MEDICAL, INDUSTRY OUTLOOK
PRNewswire | May 17, 2023
REGENXBIO Inc. announced that preclinical research in Mucopolysaccharidosis type IVA also known as Morquio syndrome, was selected for inclusion in the Foundation for the National Institutes of Health Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), clinical trial portfolio. The Consortium brings together partners such as NIH and FDA, as well as partners from private and non-profit sectors.
Sponsored by Nemours Children's Hospital, MPS IVA is one of eight programs selected as part of AMP® BGTC's first clinical portfolio to help accelerate the development of bespoke gene therapies, with the goal of streamlining the regulatory approval process. MPS IVA is a metabolic condition that primarily affects the skeleton, and is estimated to impact 1 in 200,000 to 300,000 individuals.
"I am proud of our scientists who led this important clinical research work for REGENXBIO. Our mission to deliver the curative potential of gene therapy reflects not only patients that can be impacted by our clinical pipeline, but also all patients who should have the opportunity to be positively impacted by gene therapy," said Kenneth T. Mills, President and CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to see this program advancing important science into the clinic."
Criteria for selection included the adequacy of the gene for insertion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment and an overall readiness for entering into a clinical trial. REGENXBIO will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases. Use of the NAV® Technology Platform will help address the goal of making gene therapy more accessible by creating a platform approach with standardized processes to deliver novel therapies for many different genetic disorders. These programs together further validate the versatility of NAV® vectors and will provide additional data that collectively drive the advancement of the AAV gene therapy field.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
CELL AND GENE THERAPY
PR Newswire | May 12, 2023
Mission Bio, the single-cell DNA and multi-omics company, announced today the Tapestri® Genome Editing Solution, an end-to-end product for genome editing analysis. The product will be previewed next week at the American Society of Gene and Cell Therapy Conference (ASGCT) 26th Annual Meeting. By enabling robust single-cell insights impacting both efficacy and safety, the solution will be a powerful analytical tool for developing the next generation of gene-edited therapies.
The first CRISPR-modified therapy is now under regulatory review, and many similar cell-based therapies are expected to follow for multiple intractable diseases. However, genome editing can result in complex, heterogeneous mixtures of edits that make it challenging to apply a level of process control over genome-edited cell products. The Tapestri® Genome Editing Solution addresses these challenges by measuring gene editing outcomes at single-cell resolution, capturing the co-occurrence of on- and off-target edits, as well as the zygosity of edits, which conventional bulk analyses cannot. Additionally, this analysis can be completed within days by processing thousands of cells at a time without any prior selection, while conventional analytical methods require months for clonal outgrowth.
An early iteration of the Tapestri® Genome Editing Solution is currently being tested by key genome editing researchers and leading cell therapy developers in academia and industry, who are providing vital feedback on the analysis. Mission Bio recently collaborated with the National Institute of Standards and Technology (NIST) in the Genome Editing Consortium, which provided qualified samples to collaborators to assess technologies that report variant size and frequency within a mixed cell population. Samantha Maragh, NIST Genome Editing Program Leader, will present results of the study at 12:00 p.m. PT on May 17 (Poster 533) at the ASGCT Annual Meeting.
"We look forward to pulling back the curtain on our end-to-end Genome Editing Solution at ASGCT," said Todd Druley, MD, PhD, Chief Medical Officer at Mission Bio. "The data acquired under the Genome Editing Consortium further demonstrates the Tapestri® Platform's potential as a standard analysis tool within the genome editing community. Given the heterogeneous results of gene editing strategies, there is a great need to address both industry and regulatory genome editing concerns with a consistent and highly precise technology for accurately measuring gene editing outcomes, and our new offering will be a complete solution to do just that."
About Mission Bio
Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.
The company's Tapestri® Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri® is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri® Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.
Globenewswire | May 15, 2023
TriLink BioTechnologies a Maravai LifeSciences company and global provider of life science reagents and services, has launched its newest cap analog, CleanCap® M6 . The latest CleanCap analog is expected to help developers and researchers maximize the impact of their mRNA therapeutics and vaccines while reducing overall manufacturing costs, bringing life-changing medicines to market faster.
With the introduction of the CleanCap M6 technology, legacy capping methods like enzymatic and ARCA may be a thing of the past. By combining the modification of 3’OMe on the m7-Guanosine and the additional methyl modification on the +1 Adenosine, the CleanCap M6 analog is expected to improve mRNA potency with the highest protein expression of any CleanCap analog yet. Combined with technology’s single-pot reaction capabilities that shrink manufacturing times and increase mRNA yields with a purity profile of >95%, CleanCap analogs are novel mRNA Cap structures, which may improve the benefits of an mRNA therapeutic or vaccine.
“TriLink’s portfolio of CleanCap mRNA capping technology is expected to be the new go-to capping method in the very near future - it is the future,” shared Kate Broderick Ph.D., Chief Innovation Officer at Maravai LifeSciences. “What excites me most about this novel analog is its ability to make mRNA an even more powerful therapeutic than we have seen previously.”
The company’s first CleanCap analog launched in 2017 and this capping technology has been incorporated in one of the first commercially approved COVID-19 vaccines. With the introduction of CleanCap M6, TriLink has developed its most robust analog to date. Studies have shown that the innovative product increases mRNA expression by 30% or more versus enzymatic capping methods and is estimated to reduce manufacturing costs by 20-40%.
“mRNA is clearly an emerging therapeutic modality where mRNA vaccines and therapeutics are expanding rapidly,” added Drew Burch, EVP of TriLink’s Nucleic Acid Products division. “CleanCap M6, and the entire CleanCap portfolio, joins a strong roster of synthesized nucleic acids, NTPs, and more that we expect to continue to grow.”
In addition to the CleanCap mRNA capping portfolio, TriLink has scale-up expertise and unique mRNA, oligonucleotide & plasmid production capabilities for companies focused on therapeutic, vaccine, diagnostic breakthroughs, leaning on its 25-plus years of experience to provide superior CDMO services.
Data and customer insights garnered from a market assessment conducted by a third-party consulting firm on behalf of TriLink BioTechnologies in November 2022.
Maravai is a leading life sciences company providing critical products to enable the development of drug therapies, diagnostics, and novel vaccines. Maravai’s companies are leaders in providing products and services in the fields of nucleic acid synthesis and biologics safety testing to many of the world’s leading biopharmaceutical, vaccine, diagnostics, and cell and gene therapy companies.