Explore more Categories
Artificial Intelligence involves the science and engineering of developing intelligent machines
The manufacturing world is at a cusp of a new revolution that will redefine conventional business models
IoT will have a major impact on businesses and consumers in the future
The world is creating 2.5 exabytes of data daily
Our research seeks to connect-the-dots across multiple trends
Personal Robots are Set to Be a $19 Billion Market
Strategic Analysis of BREXIT and its Implications to Industries, Economies and Societies
iFrost is a digital platform for interactive and dynamic reporting of data.
The TOEs are a great source of information which gives corporates critical information on companies to action for their strategic investments either as an open source platform or to collaborate and to develop successful products
A new wave of gene therapies is set to revolutionize treatments over the next decade for inherited diseases which are either untreatable or which do not have a satisfactory treatment outcome at present
save 10 %
* Required Fields
Pay by invoice
The drug approvals of Yescarta (axicabtagene ciloleucel), Luxturna (Voretigene neparvovec) and Kymriah (Tisagenlecleucel) have turned a leaf in the pharmaceutical chapter of gene therapy development. Currently, genetic disorders do not have satisfactory treatment outcomes. This is in part due to the inability of small molecule drugs and enzyme replacement therapies, usually prescribed for inherited diseases, to correct the root cause of the disorders. In other words, they are not corrective therapies. Gene therapy offers exactly what small molecule drugs and enzyme therapy therapies don't- they can offer corrective solutions for inherited disorders.A new wave of gene therapy breakthroughs can be expected to follow in the next few years after a two-decade long hiatus that witnessed very few proven gene therapy drug candidates. This potential in-flux of gene therapy candidates is due to the two fundamental factors. First, the refining and development of cutting-edge vector platform both by academia and by pharmaceutical industry. This has resulted in the widely used advanced Adeno-associated viral vectors and Lentiviral vectors, both of which are used according to the applicability. Second, the development of novel gene editing tools such as TALENs, Meganucleases, CRISPR/Cas along with the improvement of old techniques such as ZFNs have helped improve the translational capability of gene therapies currently in clinical development.In the following study, the development of gene editing tools as well as gene-transfer enabling vectors is traced over the last decade or so while also putting into perspective the number of gene therapy candidates currently in clinical development in addition to the various potential cures and satisfactory treatments that await patients affected by genetic diseases .
Precision Immunotherapy Innovations Transforming Human Health and Wellness
Global Single Cell Genomics and Proteomics Market, Forecast to 2023
Chinese Clinical Laboratories Market, Forecast to 2025
Vietnamese Clinical Laboratories Market, Forecast to 2025
Breakthroughs in Non-Alcoholic Steatohepatitis Management
Thailand Clinical Laboratories Market, Forecast to 2025
Malaysian Clinical Laboratories Market, Forecast to 2025
Microbiome Technologies Fueling Human Health and Wellness
Analysis of the Singapore Clinical Laboratories Market, Forecast to 2021
US Medical Marijuana Market, Forecast to 2022
fleet management market
residential energy storage market
fuel cell market
cloud security market
neurology devices market
aerospace industry outlook
solar cell market
power electronics market
smart meter market
Fill in the fields below to receive a sample report
Don't have an account? Create One!
Enter your Email Address here to receive a link to change password.
If you are an existing frost.com user, please register using the same email for seamless access
Already have an account? Login!
Use your Linkedin account to login or register within our store.You're just one click away.