How the commercialisation of gene and cell therapies can revolutionise modern medicine

‘If one or two CAR-T therapies get to market it will provide impetus and momentum behind the industry’ – Dr Akshay Peer, Vice President of Sales and Account Management at TrakCel discusses the revolutionary steps that cell and gene therapies are making through their increased commercialisation.

In a field that is constantly seeing developments, Dr Peer outlines CRISPR and CAR-T therapies as forms of gene editing that can dramatically change the face of gene therapies and their use within modern medicine. He believes that as the commercialisation of these treatments increases, the regularity of their use will as well.

This is due to the surge in ‘positive public opinion’ that will arise from the successful implementation of the therapies. Dr Peer identifies the positivity that already exists around the new therapies, with ‘everyone looking at when these therapies will come to market and how much they will cost’. Although not going into detail about the financial outcome, he outlines his hope that when the public can see the lives of adults and children enhanced by using these therapies, any uncertain opinions will change to ones of optimism and confidence.

Peer acknowledges the lack of information that can exist around the new therapies due to an unfamiliarity in their usage; ‘People can sometimes get ahead of themselves and not understand completely what we’re trying to do here in this industry’. However, he is sure that this will change once there is evidence of patients who are successfully treated.

There is clearly excitement developing around the use of gene therapies, outlined by Dr Peer.

Watch the full interview, filmed at Cell Therapy Manufacturing & Gene Therapy Congress, with Dr Peer above or here.

Retrieving Memories with CRISPR

While we still can’t retrieve those life milestones or momentous occasions, like wedding memories – MIT biological engineers have devised a way to record complex histories in the DNA of human cells, allowing them to retrieve “memories” of past events, such as inflammation, by sequencing the DNA.

This analog memory storage system — the first that can record the duration and/or intensity of events in human cells — could also help scientists study how cells differentiate into various tissues during embryonic development, how cells experience environmental conditions, and how they undergo genetic changes that lead to disease.

This technology should offer insights into how gene regulation and other events within cells contribute to disease and development.

Analog memory
Many scientists have devised ways to record digital information in living cells. Using enzymes called recombinases, they program cells to flip sections of their DNA when a particular event occurs, such as exposure to a particular chemical. However, that method reveals only whether the event occurred, not how much exposure there was or how long it lasted.

Researchers have previously devised ways to record that kind of analog information in bacteria, but until now, no one has achieved it in human cells.

The new MIT approach is based on the genome-editing system known as CRISPR, which consists of a DNA-cutting enzyme called Cas9 and a short RNA strand that guides the enzyme to a specific area of the genome, directing Cas9 where to make its cut.  Continue reading “Retrieving Memories with CRISPR”

The New Age of CRISPR

The federal biosafety and ethics panel has unanimously approved the first study in patients of the genome-editing technology CRISPR-Cas9, in an experiment that would use CRISPR to create genetically altered immune cells to attack three kinds of cancer.

It had been widely expected that the first human use of CRISPR would be a 2017 clinical trial by Editas Medicine, which announced last year that it plans to use CRISPR to try to treat a rare form of blindness called Leber congenital amaurosis. Only a few hundred people in the US have that disease. The possibility that a study siccing CRISPR on cancer will happen first suggests that the revolutionary genome-editing technology might be used against common diseases sooner than once thought.

The experiment, proposed by scientists at the University of Pennsylvania, still needs the approval of the medical centers where it would be conducted, as well as from the Food and Drug Administration, which oversees the use of experimental treatments in people. If the study gets those OKs, it would enroll patients with multiple myeloma, melanoma, and sarcoma, and be funded by the Parker Institute for Cancer Immunotherapy, which was launched this year by tech mogul Sean Parker.

Continue reading “The New Age of CRISPR”

Francis Crick Institute Scientists Granted License to Edit Genes in Human Embryos – What will the Initial Impacts be?

In a global first, the UK’s Human Fertility and Embryology Authority (HFEA) have granted permission to a group of London-based scientist to edit the genomes of human embryos for research. Announced on 1st February 2016, the endorsement is the first of its kind by any national regulatory authority.

The approved research application from the Francis Crick Institute led by Dr Kathy Niakan is aimed at identifying and understanding the gene human embryos need to develop successfully. The research team will look at the first seven days of a fertilised egg’s development in the hope of providing important knowledge for understanding how a healthy human embryo develops.

Dr Niakan will be using CRISPR-Cas9 technology which enables the scientists to modify, identify, or replace genetic defects.

Commenting on the news, Paul Nurse, Director of the Crick, explained: “I am delighted that the HFEA has approved Dr Niakan’s application. Dr Niakan’s proposed research is important for understanding how a healthy human embryo develops and will enhance our understanding of IVF success rates, by looking at the very earliest stage of human development.”

So what will be the initial impact of this authorisation?

Controversy

With a topic such as genetically modified human genomes, controversy will not be far behind, with those opposed to the authorisation believing that this will open the door to ‘GM babies’.  It must though be made clear that this authorisation specifically states that it will be illegal for the scientists to implant the modified embryos into women.

Speaking on the ethical implications of the decision, Dr Sarah Chan from the University of Edinburgh said:

“The use of genome editing technologies in embryo research touches on some sensitive issues, therefore it is appropriate that this research and its ethical implications have been carefully considered by the HFEA before being given approval to proceed.”

“We should feel confident that our regulatory system in this area is functioning well to keep science aligned with social interests.”

IVF

Moving away from the controversy, it is good news for those receiving IVF treatment. With the increased knowledge that will be gained from the research, IVF success rates should dramatically improve thanks to the focus on miscarriage and fertility. Speaking to the BBC, Dr Kathy Niakan has said that the reason why she applied for the approval to edit human embryos was that “we would really like to understand the gene needed for a human embryo to develop successfully into a healthy baby”.

Dr Niakan went further to explain that “the reason why it is so important is because miscarriages and infertility are extremely common, but they’re not very well understood”.

Ethical Approval

The research is still in need of ethical approval before the programme can begin, but it is looking likely that this will be granted and the research will begin in the next few months.

With the research likely to add to the basic scientific knowledge needed for assisting infertile couples becoming parents, and to reduce the heartbreak of miscarriage, this is but one practical positive that will hopefully come from the work of Dr Niakan and her team.

Not only does the research have the potential to greatly assist in IVF treatment, it is another display of the excellent position the UK holds in the genome editing field of research and the advances being made by UK scientists.

At Cell Therapy Manufacturing & Gene Therapy Congress on 2nd – 5th February in Brussels, industry leaders will discuss technical and commercial strategies to deliver gene therapy products to patients. See the full agenda and book tickets at https://celltherapy.knnlab.com/.