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Gene Editing

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How should established biopharma companies position themselves to respond to, and capitalize on, the emerging science/technology of gene editing?

Biotechnology
Gene Editing
Genomics
Ethics
Regulation
Rob Walsh
11 days ago

8 answers

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A very important aspect of gene editing is to avoid off target effects. Established biopharma companies have the volume and resources to exhaustively test CRISPR guide RNAs using whole genome sequencing. Ideally using patient's cells, as everyone's genome is unique and thus offers unique opportunities for off-target effects.

Leonardo Ferreira
11 days ago
Compared to few years ao there has been a rapid surge of new nucleases being reported offering much more specifity. Earlier we only had known about cas9, but today we have several nucleases being reported. - Venkatramana 4 days ago
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Using the whole genome sequencing analysis, is an unique and thus offers unique opportunities for off-target effects in researches regarding process of transcription of ADN.

Aurelian Udristioiu
11 days ago
I agree, WGS on individaul humans is relavant. There is a great deal of innovation happening in the space. Illumina recently accqured PACBio and now has teh ability to integrate Long read technology and improve the data quality - Venkatramana 4 days ago
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n/a

Aurelian Udristioiu
11 days ago
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n/a

Aurelian Udristioiu
11 days ago
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Bio-engineering offers fascinating possibilities, but also deep ethical pitfalls. A further problem, the legal environment is often not defined, or at least official interpretation of local laws are still pending. If pharma companies want to follow this path, they need a strong Ethics & Compliance department.

Patrick Henz
11 days ago
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Gene mutations are a part of normal biology. It has now been developed into a tool, and as with all tools, it can be used well or badly, to a benefit or to harm. (The use and misuse of nuclear power might be a relevant parallel here.)
The main issue is that science does not understand well enough what each gene does and even less how functions of various genes overlap. Consequently, gene manipulations are at present a game of a “blind leading the blind”, and is likely to remain this way for some time. As to the actual outcome of gene modification in humans, it is a game of roulette; will your number come up?
Pharma industry might be well advised to consider gene editing as a part of the overall concept of precision / personalized medicine.

Karel Petrak
11 days ago
I agree the key is to have acces to highly validate target site on whihc gene editing tools can be applied. A through understaning on the gene funcation and the underalying phenotypes is essential. - Venkatramana 4 days ago
My concern is that a whole generation may be unknowing participants/guinea pigs in experiments in which the validity of the outcomes and the repercussions are unprove.n. - Apollone 3 days ago
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As we learn more about the impact of CRISPR technology on healthcare delivery and outcomes, it is likely that large drug companies will want to be in the business of offering highly individualized treatments for specific ailments, especially as the technology overtakes the need for medications where they currently make their profits.

CRISPR technology has at it's heart the promise of a robust shared database of trials and results for any conceivable disease. If ould reduce surgeries, impact short and long term treatment plans, and holds the promise of reducing healthcare costs significantly. Stay tuned and watch emerging developments impact our future.

Sandy Waters
7 days ago
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It may be too early to consider applications of AI to technologies such as CRISPR shaping future healthcare delivery infrastructure and insurance business. However, AI could and should be used to resolve technical issues such as off-target effects of CRISPR and subsequent serious side effects, efficacy of homology-directed repair, viability of edited cells, immunogenicity of therapeutic components, and also in vivo efficiency etc. when translated from animal models to clinical studies.

Karel Petrak
7 days ago

Have some input?