Regulatory Framework for Gene Therapies Incorpora:ng Human Genome Edi:ng
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1 Regulatory Framework for Gene Therapies Incorpora:ng Human Genome Edi:ng Anna Kwilas, Ph.D. CMC Reviewer, Gene Therapy Branch Division of Cellular and Gene Therapies Office of Tissues and Advanced Therapies Center for Biologics Evalua:on and Research Food and Drug Administra:on European Biopharmaceu8cal Enterprises 6th Annual Regulatory Conference December 5, 2017
2 Gene Therapy & Genome Edi:ng Gene therapy products mediate their effects by transcrip:on or transla:on of transferred gene:c material, or by specifically altering host gene:c sequences. Human genome edi:ng is a process by which DNA is inserted, deleted, or replaced in the human genome using engineered site-specific nucleases and is therefore regulated as a gene therapy. 2
3 Regula:on of Genome Edi:ng Products CBER has been regula:ng gene therapy products since 1989; the first submissions for genome edi:ng products were received in INDs, 6 Pre-INDs, 6 Pre-pre-INDs Science-based approach Benefit-Risk analyses Poten:al to correct or remove defec:ve genes Risk of off-target genome modifica:on Unknown long term effects of on- or off-target genome edi:ng 3
4 Considera:ons for Developing Human Genome Edi:ng Products Type & degree of modifica:on needed Nuclease design Op:miza:on of targe:ng elements Delivery method Viral vectors, plasmid DNA, mrna, protein (RNP) Direct administra:on Modifica:on of cells ex vivo 4
5 Considera:ons for Developing Human Genome Edi:ng Products Safety and efficacy Op:miza:on of genome edi:ng component expression Target valida:on studies Preclinical studies What models are available/appropriate? What will you monitor sequence, expression, func:on? Clinical trial design, pa:ent monitoring, long-term follow-up 5
6 Human Genome Edi:ng Safety Concerns Specificity Minimizing off-target edi:ng events Sensi:vity of off-target screening methods Addi:onal adverse effects due to genomic DNA cleavage at on- and off-target sites Transloca:ons, inversions, etc. Immunogenicity Adverse impact of the delivery system 6
7 Challenges to Addressing Human Genome Edi:ng Safety Concerns Mul:ple methods for predic:ng and iden:fying offtarget genomic modifica:ons Not all genomic modifica:ons lead to adverse biological consequences Animal models may be of limited value Iden:fying and evalua:ng off-target genomic modifica:ons Determining immunogenicity Evalua:ng the delivery system 7
8 Methods for Iden:fying Intra- Chromosomal Off-Target Modifica:ons In silico methods Computa:onal methods iden:fying areas of homology to targe:ng sequence (e.g. BowTie2, BFAST, Cas-Off-Finder) Plaeorms are based on different algorithms and ofen give different results Cellular methods PCR amplifica:on of tagged sequences allows iden:fica:on of edited sites (e.g. Guide Seq, BLESS/BLISS, IDLV Capture) Off-target edi:ng events may be cell type specific Biochemical Methods Sequencing of edited, fragmented DNA (e.g. SELEX, Circle Seq, DiGenome Seq) May give rise to many false posi:ve hits 8
9 Methods for Iden:fying Inter- Chromosomal Off-Target Modifica:ons In silico modeling Cellular approaches Unidirec:onal sequencing (e.g. HTGTS, AMP-seq, UDiTaS) Imaging based genome analysis (e.g. BioNano, FISH, karyotyping) Whole genome sequencing 9
10 Assessing the Safety of Human Genome Edi:ng Products How are nucleases and donor sequences produced? What are the kine:cs of nuclease cleavage ac:vity? Has there been thorough evalua:on of poten:al off-target sites? Types Downstream consequences Ra:o of cleavage at on- versus off-target sites 10
11 Assessing the Safety of Human Genome Edi:ng Products What models have been used to assess safety and ac:vity? Are genome edi:ng components ac:ve in the model? Is model informa:ve for on- and off-target edi:ng? Has safety of delivery vector been assessed? In the case of direct administra:on, have off-target cells/:ssues been characterized? Has data been generated to inform long term follow-up of poten:al study subjects? 11
12 Early Communica:on with CBER/OTAT Pre-pre-IND interac:ons Non-binding, informal scien:fic discussions between CBER/OTAT nonclinical review disciplines (P/T & CMC) and the sponsor Ini:al targeted discussion of specific issues Primary contact: Mercedes Serabian Pre-IND mee:ngs Non-binding, but formal mee:ng between FDA and sponsor (with minutes generated) Mee:ng package should include summary data and sound scien:fic principles to support use of a specific product in a specific pa:ent popula:on hlps:///downloads/drugs/guidances/ ucm pdf 12
13 Summary Gene therapies based on genome edi:ng technologies require careful design and thorough evalua:on On-target edi:ng efficiency Off-target edi:ng effects Delivery method Immunogenicity Evalua:on should consist of: Appropriate models Mul:ple orthogonal methods We do not endorse any specific methods, you should use what works best in your system and provide jus:fica:on for your chosen tools Regulated using a science based approach, with considera:on of the benefits & risks of each product 13
14 CBER Contact Informa:on Anna Kwilas Gene Therapy Branch, Division of Cellular & Gene Therapies OTAT/CBER/FDA WO Bldg. 71 Room New Hampshire Ave. Silver Spring, MD Tel: Regulatory Ques8ons: Contact the Regulatory Management Staff in OTAT at or References for the regulatory process for OTAT hlp:///biologicsbloodvaccines/guidancecomplianceregulatoryinforma:on/ OtherRecommenda:onsforManufacturers/ucm htm OTAT Learn Webinar Series: hlp:///biologicsbloodvaccines/newsevents/ucm htm 14
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