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.

Purification of Recombinant AAV Vectors for Preclinical Applications

Cesium chloride- and iodixanol-based density gradients represent the core step in most protocols for serotype-independent adeno-associated virus purification.
Embryos after gene editing and cell division. A recent Nature study found that paternal gene mutations in human embryos are best replaced with maternal genes. [Source: OHSU]

CRISPR Corrects Disease Mutation in Human Embryos

For the first time in the U.S., scientists use CRISPR to replace a mutated gene in a human embryo.
Rainbow DNA

A Roadmap and Wish List for Synthetic Genomics

Within the huge synthetic biology world is an enormous variety of ideas and approaches, like custom-made proteins, CAR-T medicines, genetically engineered crops, and more. One sector still in its infancy is synthetic genomics, where instead of one protein or gene, entire genomes are designed, synthesized, and implemented. To make synthetic genomics bloom, it needs new innovations and new support.
Abstract medical background with DNA helix, genetic code and mos

Gene Drive Modified to Run Allelic Errands

A new gene drive system introduces gears well suited to the transmission of relatively slight genetic variants, that is, allelic variants involving only one...
The Cell and Gene Therapy (CGT) Catapult will partner with Freeline Therapeutics to build the gene therapy developer's proprietary manufacturing platform at the CGT Catapult’s large-scale GMP manufacturing center in Stevenage

U.K.’s CGT Catapult Partners with Freeline to Build Gene Therapy Manufacturing Platform

Freeline said it plans to deploy its manufacturing platform for production of clinical supply in one of the CGT Catapult’s newly built manufacturing center modules

Synthesizing Error-Free DNA from RNA

New enzyme will increase precision in genetic research and could dramatically improve medicine based on a person's genetic makeup

Voyager Exercises Options for Regenx Gene Therapy Vectors

Regenx grants Voyager a nonexclusive worldwide commercial license to three NAV vector sequences developed through Regenxbio’s NAV Technology Platform, each for the treatment of a neurological disease

Pfizer, Sangamo Report Positive Phase I/II Data for Severe Hemophilia A Gene Therapy

The five patients all received the 3e13 vg/kg dose of giroctocogene fitelparvovec and underwent follow-up assessments over periods that varied per patient, stretching up to 61 weeks for the longest-treated participant.

Bioluminescent Torch Passed from Glowing to Dim Fungi

A set of bioluminescence genes has been transferred from one eukaryote, a glowing fungus called Neonothopanus nambi, to another eukaryote, a non-glowing fungus called...