Genome Editing

Spark Therapeutics Plans $575M Philadelphia Gene Therapy Center

The 500,000-square-foot center will serve as a Roche global center of excellence for gene therapy manufacturing, facilitating collaborations with Drexel and other stakeholders in Philadelphia’s growing life sciences, which has focused on recent years on cell and gene therapy.

Genome-Wide CRISPR Editing for Improved Protein Expression in Yeast

In this GEN webinar, sponsored by Inscripta, our distinguished presenter, Dr. Eric Abbate, will demonstrate a novel multiplexed genome-wide editing approach to optimize the cellobiohydrolase I enzyme (CBH1) production in S. cerevisiae. Additionally, we will learn how multiplexed and trackable editing libraries enable the discovery of new targets. These libraries comprise a wide range of edit types and targets, including genome-wide and targeted knockouts, short deletions, alternate codon, transcription factor binding sites, and terminator libraries.

Gene Study May Lead to New Late-Onset Retinal Degeneration Therapies

People with L-ORD develop abnormal blood vessel growth and deposits of apolipoprotein E, which is involved in fat metabolism within the retina. Symptoms, including difficulty seeing in the dark and loss of central vision, usually appear around age 50–60. As L-ORD progresses, cells in the retinal pigment epithelium shrink and die. Loss of RPE leads to loss of photoreceptors and loss of vision.

Helen Sabzevari, PhD, of Precigen Connects Cancer and the Immune System on “Close to...

Precigen recently shared positive news on the clinical progress of five pipeline candidates. Three were developed using Precigen’s Ultra-CAR-T platform: PRGN-3005 being developed for advanced, recurrent platinum resistant ovarian cancer; PRGN-3006 for relapsed or refractory acute myeloid leukemia; and PRGN-3007, for which the FDA has cleared IND application to launch a Phase I/Ib open-label trial in advanced ROR1+ hematological and solid tumors.
Human microbiome, conceptual illustration

CRISPR Pioneers Doudna and Banfield Introduce Microbiome Community Editing

Combining two, new, CRISPR-based, approaches has led to the development of a strategy to edit the genomes of specific organisms in microbial communities. A screening technique called ET-seq (environmental transformation sequencing) together with a targeted delivery system called DNA-editing All-in-one RNA-guided CRISPR Cas Transposase (DART), allows for the addition or modification of genes within a community of many different species simultaneously, or "community editing."
Genetic engineering concept.

Powering Up the U.K.’s Advanced Therapy Workforce

The Cell and Gene Therapy MSc was designed to provide students with an in-depth insight into the cutting-edge and rapidly developing field of biological therapies. The program is delivered by scientists and clinicians researching, developing, and testing new treatments for genetically inherited and acquired diseases using gene delivery technology, stem cell manipulation and DNA repair techniques.

George Church, PhD, Recounts Decades of Research, 38 Companies on “Close to the Edge”

Church is a pioneer of genomics with numerous accomplishments in the areas of genome mapping, sequencing, editing, and writing. Since 1986, Church has been on the faculty at Harvard, where he is a professor of genetics at Harvard Medical School and a founding member of the Wyss Institute, for which he oversees the directed evolution of molecules, polymers, and whole genomes to create new tools with applications in regenerative medicine and bio-production of chemicals.
Adeno-associated viruses, 3D illustration

Extending Biosafety Testing to Gene Therapy Products

In biosafety testing, the expression “dot your i’s and cross your t’s” comes to mind. If only biosafety testing were so simple. To overcome the biosafety challenges posed by viral vectors, biomanufacturers are applying Bac-to-AAV, 2D-HPLC, QbD, LC-MS/MS, and other technologies.

Astellas, Dyno Launch Up-to-$1.6B+ Gene Therapy Collaboration

Astellas agreed to pay Dyno $18 million upfront, additional payments of an undisclosed amount during the research phase of the collaboration, as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential total value of future milestone and royalty payments to Dyno exceeds $1.6 billion, based on $235 million per product. Astellas is the fourth biopharma giant to partner with Dyno on collaborations based on CapsidMap.
Medical pharmacology

Cell and Gene Therapy Sector Needs CMC Clarity

For therapeutic proteins and monoclonal antibody- (mAb) based medicines, CMC requirements are well established. And more importantly developers know what reviewers look for and, to a greater or lesser extent, manufacturing methods are standardized. Cell and gene therapy developers also have CMC guidelines. For example, in 2020 the FDA published information for gene therapy developers.