Roundup: Monthly Biotech Updates, November 2018

The month of November has been a very busy and exciting one, with surprising announcements and some setbacks for all the Biotech enthusiasts. Here is a collection of the top biotech news for the month of November – Roundup: Monthly Biotech Updates for November 2018.

Gene Editing_CRISPR


Big News

Chinese CRISPR Scientist Claimed To Have Created World’s First Genetically Modified Babies

A Chinese scientist gave a surprise announcement on the eve of an international human gene-editing summit. He Jiankui, from a university in Shenzhen has told the Associated Press that he has succeeded in helping create the world’s first genetically-edited babies. As per the claims of the Chinese researcher, twin girls were born earlier this month. Dr. Jiankui said that he edited embryos for seven couples during fertility treatments using CRISPR technology to remove the CCR5 gene and resulting in one pregnancy until now. This plays a critical role in enabling many forms of the HIV virus to infect cells.

He said the gene editing occurred during IVF or lab-dish fertilization. First, sperm was “washed” to separate it from semen, the fluid where HIV can lurk. A single sperm was placed into a single egg to create an embryo. Then the gene editing tool was used. When the embryos were 3 to 5 days old, a few cells were removed and checked for editing. Couples could choose whether to use edited or unedited embryos for pregnancy attempts. Eleven embryos were used in six attempts before the twin pregnancy was achieved, He said. Tests suggest that one twin had both copies of the intended gene-altered and the other twin had just one altered, with no evidence of harm to other genes, He said. People with one copy can still get HIV. Jiankui He had planned to discuss his work on November 28 in Hong Kong at the second International Summit on Human Genome Editing.

However, a lot of curiosity, comments and questions have been raised over the claims by the Chinese scientist on his dramatic CRISPR results. If the results are true, it would be a profound leap in science and ethics. This kind of gene editing is banned in most countries as the technology is still experimental and DNA changes can pass to future generations, potentially with unforeseen side-effects. Many mainstream scientists think it is too unsafe to try, and some denounced the Chinese report as human experimentation. That announcement sparked outrage from many researchers and ethicists who say implanting edited embryos to create babies is premature and exposes the children to unnecessary health risks. Opponents also fear the creation of “designer babies,” children edited to enhance their intelligence, athleticism or other traits. Read More

China Orders Halt to Research in Gene Editing

China’s science ministry on Thursday ordered that anyone conducting research in gene editing halt their activities. The order came as organizers of a biomedical conference where a Chinese scientist defended his claim that he has created the world’s first genetically-edited babies denounced his work as irresponsible. The leaders of the Second International Summit on Human Genome Editing issued a statement Thursday on the last day of their conference in Hong Kong criticizing He Jiankui’s claim as “deeply disturbing.”

Dr. He told his colleagues he conducted his research in secret. His work has not been independently verified, and Dr. He has not submitted his report to any scientific journals where it could be examined by experts. In their statement Thursday, the summit’s organizers said that even if “the modifications are verified, the procedure was irresponsible and failed to conform with international norms.” Dr. He was supposed to speak before the summit again Thursday but canceled his appearance.

He’s claims has set off a firestorm of skepticism and criticism. The Southern University of Science and Technology, the university in the southern Chinese city in Shenzhen that employs him, says he has been on unpaid leave since February. The school denounced his research for violating “academic ethics and codes of conduct,” and the Chinese government is urging local authorities to launch an investigation into He’s work.

Genetic editing has the potential to remove inherited diseases from the gene pool, but scientists and ethicists worry it could be used to create so-called “designer babies.” They also worry any genetic changes could lead to other genes being altered in unpredictable ways.

96% of People Found To Have Pre-Existing Immunity to CRISPR-Cas9 gene in Research

Although the first news of this research result was announced at the very end of October, it was a big set back for gene CRISPR companies that shook the entire biotech/CRISPR related industry for the weeks that followed. While CRISPR-Cas9 gene editing therapeutics was advancing towards their first-ever human tests. A newly published research from Charité – Universitätsmedizin Berlin totally overshadowed this advancement.

The study had shown that a key component of CRISPR-Cas9 gene editing could attack, and possibly even be disabled by the human immune systems. Even more critical is the fact that this might be true for the large majority of people. Publication of a separate study in January, with a similar conclusion, had precipitated a massive drop in stock prices of CRISPR companies, such as CRISPR Therapeutics (NASDAQ: CRSP), Editas Medicine (NASDAQ: EDIT) and Intellia Therapeutics (NASDAQ: NTLA). While the previous paper only disrupted the stock market, the newly published paper in Nature Medicine has researchers concerned about the looming human trials.

In January, a research team led by Matthew Porteus of the Stanford School of Medicine found that 65% of donors had antibodies against Cas9 from S. pyogenes, but couldn’t detect T cell activity against that enzyme. Previous research from other teams has shown pre-existing immunity in lab animals. The published research from Charité – Universitätsmedizin Berlin has confirmed the results produced by The Porteus Lab. Blood samples from 48 healthy volunteers were exposed to Cas9 (a DNA-cutting enzyme) derived from a bacterium called Streptococcus pyogenes. Of the 48 volunteers, 41 (85%) samples had antibodies against Cas9 and 46 (96%) had T-cell based immunity. Read More

Second International Summit on Human Genome Editing at Hongkong

The Academy of Sciences of Hong Kong, the Royal Society of the United Kingdom, and the U.S. National Academy of Sciences and U.S. National Academy of Medicine organized the Second International Summit on Human Genome Editing in Hong Kong to assess the evolving scientific landscape, possible clinical applications, and attendant societal reactions to human genome editing. While we, the organizing committee of the second summit, applaud the rapid advance of somatic gene editing into clinical trials, we continue to believe that proceeding with any clinical use of germline editing remains irresponsible at this time.

Human Genome Editing Research – The organizing committee concludes that the scientific understanding and technical requirements for clinical practice remain too uncertain and the risks too great to permit clinical trials of germline editing at this time. Progress over the last three years and the discussions at the current summit, however, suggest that it is time to define a rigorous, responsible translational pathway toward such trials.

A Proposed Translational Pathway – A translational pathway to germline editing will require adhering to widely accepted standards for clinical research, including criteria articulated in genome editing guidance documents published in the last three years.

Report of Clinical Use of Germline Editing – About the claim that human embryos had been edited and implanted, resulting in a pregnancy and the birth of twins. The procedure was irresponsible and failed to conform to international norms. We recommend an independent assessment to verify this claim and to ascertain whether the claimed DNA modifications have occurred.

An Ongoing International Forum – The organizing committee calls for an ongoing international forum to foster broad public dialogue, develop strategies for increasing equitable access to meet the needs of underserved populations, speed the development of regulatory science, provide a clearinghouse for information about governance options, contribute to the development of common regulatory standards, and enhance coordination of research and clinical applications through an international registry of planned and ongoing experiments.

Moderna Sets Plans For Biggest IPO for VC-Backed Biotech Company in the USA

Moderna Therapeutics (MRNA) is planned to become the largest initial public offering of a venture capital-backed biotech company, setting IPO terms that could raise $521 million. With plans to offer 21.7 million shares at between $22 and $24 apiece on the NASDAQ under the ticker symbol MRNA. The public debut could raise up to $521.7 million, which would make it the largest IPO ever for a VC-backed biotech in the US, per PitchBook data. The company’s significant stockholders include Flagship Pioneering (19.5% pre-IPO stake) and AstraZeneca (8.4%). Morgan Stanley, Goldman Sachs, and JP Morgan are acting as the lead underwriters for the IPO. The Moderna IPO plans to offer 21.7 million shares priced in the range of $22 to $24, that gives it a market valuation near $8 billion.

Moderna is the most valuable VC-backed business in Massachusetts, per PitchBook data. It has raised around $1.6 billion in total venture funding, including a $125 million round in May from the VC arm of Merck that valued it at over $7 billion. That funding is part of a continued partnership between the companies, which are working together to develop a personalized cancer vaccine. Moderna’s other backers include Sequoia Capital China, ArrowMark Partners, and EDBI.

Founded in 2011, the company develops drugs for the treatment of infectious diseases, genetic disorders, cancer, and cardiovascular diseases, by using messenger RNA technology to convey genetic information within cells. Moderna says its mission is to create “a new generation of transformative medicines for patients,” according to the Moderna IPO prospectus. It has a pipeline of 21 development programs, of which 10 have entered clinical studies.

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