Regulatory Requirements for CRISPR Therapeutics. Bill Lundberg, MD February 2017
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1 Regulatory Requirements for CRISPR Therapeutics Bill Lundberg, MD February 2017
2 Forward Looking Statements This document contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to statements concerning the timing of our preclinical studies and the intellectual property protection of our technology. All statements, other than statements of historical facts, contained in this document, including statements regarding the Company s strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, and objectives of management, are forward-looking statements. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies for the Company s product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; expectations for regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for our technology; and other factors discussed in the Risk Factors section of the Company s most recent registration statement on Form S-1 (file no ), which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this document represent the Company s views as of the date of this document. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company s views as of any date subsequent to the date of this document. I am a paid employee, shareholder and officer of CRISPR Therapeutics 2
3 CRISPR/Cas9: The Next Medical Breakthrough A new technology for editing defective genes has raised hopes for a future generation of medicines 3
4 CRISPR/Cas9 Mechanism of Action Cas9 DNA Guide RNA (grna) DISRUPTION CORRECTION GENE REGULATION Hemoglobinopathies Immuno-oncology Duchenne muscular dystrophy Hurler syndrome Hemophilia Cystic fibrosis Multiple diseases 4
5 CRISPR-Based Therapies Personalization Duchenne muscular dystrophy: One disease many mutations Autologous therapies: The ultimate personalization Rapid approval across different mutations after initial trials is challenging in current environment Rely on harvesting a patient s own cells, editing them and re-introducing them back to the patient 5
6 CRISPR-based Therapeutics Ex Vivo & In Vivo Ex vivo In vivo Delivery formats Nucleic acid Liposome Viral particle Engineered cell 6
7 Regulatory Requirements for Approval The FDA must approve a BLA if it shows that the proposed product is safe, pure, and potent and the facilities where the product is made, processed, packed, or held comply with good manufacturing practice (GMP). PHSA 351(a)(2)(C); 21 C.F.R (a) Safe Pure Potent 7
8 FDA Regulations Relevant for Genome Editing Human cells, tissue or cellular-based products (HCT/P) Section 361, Public Health Service Act, infectious disease Regulatory Law: 21 CFR (d) Biologics Section 351, Public Health Service Act, premarket, approval, safety and effectiveness Regulatory Law: 21 CFR 600 Drugs Food, Drug and Cosmetic Art Regulatory Law: 21 CFR 200 Human cells, tissue or cellular-based products (HCT/P) Section 361, Public Health Service Act, infectious disease 88
9 CFR Specifications for Biologic Products Safety Code of Federal Regulation , , , Endpoint Free from opportunistic viruses, mycoplasma and endotoxins Bacterial and fungal sterility Purity Free of extraneous materials Identity Specific test to distinguish it from other similar substances Constituent Materials Identify all ingredients, preservatives, diluents, adjuvants and excipients Potency Assay for biological function 9
10 Evolution of CBER s Products Proteins purified from plasma Recombinant Proteins Cell and Gene Therapies Example: Factor VIII Concentrate (licensed) Recombinant Factor VIII (licensed) Factor VIII Gene Therapy (in development) 10
11 Reorganization of CBER - OTAT Office of the Director (OD) Office of Blood Research and Review (OBRR) Office of Cellular, Tissue and Gene Therapies (OCTGT) Office of Vaccines Research and Review (OVRR) Office of Biostatistics and Epidemiology (OBE) Office of Compliance and Biologics Quality (OCBQ) Office of Communications, Outreach and Development (OCOD) Office of Management (OM) OBRR s Division of Hematology Clinical Review Division of Hematology Research and Review Office of the Director (OD) Office of Blood Research and Review (OBRR) Office of Tissues and Advanced Therapies (OTAT) Office of Vaccines Research and Review (OVRR) Office of Biostatistics and Epidemiology (OBE) Office of Compliance and Biologics Quality (OCBQ) Office of Communications, Outreach and Development (OCOD) Office of Management (OM) Effective Date: October 16,
12 Nucleases for Gene Editing Zinc Finger Nucleases (ZFNs) Transcription Activator-Like Effector Nucleases (TALENs) Engineered Meganucleases Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9)
13 Selected Guidance Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products (November 2013) Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products (June 2015) Guidance for FDA Review Staff and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), 2008 Guidance for Industry: Gene Therapy Clinical Trials- Observing Subjects for Delayed Adverse Events (November 2006)
14 Desirable Scientific Considerations Efficient delivery On target gene modification Targeted expression Long lasting expression Depends on indication Immune response to vector or transgene Off target editing Off target expression Insertional mutagenesis Undesirable Other Considerations Potential germline transmission
15 Ex-vivo Editing of Human CD34+ Cells CCR5 Precedent with Zfn Nucleases (SGMO) Evaluating ZFN specificity in CD34 + HSPCs Deep sequencing at CCR5 target site (and all identified off-target sites) 53BP1 double-strand break assessment in control and treated CD34+ hematopoietic stem and progenitor cells (HSPCs) Standard in vitro safety assays Soft agar transformation assay in control and treated human fibroblast cell line Karyotype analysis of control and treated CD34 + HSPCs Tumorigenicity in NSG Mice Patient dose derived from 3 separate donors 5-monthduration to assess potential tumor formation 15
16 In-vivo Editing Albumin/hF9 Precedent with Zfn Nucleases (SGMO) Surrogate ZFNs and hf9 donor components required due to species-specific differences in DNA sequences at the albumin intron 1 target locus Mouse Nonhuman primate: cynomolgus and rhesus monkeys Parameters determined Single-Dose IV administration 1:1:8 ratio for ZFN1:ZFN2:hF9 donor Co-administration of three AAV vectors on Day 1 Pharmacology/Toxicology studies Pilot and GLP pharmacodynamics (PD), biodistribution (BD) and toxicology studies of AAV2/6 vectors in C57BL/6 mice A sequential-dose PF, BD and toxicity study evaluating single- or co-administration of AAV2/8 vectors in rhesus monkeys; cynomolgus monkey bridging study A dose range-finding and dose-ratio study of AAV2/6 vectors in cynomolgus monkeys Biodistribution evaluation FDA recommended BD evaluation for each new AAV2/6 project 16
17 Clinical Dose Selection/Justification Identify target expression/effect from clinical data, model systems or pharacometric modeling Understand relationship between genetic modification(s) and pharmacodynamic effect, and if cell autonomous or not Establish target editing rate (allelic rate vs cell rate) at target tissue Determine delivery relationship between dose and target editing rate Buid Dose-Exposure-Effect model and if possible test experimentally 17
18 EU: Advanced Therapy Medicinal Products (ATMP) Regulatory Framework Robust ATMP regulatory framework in place Regulatory engagement at the National Level (CA) vs EMA Level Dedicated committee for advanced therapies (CAT) implemented at EMA level ATMP related guidelines available, more to come 18
19 EMA Guidelines for ATMPs 19
20 ATMPs That Are or Were EU Approved ATMP Category Status ChondroCelect Glybera MACI Provenge Holoclar Imlygic TEP, Repair of symptomatic cartilage defects of the knee (1-5cm²) GTMP, Treatment of lipoprotein lipase deficiency TEP, combined Repair of symptomatic cartilage defects of the knee (3-20cm²) CTMP, Treatment of prostate cancer TEP, Treatment of limbal stem cell deficiency due to ocular burns GTMP Oncolytic virus for treatment of melanoma approved approved approved, now suspended approved, no longer authorized Approved approved Biopharma Excellence,
21 The challenges for ATMPs General Shortcomings in all part of the dossier Quality, e.g. Characterization / Potency assay Comparability following manufacturing changes Nonclinical, e.g. Justification for alternative strategies Biodistribution, tumorigenicity studies Clinical, e.g. Mechanism of action may not be directly linked to the nature of the product Issues with study design: endpoints, comparator, size Safety considerations for additional procedures, e.g. surgery Biopharma Excellence,
22 Special regulatory tools for ATMPs ATMP classification at EMA level To obtain confirmation or clarity on ATMP status and category ATMP certification Assessment of Module 3 (Quality) and Module 4 (Nonclinical) data by CAT for SMEs Need to balance effort vs. benefit Meeting with EMA Innovation Task Force Procedural aspects, legally not binding Biopharma Excellence,
23 Issues Identified by Stakeholders: Follow-up From EMA ATMP Workshop On 27 May 2016 EMA hosted a workshop aimed to foster ATMP development and enable expanded patient access in the EU. Ongoing EU initiatives to optimize the current regulatory framework to facilitate ATMP development..a proposal to revise the legislation underpinning ATMPs in Europe is currently not foreseen. ATMP scientific and regulatory guidance Tailored GMP and GLP for ATMP Focus on benefit-risk assessment by the CAT Orphan similarity of ATMPs Procedural guideline on the evaluation of ATMPs for MAA Support to more transparency in hospital exemption 23
24 Human Genome Editing: Science Ethics & Governance Report from the NAS/NAM/FDA and other co-sponsors released Tuesday February 14, 2017 Recommendations NON-HERITABLE (Somatic Cell) editing clinical trials should be limited to treating or preventing disease or disability at this time HERITABLE (Germline) editing clinical trials could one day be permitted for serious conditions 24
25 Principles for Governance of Human Genome Editing Promoting well-being: beneficence and non-maleficence. Transparency: openness and sharing of information Due care: proceeding carefully supported by sufficient evidence Responsible science: highest standards of research Respect for persons: personal dignity, choice, individual decisions. All people have equal moral value, regardless of their genetic qualities. Fairness: Like cases be treated alike, and that risks and benefits be equitably distributed (distributive justice). Transnational cooperation: [International] collaborative approaches to research and governance while respecting different cultural contexts. 25
26 CRISPR Therapeutics Position on Genome Editing December 2015 We are committed to: Using gene editing technologies, including CRISPR/Cas9, only for patient benefit. Focusing on treating serious diseases of high unmet need. Refraining from directly modifying germline cells, including sperm, egg or embryonic tissue, or developing any clinical applications of germline gene editing. 26
27 27
28 Thank you Acknowledgements - FDA CBER, EMA - Biopharma Excellence
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