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The Future of Human Gene Editing—Will CRISPR Therapeutics AG Lead the Way?

The percentage of the population who suffer from genetic disorders is five percent or less. But recently, scientists have determined that as much as 20 percent of the population have disease-related genetic mutations. Given this, gene-editing techniques – notably, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) – have garnered attention for potential treatment opportunities. And CRISPR Therapeutics AG, a leader in gene editing, appears to be paving the way for these precision medicine techniques.

crispr therapeutics, gene symbol
One day we will no longer have to rely on hit or miss treatments.

While planned trials at CRISPR Therapeutics is exciting, gene editing has yet to be proven as an effective treatment. Likewise, this precision medicine approach to healthcare may pose some risks and challenges as human trials advance.

However, with over 6,000 different known genetic disorders, the potential for success is great. And companies like CRISPR Therapeutics are investing heavily on its promise as a treatment for the future.

CRISPR as a Gene Editing Treatment for Patients

The use of CRISPR for gene editing is not new. CRISPR is being used in agriculture, biofuels and other health areas to advance innovation. In this regard, CRISPR Therapeutics represents one of the original companies to explore this gene-editing technique in human healthcare. By using a viral defense mechanism found in bacteria, CRISPR can disrupt, correct or regulate disease-related genes. And by targeting specific DNA areas, CRISPR Therapeutics hopes to add this approach to the scope of precision medicine care.

At the current time, CRISPR Therapeutics is exploring ways to use gene editing in a number of human health conditions. Patients with genetic blood disorders like sickle cell disease and B-thalassemia are undergoing trials for Phase I/II. Additional areas where CRISPR Therapeutics plans to experiment with treatment include cancer, muscular dystrophy, cystic fibrosis, and others. Once there is an identification of specific gene targets, gene editing could manage the disease more effectively, if not cure it altogether.

a photo quote of Samarth Kulkarni on CRISPR Therapeutics
Dr. Samarth Kulkarni weighs in on what to expect as they bring CRISPR technology to patients.

CRISPR Therapeutics—A Company with Great Potential

CRISPR Therapeutics formed in late 2013. Since that time, the company has been extremely active in its gene editing pursuits. One of its three co-founders was Dr. Emmanuelle Charpentier, who is recognized as one of the original developers of the CRISPR technique. The development of gene-editing platforms helped form partnerships with key companies in the healthcare field.

Specifically, CRISPR Therapeutics entered a joint venture with Bayer Healthcare as well as with Vertex Pharmaceuticals. These and other collaborations have allowed CRISPR Therapeutics to significantly accelerate its investigational studies.

Most recently, CRISPR Therapeutics announced that it had treated its first patient with its flagship treatment, CTX001. The CTX001 human trials with Vertex are investigating treatments in patients with B-Thalassemia. A similar CTX001 trial is also being started with patients with sickle cell disease. Results may be ready by 2022, and if favorable, CRISPR Therapeutics could usher in a new era of gene editing therapeutics.

CRISPR Therapeutics has developed transformative medicines to treat diseases, according to CEO Samarth Kulkarni.
In medicine, the first step is always the biggest step.

The Potential Impact (And Concerns) Over Gene Editing in Humans

At this point in time, CRISPR Therapeutics has yet to generate any significant revenue from gene-editing activities. This is primarily due to the fact that treatments need more proof to show that they are safe and effective. Potential concerns over gene editing using CRISPR as a therapeutics tool include gene editing missing its targets, as well as the possibility that the gene edits could trigger cancer formation. This factor is the reason why the current clinical trials are so important in predicting the future of gene editing in managing human diseases.

Though concerns exist, optimism far exceeds pessimism in this regard. In fact, stock prices of CRISPR Therapeutics are rising, and the company has a current market cap of $2.06 billion. It still may be several years before an actual gene-editing treatment may come to market. But the impact a successful treatment could yield has patients and investors alike very excited. Certainly, similar companies exist. However, CRISPR Therapeutics looks to be the most likely to introduce a gene-editing breakthrough in the healthcare field.

The Fourth Industrial Revolution: 3D Printing and Additive Manufacturing Technologies

There was great enthusiasm for 3D technology when it was first introduced. Many even predicted that there would be a presence of a 3D printer in every home. Consumers could “print” anything they needed ranging from kids’ toys to automotive parts, to a lot more. Like many Bold Ideas, 3D printing is taking time to make a significant societal impact that was first touted and predicted. However, it doesn’t mean additive manufacturing technologies are not incredibly beneficial and are changing industries.

In this exclusive Synapse Summit interview with Scott Gebicke, Vice President global energy and industrial conglomerates at Jabil, he discusses the future of 3D printing and its impact on manufacturing services.

 

Today, a variety of industries utilize additive manufacturing technologies. These not only include product manufacturing but also medical, aerospace and construction sectors. Rather quietly, advances in 3D printing technology are allowing businesses to thrive and innovations to occur. 3D printing technology also offers new solutions to solve some of the world’s most challenging problems. As a result, taking a fresh look at additive manufacturing technologies is certainly worthwhile.

3D Printing Technology’s Rocky Start

It is important to understand what 3D printing is before anyone can fully appreciate its advantages and impact. Essentially, 3D printing and additive manufacturing technologies are synonymous terms. Both refer to the ability to create objects using layer-based manufacturing techniques. To reconstruct a design, special software adds materials in layers along with a 3D printer. These materials can involve wood, plastics, metal, and others.

3D printing, joshua harker quoted
Independence is one of the potential effects of 3D printing.

Additive manufacturing technologies offered great hope and excitement initially for everyone. But in time, the reality was not quite as profound as the vision because a special degree was part of the requirement to manage the software and 3D printer. Likewise, object design skills were essential talents and highly a requirement to truly reap the benefits at the consumer level. In time, it has become increasingly evident that additive manufacturing technologies is something the masses are not yet ready for.

additive manufacturing technologies, tooth made from 3d printing
Human health will significantly benefit from additive manufacturing technologies.

Realizing the Advantages of Additive Manufacturing Technologies

3D printing technology, despite its absence in homes today, still offers tremendous benefits. For one, 3D printing technology requires fewer labor resources resulting in reduced cost in manufacturing. Likewise, 3D printing technology allows for just-in-time production and less inventory management expense. And, once digitalized, additive manufacturing technologies allows fewer mistakes and more rapid changes when required. All of these offer powerful advantages in manufacturing.

These insights show us the recognition that 3D printing technology is primarily a wonderful manufacturing tool for companies. And in fact, the way many industries utilize additive manufacturing technologies is extremely exciting. For example, footwear, hearing aids, and orthodontics are being custom-made for individuals using 3D printing technology. Likewise, additive manufacturing technologies offer new geometric configurations advancing product design. And increasingly, materials are being improved that reduce weight and are easier with which to work. Product flexibility, customization, and production speed are all clear advantages that 3D printing technology provides.

Jabil is a leader in engineering, manufacturing and intelligent supply chain solutions. They are experts in the use of 3D printing to expedite the process of prototyping and the reduction of errors. By using 3D printing technology, Jabil is able to deliver parts in hours instead of days and it provides their designers the ability to analyze and adjust the final product. They are working with many global customers to help transform the factory of the future including how shoes are made, which Scott Gebicke describes in his interview.

The Societal and Environmental Impacts of 3D Printing Technology

While the manufacturing advantages are noteworthy, 3D printing technology is also providing innovative solutions in other areas. From improving human health and living up to environmental sustainability, additive manufacturing technologies are making a difference. The following showcase just a few of these incredible applications for 3D printing technology.

  • Disaster Relief Solutions – Environmental disasters pose serious threats to our well-being, and solutions are often needed to reduce human suffering. 3D printing has been used in this regard to improve dire situations. For example, Field Ready provided on-site 3D-printed waterpipe fittings during the 2015 Nepal earthquake. Likewise, Contour Crafting has utilized additive manufacturing technologies to accelerate home rebuilding after disasters.
  • Housing Solutions – In many areas throughout the world, adequate housing is a problem. From urban homeless to impoverished regions, however, 3D printing technology offers new solutions here as well. For instance, Framlab is a design agency seeking to create 3D printed pods to alleviate homelessness in New York City. ICON and New Story teamed up to create a 3D-printed home within a 24-hour time period. Priced at $4000, the 800 square-foot home offers a design for affordable housing.
3D printing technology, housing units of honeycomb
Framlab, Oslo and New York City-based creative design agency, uses 3D printed modules in their designs.
  • Health and Disability Solutions – The potential use of additive manufacturing technologies to create bodily organs has been exciting and highly anticipated. Likewise, dental and orthodontic uses of 3D printing technology are also incredibly interesting. But other companies like 3D Life Prints are utilizing these technologies to provide innovative prosthetics. And Glaze Prosthetics creates “cool” superhero-designed prosthetics to enhance their aesthetic effects.
  • Environmental Protection Solutions – Perhaps one of the more intriguing uses of 3D printing technology relates to the blue economy. Threats to coral reefs are well appreciated today as a result of climate change. But collaborations among organizations are using additive manufacturing technologies to create potential solutions. Emerging Objects, Boston Ceramics, and the SECORE organization teamed up to create 3D-printed substrates for coral larvae. This could protect coral reefs and enhance their resilience in the process.

Trends in 3D Printing Technology Moving Forward

The previously mentioned uses of 3D printing technology to aid human health and society are impressive. At the same time, many new 3D-printed designs offer better configurations and reduced weight. These changes have positive influences on energy utilization and sustainability especially when it comes to urban planning. The beneficial influences that this new technology can have on global problems are continuing to be unveiled.

additive manufacturing companies, barrack obama quoted
The way we make things is changing.

The potential for 3D printing technology remains significant in the future. Indeed, most of the advantages of additive manufacturing technologies are within manufacturing areas. But new and exciting applications continue to emerge even outside of these production areas. Likewise, recent trends show that expertise in 3D printing technology is advancing. And materials available for 3D printing continue to get better. In all likelihood, the variety of uses that 3D printing technology offers will continue to expand.