ALLERGAN ENTERS STRATEGIC R&D ALLIANCE WITH EDITAS MEDICINE TO DISCOVER AND DEVELOP CRISPR GENE EDITING PROGRAMS FOR OCULAR DISEASES
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1 ALLERGAN ENTERS STRATEGIC R&D ALLIANCE WITH EDITAS MEDICINE TO DISCOVER AND DEVELOP CRISPR GENE EDITING PROGRAMS FOR OCULAR DISEASES Novel Development Programs for Potential Treatments of Serious Ocular Diseases
2 ALLERGAN CAUTIONARY STATEMENT REGARDING FORWARD-LOOKING STATEMENTS Statements contained in this presentation that refer to future events or other non-historical facts are forward-looking statements that reflect Allergan's current perspective of existing trends and information as of the date of this release. Except as expressly required by law, Allergan disclaims any intent or obligation to update these forward-looking statements. Actual results may differ materially from Allergan's current expectations depending upon a number of factors affecting Allergan's business. These factors include, among others, the difficulty of predicting future clinical results based on prior clinical results; the timing or outcome of FDA approvals or actions, if any; the impact of competitive products and pricing; market acceptance of and continued demand for Allergan's products; difficulties or delays in manufacturing; and other risks and uncertainties detailed in Allergan's periodic public filings with the Securities and Exchange Commission, including but not limited to Allergan's Annual Report on Form 10-K for the year ended December 31, 2016 (certain of such periodic public filings having been filed under the "Actavis plc" name). Except as expressly required by law, Allergan disclaims any intent or obligation to update these forward-looking statements. 2
3 EDITAS MEDICINE CRISPR GENOME EDITING PLATFORM NOVEL DEVELOPMENT PROGRAMS FOR POTENTIAL TREATMENT OF HIGH UNMET NEED EYE CARE DISEASES The Alliance Editas Medicine is a biotech company pioneering a highly innovative genome editing technology (CRISPR), aiming to treat genetically inherited diseases with significant unmet need. $90M upfront payment for exclusive access to Editas Medicine s ocular pipeline and the option to license up to 5 early stage programs plus contingent development and commercialization milestones and royalties The Technology CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats): potentially revolutionary genome editing approach which targets specific stretches of genetic code to precisely edit DNA. Leber Congenital Amaurosis 10 (LCA10) is Editas Medicine s lead program and has the potential to treat a form of genetic blindness. The Opportunity Potential to achieve specific, targeted modifications in a patient s DNA providing an attractive early stage opportunity to treat a large number of genetic diseases. Reinforces Allergan s commitment to advancing innovation across critical diseases with high unmet medical need and is complementary to ongoing eye care development programs. 3
4 CRISPR TECHNOLOGY HOW DOES IT WORK? Genome editing with CRISPR technology CRISPR technology uses a protein-rna complex composed of either the protein Cas9 or Cpf1 bound with a guide RNA (grna) designed to recognize a particular DNA sequence. The nuclease complex can be engineered to perform one of three genome editing functions; cutting and removing, cutting and revising, or cutting and replacing genomic sequences associated with disease. Cas9 and Cpf1 are enzyme/guide RNA complexes that use the traditional RNA/DNA base pairing mechanism to precisely locate specific DNA sequences with the goal of modifying or editing a disease-associated genomic location. By changing the composition of the guide RNA, the Cas9 or Cpf1 nuclease complex can be reprogrammed to target nearly any genomic location. Guide RNA Guide Sequence DNA Cut Sites Nuclease Guide RNA Images adapted from Editas corporate presentation 4
5 LEBER CONGENITAL AMAUROSIS 10 (LCA10) IS EDITAS MEDICINE S LEAD PROGRAM FOR SERIOUS OCULAR DISEASES LCA10 is a genetic form of progressive blindness Severe retinal disease which often presents at infancy or early childhood CEP290 gene plays an important role in cilia formation. Inherited mutation in the CEP290 gene leads to defective cilia resulting in loss of photoreceptor structure and function, causing degeneration Chromosome 12 position Mutated CEP290 Gene Source: Editas Medicine corporate deck and LCA10 Development Status Clear understanding of genetic basis for disease and clinical endpoints Eye allows targeted local delivery using existing gene delivery vectors Currently in pre-clinical development There are no approved treatments or clinical trials in either US or Europe 5
6 FURTHER EXPANSION INTO RETINA WILL ALLOW US TO FULLY PARTICIPATE IN THE EYE CARE MARKET Rational for CRISPR-based therapeutics for inherited retinal diseases Retina Dry Eye Inflammation Antiinfectives Allergan presence Growing Allergan presence Glaucoma Bubbles represent graphically market size Significant diseases with no treatments Proven Delivery Retinal gene therapy using AAV and LV in the clinic AAV successfully targets RPE & photoreceptors Confined, immune privileged location Limited immune response Oncogenic activity easily detected, confined and potentially amenable to laser ablation Clear, non-invasive endpoints Visual acuity highly measurable Built-in-case control; compare treated eye to untreated eye in physiologically similar environment 6
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