Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. function sanitize_gpt_value2(gptValue) vOut += aTags[i].trim().replace(reg, '-').substring(0,40); var aTags = gptValue.split(','); These issues are delaying activation of new sites, prompting players to postpone new clinical trials. “Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review,” commented Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, adding “The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.”. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Oncology will remain the key area of focus for gene therapy applications. Thank you for subscribing! In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centers. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product. But the drug's price could be $3 million per … The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites. However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. "Gene Therapy - Global Market Trajectory & Analytics", https://www.researchandmarkets.com/r/x4ed1e. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. $(document).ready(function() { In the coming years, there will be significant improvement in the number of approvals for new gene therapies. US FDA releases seven guidance documents, updates its policies on gene therapy products with the industry sector booming. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals. Another guidance of the FDA is focused on ‘Human Gene Therapy for Hemophilia’, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrollments for protecting participants from the risk of infection. Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research. - Last updated on { In a Major Setback to the Healthcare System, Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed. dataLayer.push(dataLayerNews); Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled ‘Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up’ determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.