Washington, DC - Nov. 6, 2019

The American Society of Gene and Cell Therapy held a 3 day summit to discuss policy and other issues around gene editing and gene therapy.

  • Day 1 addressed regulatory issues in gene therapy development
  • Day 2 dealt with payment policies around gene therapy
  • Day 3 (the day I attended) was focused on the ethical, societal, and policy issues around germline gene editing

Links to some of the presentations can be found on ASGCT’s website.

Gene editing is the process of altering a cell’s DNA. This is usually done to fix a mutation or give cells a particular trait. Currently, the most popular way to edit cells uses CRISPR. This policy summit focused on heritable gene editing, where scientists make edits which can be passed on to future generations. With the recent discovery and popularity of CRISPR this summit is fairly timely. This summit became quite necessary after a scientist edited the embryos of 2 twins, and brought those children to term last October. In this procedure the scientist used in-vitro fertilization to create embryos, then used CRISPR-Cas9 to edit the CRC5 gene. This edit is intended to make the children resistant to HIV. News of this procedure was met with fairly widespread criticism, although it was not surprising to many in the field. A very interesting article about the state of the field at the time was recently published in Science magazine.

These are my main takeaways from the summit:

Germline gene editing is unneccesary

Francis Collins, the director of the NIH, was the first person to bring this up, but he was far from the last. In the case of many inherited diseases (like choroideremia), there is no need to do any editing of embryos. Instead, during the in-virto fertilization process, clinicians can use pre-implantation gentic diagnosis to select embryos that do not have the disease causing mutation and implant those.

Determining global policy is difficult

Who determines global policies, and how do those get enforced? On this topic the World Health Organization has taken the lead on developing guidelines for when gene editing should be used. Dr. Margaret Hambur serves as a chair on the WHO expert advisory committee on governance of human genome editing. At this summit she gave an update on their progress and described the process they are going through. As you can imagine, a large global beurocracy making important and difficult decisions about humanity and technology will move fairly slowly, but they are making progress. Some at the conference, including Dr. Collins, gently criticized the organization for getting a little bit off topic and working on non-germline gene editing.

What does a global moratorium on germline gene editing even mean? Individual countries will need to create legislature in order to enforce the WHO’s guidelines. But the WHO’s decision also carries some weight as individual scientists and institutions will be less likely to go against the scientific consensus.

Reproductive rights are complicated

A few ethicists brought up some thought provoking points about reproductive rights and gene editing. Imagine someone with cancer. If a controversial (but effective) therapy exists, they have 2 options:

  • Receive the treatment and get cured
  • Don’t recieve the treatment, and continue to suffer

Germline gene editing is not the same as a cancer treatment. There are more options at play:

  • Use gene editing to have a healthy genetically related child
  • Have a child with a disease
  • Have a child by other means
    • Adoption, PGD, surrogate parent

Having a genetically related child without disease is not the same as curing a disease. It is the parents' wish, not a human right. This is an interesting idea that I had not considered before.

Update (2/23/2020)

He Jiankui

Dr. He Jiankui is the scientist who conducted the experiment (treatment?) on the twins. Some people asserted that Dr. He did not believe he was out of line from what the scientific community believed to be moral. He had discussions with a variety of people (see the article I linked above) and has claimed he thought he was doing something only slightly controversial (like the first IVF babies.) Dr. Ben Hurlbut brought up an interesting point: much of the reporting on this story has racist undertones. The fact that Dr. He is a Chinese scientist is front and center of the reporting.

I am not defending Dr. He, he is definitely at fault. The ethics approval certificate was forged and the consent given by the couple was not well informed.

Dr. He has been sentenced to 3 years in prison for carrying out “illegal medical practices.”