Electroporation is the application of an electrical pulse across a cell membrane to
temporarily disrupt the cell membrane in order to enable the uptake of exogenous
molecules (such as genes or mRNAs) found in the medium to either the cytoplasm or into
the nucleus.
Electroporation provides a highly effective alternative to viral vector-based transfection. It is
very easy to use with little tox effects and can be scaled up with little difficulty.
Gene Therapy 2.0: Gene Editing Primer; New Tools Emerging To
Gene Therapy 2.0: Gene Editing Primer; New Tools Emerging To yesterday at 4:07am Post by Ma$†¡N® on yesterday at 4:07am
Gene Editing Primer; New Tools Emerging To Do Great Things (PIPER REPOT 140pages May19)
In this report we take a closer look at gene editing platforms as they rapidly advance towards deployment in an ever-expanding armamentarium of GenomeRx therapeutics. This builds upon our prior overviews on gene therapy and genetic engineering of lymphocytes (CAR-T/TCR) as we continue to see the genomic revolution leading to transformational platforms and delivering breakthroughs for patients. The landscape is rapidly expanding with Zn fingers, TALENs, MegaTALs, ARCUS and CRISPR/Cas-9. In this report we provide an overview of these technologies which will also help guide discussions at our upcoming GenomeRx Symposium (May 20th in NYC) where we will have 2 panels devoted to this topic. Most of the companies in this report are also returning on May 21st and are available for 1x1 meetings with investors. We are hosting a call at 1PM ET today to review our analysis; please contact us or your PJC rep for details. • Gene editing in a nutshell: Simply put, gene editing platforms are genetic scissors which can cut DNA and lead to knockout or correction or insertion of genetic sequences. Of course it's not nearly so simple as these therapeutics have to be designed for high efficacy as well as precision to avoid excessively chopping up the cell's DNA. Additionally these 'scissors' tend to be large proteins which need to gain access into the cells, requiring delivery strategies which are also complex. Lastly, the way in which the scissors lead to genetic alterations itself is complicated. • But with complexity comes great power: The ability to alter host DNA in a precise way has the potential to unlock a substantial amount of unmet medical need heretofore untapped by existing technologies, including "basic" gene therapy approaches. As the genetic causes of an increasing number of diseases are unraveled, a growing number of opportunities should be addressed by these unique tools. • Some things they can do: Progress is already being made deploying gene editing platforms to knock out: 1) the CCR5 receptor to treat HIV; 2) the BCL11a enhancer to treat hemoglobinopathies; and 3) T-cell receptors and other proteins to enable allogeneic and enhanced CAR-T and TIL products. Current efforts are focused on ex-vivo applications of these tools, but longer term potential exists for in-vivo therapeutics. Advances are also quickly being made in correcting specific mutations and inserting new genes in a very targeted fashion. • Imagine the possibilities: The value of precisely correcting DNA is immeasurable if accomplished with precision and control. Already discussion has begun whether these products may be suitable for germline correction of disease (e.g., in utero); while this debate rages, the platforms continue to evolve and improve in a direction where someday this should be feasible. Until then, we expect there will be no shortage of opportunity for these platforms across various therapeutic applications. DOWNLOAD: PIPER GENE EDITING REPORT PART1.pdf (675.38 KB) |
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Re: Gene Therapy 2.0: Gene Editing Primer; New Tools Emerging To
Gene Therapy 2.0: Gene Editing Primer; New Tools Emerging To yesterday at 4:16am Post by Ma$†¡N® on yesterday at 4:16am
DOWNLOAD: PIPER GENE EDITING REPORT PART2.pdf (948.94 KB)
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Re: Gene Therapy 2.0: Gene Editing Primer; New Tools Emerging To
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