Massachusetts as the ideal place for companies to grow and thrive.” About the MIMIX™ Technology The MIMIX patch is designed to be the easiest and most effective way to deliver vaccines and therapeutics. The patch releases treatments to the body at their most beneficial rate and duration. For vaccines, the controlled release simulates the pace of a natural infection, helping the body produce a slow, strong, and enduring ramp-up of immune response, ultimately boosting a vaccine’s effectiveness. Engineered for stability, Vaxess’ patch does not require refrigeration and can be shipped to and applied in low resource settings. It is virtually painless and resembles an adhesive bandage. After the minutes-long prescribed wear time, the MIMIX patch is removed and discarded while leaving behind microscopic intradermal depots to present the therapeutic payloads to the immune system over ensuing days or weeks. About Vaxess Technologies Vaxess Technologies is developing the MIMIX™ sustained release patch technology, originally conceived at MIT and Tufts University. MIMIX™ uses the unique qualities of silk proteins and breakthrough immune activating biology to enable best-in-class vaccines and therapeutics. Vaxess has raised more than $60M in grant and venture capital funding from groups such as The Engine, BARDA, DARPA, NIH, NSF and the Gates Foundation. A MIMIX Phase I proof-of-concept trial using seasonal flu and COVID-19 antigens is slated to begin in the first half of 2022.

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