Emerging Gene Therapies - Trends within the Technological, Clinical, Regulatory and Competitive Landscape
Summary Genomic surgery is increasingly a reality.The last two years have seen the approval of four gene therapies by the FDA - (1) Luxturna, the first in vivo gene transfer therapy (2) Imlygic, an novel immunotherapy, (3) Kymriah and (4) Yescarta, two Chimeric Antigen Receptor T - Cell therapies targeting CD19 for oncological conditions.
Over the next two years, a number of key events will determine the clinical tractability and commercial interest of gene editing approaches. These will be pivotal within the gene therapy field.
Until recently, the field has had limited success outside of CAR-T therapies.Ex vivo cell therapy Strimvelis for ADA-SCID was approved in 2016, but has subsequently been sold by its developer GSK, due to lack of profitability.
The first exon skipping therapy, ExonDys 51, for Duchenne Muscular Dystrophy was approved controversially by the FDA in 2016, but failed to gain approval in Europe based on its efficacy, and its US sales are reportedly slow.
A key challenge in gene therapy development has been delivering enough product into the target tissue.This has meant many gene therapy products have struggled to achieve a high enough level of therapeutic gene expression to induce a clinical effect.
Naturally, selection of therapy areas for the development of novel gene therapy products has been influenced by these technical challenges. Drug developers have prioritized conditions with well-understood pathology, those with simple genetics that are able to be corrected using simpler editing approaches, and those in which the delivery problem is minimized.
Due to the relative ease of achieving high levels of gene therapy product to cells ex vivo, development has been focused in areas where cellular therapy is an established part of clinical procedure - for instance, in the hemoglobinopathies (e.g. beta thalassemia, sickle cell disease) and the lysosomal disorders, where hematopoietic stem cell transfer is commonly used as part of clinical care. Ex vivo products have also seen success in oncology, where ex vivo modification of T-Cells has to express antigens as a method for immunotherapy has achieved remarkable clinical results.
The report "Emerging Gene Therapies - Trends within the Technological, Clinical, Regulatory and Competitive Landscape" provides a comprehensive overview of the emerging gene therapy market.It discusses gene therapy and the technology behind gene editing, outlining the advantages, limitations and current evidence for the platforms under development.
The report discusses relevant clinical studies targeting specific therapeutic indications and highlights examples of current challenges within the field, with a focus on therapies that target the eye, liver, and blood.
Additionally, the report provides a background to the CRISPR patent litigation, a key factor within the gene editing company landscape. It provides profiles of six companies developing gene editing platforms, considers the gene therapy interests of the main pharmaceutical companies, and discusses current regulatory trends in the development of gene therapies.
Scope - What are the key emerging products within the gene therapy landscape? - Which companies have the strongest pipeline of innovative products? - How will gene editing disrupt existing gene therapy products? - What are the regulatory trends for emerging gene therapies? - What are the interests of pharmaceutical companies within the field?
Reasons to buy - Achieve an up-to-date understanding of the area, with a comprehensive reference of key products within the gene therapy landscape, compared across technology-specific relevant characteristics such as editing mechanism and delivery vector. - Conduct competitive analysis using indication-specific, side-by-side comparisons of the latest data for key gene therapy products in the strategically relevant areas of eye, blood, and liver. - Conduct strategic analysis using an overview of gene therapy specific considerations for evaluating and developing gene therapy products - the CRISPR patent space, emerging regulatory trends, innovation leaders and the interests of pharma in gene therapy.
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