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  • Smith JA, Ng KS, Mead BE, Dopson S, Reeve B, Edwards J, Wood MJA, Carr AJ, Bure K, Karp JM, Brindley DA. Extracellular Vesicles: Commercial Potential As Byproducts of Cell Manufacturing for Research and Therapeutic Use. BioProcess International. 13(4). Read on external site. Categories: Delivery Mechanisms

    Extracellular Vesicles: Commercial Potential As Byproducts of Cell Manufacturing for Research and Therapeutic Use.

    BioProcess International. 13(4).

    Extracellular Vesicles: Commercial Potential As Byproducts of Cell Manufacturing for Research and Therapeutic Use.

    Smith JA, Ng KS, Mead BE, Dopson S, Reeve B, Edwards J, Wood MJA, Carr AJ, Bure K, Karp JM, Brindley DA.

    Abstract

    Abstract:

    Extracellular vesicles (EVs) are emerging as a potential alternative to some stem-cell–derived therapeutics. Sometimes called exosomes, they are small, secreted vesicles that can possess similar therapeutic mechanisms to whole cells, possibly representing the active pharmaceutical ingredient. In the past 15 years, academic and industry interest in EVs has exponentially increased as mounting evidence demonstrates their role in physiology and pathology as well as their therapeutic potential.

    In light of growing efforts in using EVs for research and therapy, optimizing EV manufacturing is important. However, many challenges come with their characterization, scalable manufacture, and regulatory status. Here, we briefly review the biology and therapeutic application of EVs, discuss associated challenges, and suggest how the biotechnology industry could play an important role in overcoming those challenges. Many cell manufacturing companies currently produce EVs but discard them as waste, thereby losing a potentially valuable resource with multiple purposes in a market that’s otherwise rich with an exorbitant cost of goods.

  • Brindley DA, French AL, Baptista R, Timmins N, Adams T, Wall I, Bure K. Cell Therapy Bioprocessing Technologies and Indicators of Technological Convergence. BioProcess International 12(3)s (March 2014). Read on external site. Categories: Beyond the Bench, Delivery Mechanisms

    Cell Therapy Bioprocessing Technologies and Indicators of Technological Convergence.

    BioProcess International 12(3)s (March 2014).

    Cell Therapy Bioprocessing Technologies and Indicators of Technological Convergence.

    Brindley DA, French AL, Baptista R, Timmins N, Adams T, Wall I, Bure K.

    Abstract

    Abstract:

    The cell therapy industry is undergoing a natural evolution from scientific curiosity into a commercially and clinically attractive opportunity (1). This evolution is by no means complete, and growing evidence suggests that its progression is driving significant developments in cell therapy bioprocessing — notably, convergence.

  • Culme-Seymour EJ, Davie NL, Brindley DA, Edwards-Parton S, Mason C. A decade of cell therapy clinical trials (2000–2010). Regen Med. 2012 Jul;7(4):455-62. PubMed: 22817619. Categories: Delivery Mechanisms

    A decade of cell therapy clinical trials (2000–2010).

    Regen Med. 2012 Jul;7(4):455-62.

    A decade of cell therapy clinical trials (2000–2010).

    Culme-Seymour EJ, Davie NL, Brindley DA, Edwards-Parton S, Mason C.

    Abstract

    Abstract:

    The cell therapy industry (CTI) is presently a small but potentially rapidly growing new global healthcare sector. Success is totally dependent on resolving a number of factors unique to cells as therapies, including: manufacturing, enabling technologies, regulation, reimbursement and essential infrastructure. To understand how to solve these challenges in a timely and cost-effective manner, it is essential to be able to forecast the size and resource demands of the sector for a least the next decade. Due to the highly regulated nature of medicines, one predictive method is to analyze the candidate therapies that are currently undergoing clinical trials (i.e., the future pipeline). A search was performed on the website ClinicalTrials.gov using the embedded search engine and key terms relating to ‘cell therapy’. A total of 17,362 files were extracted (27 June 2010) and individually checked for relevance using the British Standard Institute (BSI) definition of ‘cell therapy’. The resulting 2724 trials were then categorized and core information collated, including: trial phase, cell source (autologous/allogeneic), current activity of the trial and responsible national regulatory agency. Key results included: near equal numbers of autologous (46%) and allogeneic (41%) trials; many of the trials are in the later stages – Phase I (49%), Phase II (40%) or Phase III (10%); and there are significantly larger numbers of transient cell therapies (50%) as opposed to permanent cell replacement (5%). This is the first time that the number and composition of all the cell therapy trials on ClinicalTrials.gov has been researched at the level of individual entries, analyzed and published. These data have important planning and resource allocation implications for translational scientists, clinicians, healthcare providers, businesses and governments.

  • Hanna J, Wernig M, Markoulaki S, Sun CW, Meissner A, Cassady JP, Beard C, Brambrink T, Wu LC, Townes TM, Jaenisch R. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007;318(5858):1920-1923. PubMed: 18063756. Categories: Delivery Mechanisms

    Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin.

    Science 2007;318(5858):1920-1923.

    Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin.

    Hanna J, Wernig M, Markoulaki S, Sun CW, Meissner A, Cassady JP, Beard C, Brambrink T, Wu LC, Townes TM, Jaenisch R.

    Abstract

    Abstract:

    It has recently been demonstrated that mouse and human fibroblasts can be reprogrammed into an embryonic stem cell-like state by introducing combinations of four transcription factors. However, the therapeutic potential of such induced pluripotent stem (iPS) cells remained undefined. By using a humanized sickle cell anemia mouse model, we show that mice can be rescued after transplantation with hematopoietic progenitors obtained in vitro from autologous iPS cells. This was achieved after correction of the human sickle hemoglobin allele by gene-specific targeting. Our results provide proof of principle for using transcription factor-induced reprogramming combined with gene and cell therapy for disease treatment in mice. The problems associated with using retroviruses and oncogenes for reprogramming need to be resolved before iPS cells can be considered for human therapy.

  • Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol 2007;25(7):778-785. PubMed: 17603475. Categories: Delivery Mechanisms

    An improved zinc-finger nuclease architecture for highly specific genome editing.

    Nat Biotechnol 2007;25(7):778-785.

    An improved zinc-finger nuclease architecture for highly specific genome editing.

    Miller JC, Holmes MC, Wang J, Guschin DY, Lee YL, Rupniewski I, Beausejour CM, Waite AJ, Wang NS, Kim KA, Gregory PD, Pabo CO, Rebar EJ.

    Abstract

    Abstract:

    Genome editing driven by zinc-finger nucleases (ZFNs) yields high gene-modification efficiencies (>10%) by introducing a recombinogenic double-strand break into the targeted gene. The cleavage event is induced using two custom-designed ZFNs that heterodimerize upon binding DNA to form a catalytically active nuclease complex. Using the current ZFN architecture, however, cleavage-competent homodimers may also form that can limit safety or efficacy via off-target cleavage. Here we develop an improved ZFN architecture that eliminates this problem. Using structure-based design, we engineer two variant ZFNs that efficiently cleave DNA only when paired as a heterodimer. These ZFNs modify a native endogenous locus as efficiently as the parental architecture, but with a >40-fold reduction in homodimer function and much lower levels of genome-wide cleavage. This architecture provides a general means for improving the specificity of ZFNs as gene modification reagents.

  • de Grey ADNJ. Gene therapy. In: Encyclopedia of Aging, Fourth Edition (R. Schulz et al., eds.), Springer, 2006, pp. ???-???. Categories: Delivery Mechanisms

    Gene therapy.

    In: Encyclopedia of Aging, Fourth Edition (R. Schulz et al., eds.), Springer, 2006, pp. ???-???.

    Gene therapy.

    de Grey ADNJ.

    Abstract

    Abstract:

    No abstract available.

  • Kukowska-Latallo JF, Candido KA, Cao Z, Cao Z, Nigavekar SS, Majoros IJ, Thomas TP, Balogh LP, Khan MK, Baker JR. Nanoparticle targeting of anticancer drug improves therapeutic response in animal model of human epithelial cancer. Cancer Res 2005;65(12):5317-5324. PubMed: 15958579. Categories: Delivery Mechanisms

    Nanoparticle targeting of anticancer drug improves therapeutic response in animal model of human epithelial cancer.

    Cancer Res 2005;65(12):5317-5324.

    Nanoparticle targeting of anticancer drug improves therapeutic response in animal model of human epithelial cancer.

    Kukowska-Latallo JF, Candido KA, Cao Z, Cao Z, Nigavekar SS, Majoros IJ, Thomas TP, Balogh LP, Khan MK, Baker JR.

    Abstract

    Abstract:

    Prior studies suggested that nanoparticle drug delivery might improve the therapeutic response to anticancer drugs and allow the simultaneous monitoring of drug uptake by tumors. We employed modified PAMAM dendritic polymers <5 nm in diameter as carriers. Acetylated dendrimers were conjugated to folic acid as a targeting agent and then coupled to either methotrexate or tritium and either fluorescein or 6-carboxytetramethylrhodamine. These conjugates were injected i.v. into immunodeficient mice bearing human KB tumors that overexpress the folic acid receptor. In contrast to nontargeted polymer, folate-conjugated nanoparticles concentrated in the tumor and liver tissue over 4 days after administration. The tumor tissue localization of the folate-targeted polymer could be attenuated by prior i.v. injection of free folic acid. Confocal microscopy confirmed the internalization of the drug conjugates into the tumor cells. Targeting methotrexate increased its antitumor activity and markedly decreased its toxicity, allowing therapeutic responses not possible with a free drug.

  • Byrnes AP. Challenges and future prospects in gene therapy. IDrugs. 2005;8(12):993-996. PubMed: 16320132. Categories: Delivery Mechanisms

    Challenges and future prospects in gene therapy.

    IDrugs. 2005;8(12):993-996.

    Challenges and future prospects in gene therapy.

    Byrnes AP.

    Abstract

    Abstract:

    Gene therapy is a promising technique for treating disease through the modification of gene expression. It is currently being tested not only for correcting genetic defects, but also for treating cancer and other acquired diseases. Although this field is still relatively young, evidence for clinical efficacy has been observed and continued progress seems assured, as clinical trials continue to yield insights into how gene therapy can be applied and improvements are made in gene therapy tools.