Leaders in Pharmaceutical Business Intelligence (LPBI) Group

Katrine Bosley
Chief Executive, Editas Medicine
Transformative Genome Editing

Repair broken genes, Cystics fibrosis, Huntington, 6,000 genetic diseases, 25 years ago sequences,

CRISPR: ability to sequence rapidly and cheaply

• guide RNA – complex nuclease protein (Cas9)
• leverages native pairing mechanism od RNA and DNA to precisely
• diffrent Mutations require diferent Edits
• Cur & Revise, cut and remoce, cut anf repair
• Multiple Options for Delivery of CRISPR Mediicnes
• EDITAS – 4 parts
  • 1.achieve the right repaire
  • 2. specificically
  • Expanding technology to helpmore patients
  • Leber Concenital Amaurosis (LCA10) a genetic form of progressive blindness  – retinal degeneration, no tritment in US or Europe – visual accuity measures
  • CEP290 Gene Editing to Treat LCA10
  • Exon 25,26, glitch exon 27, 28
  • get rid of glitch remove the piecce of DNA
  • functional Full Length Protein
  • mRNA Expression vs Increase Full-Length Protein Expression
  • Potential to repair any broken gene
  • recombinant protein

Jason: visus mechanism as inspiration

• Augmentation vs PREVENTIVE MEDICINE

Ethical edit human germ line – is not scientific but societal, regulatory FDA advisory Committee at NIH — debate to have

Editas, CEO

• Gentic heritage – alterations in,
• On the question of Moratorium on Human Germ Line – Editas is dealing wiht adult cells
• Clinical Trials, When?? What do you work on parallel or in sequence
• 2009 raising money was hard in 2015 – parallel technology of CRISPR – understand mutation and disease
• investing in parallel is reasonable
• Clinical Trials in 2017
•  Team and Capital — IP Contraversy: intensity,
• Cas9 – Other Enzymes?? Other enzymes — features are different in 5 years we will know more
• Specificity, cut and repair is getting to Medicine
• Alternative methods will be better to be used with other methods
• Diseases to target Vision, Liver,
• Sickle Cells: Stem cells to be use in Gene Therapy vs Gene Editing – correct gene in place vs systemic

Complement your views with her Perspective by reading

CRISPR Gene Editing to Be Tested on People by 2017, Says Editas

A biotechnology company says it will test advanced gene-engineering methods to treat blindness.

• By Antonio Regalado on November 5, 2015 in MIT Technology Review

http://www.technologyreview.com/news/543181/crispr-gene-editing-to-be-tested-on-people-by-2017-says-editas/?utm_campaign=newsletters&utm_source=newsletter-weekly-biomedicine&utm_medium=email&utm_content=20151110