Gene Therapy. The electronic versions of (a) the most recent print edition of Gene Therapy; and (b) all previous print editions of Gene Therapy that were first published within the previous four (4) years, from the later of the Commencement Date or the most recent of any subsequent Renewal Date (both as defined herein), but in any event all editions first published since the Commencement Date, in each case as available for access on the Internet at the URL xxxx://xxx.xxxxxx.xxx/gt together with any additional material that the Licensor makes available to the Licensee.
Gene Therapy. Students will learn the history of gene therapy and the principal concepts behind gene therapy applications. We will cover the early gene therapy failures and the important lessons learned from these studies, which led to modified strategies based upon improved understanding of genetic components and how this knowledge led to successful translation for the treatment of human diseases like genetic disorders and cancer. Genome Editing: Students will be introduced to genome editing technologies, including transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs) and clustered regularly interspaced short palindromic repeats (CRISPR)-associated nuclease Cas9. Bioinformatics: Students will get an overview of bioinformatics resources for analysis of protein sequences. They will be introduced to immunomic and infectivity databases and learn how to deal with immunogenicity analysis. ECTS Lectures Seminars Practical work Digital learning Personal work 5 32 hours 32 hours 86 hours Assessment method 50 % Oral presentation 50 % Review of a research paper. Emerging Viral Infections: Discovery and Intervention Strategies Organization: Hannover Medical School Teaching unit coordinator: Teaching unit coordinator: Xxxxxx XXXXXXXXX, Xxxx XXXXXXXXXXX Position: Professor, Research Center for Emerging Infections and Zoonoses (RIZ) Teaching unit outline This module will address fundamental aspects of viral zoonotic pathogens, such as discovery of novel viruses and assessment of their zoonotic potential, genetic analysis and phylogeny, host restriction factors and virus adaptation to novel host species, pathogenesis, intervention strategies and the use of a molecular toolbox to investigate viral determinants of virulence and transmissibility. Some important well characterized viral pathogens will be discussed in depth to illuminate different prophylactic and therapeutic intervention strategies. Topics addressed
Gene Therapy. 4 1.16 Genentech Knowhow........................................................5 1.17 Genentech Manufacturing Knowhow..........................................5 1.18 Genentech Patent Rights..................................................5 1.19 IND......................................................................6 1.20
Gene Therapy. Gene therapy or gene-based therapies are not covered.
Gene Therapy. During the term of the Program, Millennium and Lilly agree to discuss collaborations within the Field and the area of Oncology concerning candidates for gene therapy arising from the Program. The initial opportunity to begin such discussions concerning gene therapy shall arise when a gene that is appropriate for use in gene therapy is discovered in the course of the Millennium Research Program or a [**] and is brought to the attention of the Joint Management Team. When this occurs, the parties shall have a period of [**] in which to decide whether to begin good faith negotiations with each other concerning a potential gene therapy collaboration on such a gene. If the parties decide to Confidential Materials omitted and filed separately with the Securities and Exchange Commission. Asterisks denote omissions. collaborate on a gene therapy project, the terms of such collaboration shall be governed by the terms and conditions of a separate agreement. If, either (a) a gene described in the preceding paragraph has a potential application only outside of Oncology, (b) the parties choose not to negotiate with each other concerning a collaboration within Oncology, or (c) the parties cannot agree to the terms of a collaboration within Oncology, then each party shall have a co-exclusive right to make, use and sell a Gene Therapy Drug embodying such a gene for all gene therapy applications, both within the Field and outside the Field, within the Territory. Each party shall be entitled to sublicense the co-exclusive rights to make, use and sell each Gene Therapy Drug embodying such gene to one (1) sublicensee per country. Any further licensing of the Gene Therapy Drug embodying such gene shall require the [**]. Each party shall pay the other party a [**] on its Net Sales of the Gene Therapy Product within the Territory. All royalty payments shall be made subject to the provisions of Article VIII of this Agreement. In the event that the total royalty burden of any Gene Therapy Product sold by a party is greater than [**] of the Net Sales of the Gene Therapy Product in the Territory (inclusive of the [**] paid under the terms of this Section 5.3), then that party's royalty payable to the other party shall [**] of the excess over [**] paid by that party; provided, however, the royalty payment to the other party shall not fall below [**] of Net Sales of the Gene Therapy Product in the Territory. Nothing within this Section 5.3 grants Millennium any rights to Candidate Gene...
Gene Therapy. Gene therapy remains the most promising treatment approach for most NCLs, including the transmembrane protein-deficient forms. A relatively large number of cells will need to be transduced in transmembrane protein-deficient NCLs compared to those that lack lysosomal enzymes, as the neighbouring affected cells cannot rely on cross correction from transduced cells (Xxxxxxxx et al., 2015; X. X. Xxxxxxx et al., 2019). The choice of an appropriate vector is therefore essential (Xxxxxxxxxxx, Xxxxxxxx and Xxxxxx, 2014; Xxxxxxx, Xxxxxxxx and Xxxxxxxx, 2018) and several preclinical studies have tested the efficacy of different vectors and serotypes (Xxxxxxx et al., 2004, 2006; Xxxx et al., 2015; M. E. Bosch et al., 2016; Xxxxx et al., 2017; Xxxx et al., 2019), resulting in several clinical gene therapy trials that are now underway for different forms of NCL (xxxxxxxxxxxxxx.xxx, 2019). Gene therapy has also shown promise in other lysosomal storage diseases, including Pompe disease (Xxxxx et al., 2013) and mucopolysaccharidosis type IIIB (Xxxxxxx et al., 2017). Adeno-associated viruses (AAVs) carry a single stranded DNA and naturally infect humans (Xxxxxxxxxxx, Xxxxxxxx and Xxxxxx, 2014; Xxxxxxx, Xxxxxxxx and Xxxxxxxx, 2018). This natural infection can result in problems with antibody responses to the injected virus capsid or protein product (X. X. Xxxxxxx et al., 2019). There are currently 12 different AAV serotypes with over 108 serovars that target different cells and vary in their antigenicity (Xxxxxxxxxxx, Xxxxxxxx and Xxxxxx, 2014; Xxxxxxx, Xxxxxxxx and Xxxxxxxx, 2018). The route of administration, biodistribution and efficiency of gene cassette expression all need to be considered to ensure optimal transfection and effectiveness of the delivered transgene (Xxxxxxxxxxx, Xxxxxxxx and Xxxxxx, 2014; Xxxxxxx, Xxxxxxxx and Xxxxxxxx, 2018). Preclinically, gene therapy approaches have been used for both lysosomal enzyme deficient (Xxxxxxx et al., 2004, 2006; Xxxxxxxx et al., 2012, 2014; Xxxx et al., 2015; Xxxxx et al., 2017) and transmembrane protein deficient forms of NCL (M. E. Bosch et al., 2016; Xxxx et al., 2019). An AAV2-hPPT1 construct was successfully used to rescue several pathological features in a mouse model of CLN1 disease after multiple intracranial injections (Xxxxxxx et al., 2004). AAVs were also used in a canine model of CLN2 disease, in which administration occurred via intraventricular injection of vectors into the cerebrospinal fluid (CSF), leading...
