▎ WuXi AppTec content team editor
Recently, industry media Centers Biotech and Fiere Pharma jointly launched the list of the most influential people in the biomedical field in 2022. After listing five CEOs of biotechs and large pharmaceutical companies last week, today's list presents five scientists who have made significant contributions to scientific progress in the biomedical industry over the past few years. Fierce Pharma's article states that "without science, biomedicine would not exist". In today's article, WuXi AppTec's content team will introduce the 5 scientists on the list.
Image source: References[1]
Professor George Church
Image source: Harvard University official website
Fierce Pharma's article states that Dr. George Church is a thinker, a science trailblazer, a serial entrepreneur, a status quo disruptor... He once said, "The best way to predict the future is to change it!" ”
Professor Church has been working at Harvard University for 45 years, starting as a doctoral student and now a professor of genetics. In his lab, Professor Church is a sage-like being like Master Yoda in Star Wars, and his students are eager to change the world through science. "I like that the average age in the lab is relatively small because they allow me to chase my dreams." He once said in an interview: "They don't think something is impossible yet. ”
Many of the distinguished students in his lab have pioneered biotechnology companies that seek to translate scientific breakthroughs into therapies that benefit humanity. "It's not enough to just write the patent and vote it out, you need to keep track of it." Professor Church said in a 2016 interview, "I spend one-tenth of my time making sure that the technology I develop is treated as it deserves in the market." ”
In the last 20 years, he has founded about 30 companies, and in the past 40 years, he has obtained nearly 100 patents. The 67-year-old hasn't slowed down, filing 20 patent applications last year.
Professor Church's involvement is extensive, and his ambitions and imagination can be glimpsed in the companies he founded or co-founded.
The company he co-founded, eGenesis, is committed to using CRISPR gene editing technology to modify the genome of pigs to generate organs, tissues and cells that can be transplanted into the human body, solving the problem of the shortage of transplantable organs.
Image credit: 123RF
Preclinical research by Rejuvenate Bio, co-founded by Professor Church, shows that through gene regulation, it is possible to reverse a variety of age-related diseases, including obesity, heart disease and diabetes. The company's therapies using adeno-associated virus delivery are expected to enter clinical development next year.
Founded just two years ago, Nabla Bio aims to design new antibodies using an innovative technology platform driven by artificial intelligence.
Shape Therapeutics completed a $112 million Series B round last year and reached a $3 billion research and development partnership with Roche. The company's RNA editing technology platform promises to develop next-generation mRNA therapies to prevent and treat multiple complex diseases, including Parkinson's disease and Alzheimer's disease.
Dr. Raymond Deshaies
Image source: Amgen official website
Dr. Raymond Deshaies is now Senior Vice President of Global Research at Amgen, where he previously made outstanding contributions to the field of targeted protein degradation during his 23-year scientific career at CalTech. In 2001, together with a team of Professor Craig Crews at Yale University, he published a landmark paper. The researchers designed an artificial molecule with small molecules capable of binding to the protein of interest at one end and polypeptides that bind to the E3 ligase at the other. The study found that this artificial molecule can "pull" the target protein near the E3 ligase, using a ubiquitin-mediated protein degradation system for degradation.
"In the future, this method is expected to be used to inactivate proteins under conditions and degrade disease-causing proteins." The abstract of the paper reads. This molecule is also named protein-targeting chimeric molecule 1, abbreviated as Protac-1.
Dr. Deshaies co-founded the startup Proteolix with Professor Crews. The company's proteasome inhibitor, Kyprolis, was later approved by the FDA for the treatment of multiple myeloma.
At Amgen, Dr. Deshaies is building the Induced Proximity Platform through a series of R&D collaborations and acquisitions. Amgen acquired Nuevolution for rapid compound screening using its DNA-Coded Compound Library (DEL). Dr. Deshaies' goals are not limited to targeting degrading proteins, he is also working on the development of small molecule compounds that target degrading RNA. These small molecules, called RIBOTEC, work just like PROTACs, except that they introduce RNA, rather than proteins, into the proteasome for degradation.
To achieve this goal, Amgen partnered earlier this year with Arrakis Therapeutics, a company dedicated to developing RNA-targeted therapies, to discover small molecule compounds that bind to 5 RNA targets.
Amgen has identified lead compounds that bind to RNA-degrading enzymes, and by linking these compounds to compounds found by Arrakis that bind to RNA molecules, it is possible to generate small molecule drugs that target degrading RNA.
Professor Jennifer Doudna
图片来源：The Royal Society, CC BY-SA 3.0, via Wikimedia Commons
Professor Jennifer Doudna and Professor Emmanuelle Charpentier received the Nobel Prize in Chemistry in 2020 for their outstanding contributions to CRISPR gene editing research.
In recent years, Professor Doudna has been working to advance translational research into CRISPR gene editing. Intellia Therapeutics, a biotech company she co-founded in collaboration with Regeneration, released the first clinical data supporting the safety and efficacy of CRISPR gene editing in vivo last year. This is an important milestone in the clinical application of CRISPR gene editing.
Another company she co-founded, Mammoth Biosciences, has developed a sensitive COVID-19 test based on CRISPR technology during the COVID-19 pandemic, which has been authorized for emergency use by the U.S. FDA. The company, which completed nearly $200 million in funding last year, will leverage the new Cas enzyme to develop multiple treatments and diagnostics based on CRISPR.
Outside of the medical field, Professor Doudna believes that gene editing technology has very exciting prospects in agriculture.
In addition to conducting groundbreaking research, Professor Doudna is also committed to helping young scientists chase their dreams. In 2014, she founded the Innovative Genomics Institute (IGI). It focuses on genome editing and helps translate basic research into real-world applications.
One of IGI's strategies is to match researchers and business partners, and the other is to support young entrepreneurs. IGI partners with the Tory Burch Foundation to support female biotechnology founders and has its own women entrepreneurs program to support future entrepreneurs who "come from an academic background and seek to further advance innovation and start a company," Professor Doudna said.
"In biology, we now have a very important opportunity to find ways to better connect academia to transformational opportunities." Professor Doudna said, "The challenge we faced was how to identify projects that could move forward to the next phase. These projects are sometimes not obvious, but if you succeed, the results are amazing! ”
Dr. Arthur Levin
Image credit: Avidity Biosciences
Dr. Arthur Levin is a pioneer in RNA therapy and has played a key role in the development of several oligonucleotide therapies, including the first approved antisense oligonucleotide drug and the first in-research therapy to target microRNAs in clinical trials. Over the past 30 years, he has made significant contributions to the research and development efforts of Roche, Ionis Pharmaceuticals, Santaris Pharma and miRagen Therapeutics.
He is currently the Chief Scientific Officer of Avidity Biosciences, inc., which is working on developing innovative therapeutic models called antibody oligonucleotide-coupled drugs. This technique combines the specificity of monoclonal antibodies with the accuracy of oligonucleotide therapy to precisely interfere with gene expression in target tissues.
Image source: Avidity Biosciences website
This technology platform has generated r&D pipelines for the treatment of a variety of serious muscle diseases, with the main therapy for the treatment of type 1 ankysmic dystrophy (DM1) already in Phase 1 clinical development.
Going forward, Dr. Levin expects that this technology platform can be used to develop innovative therapies that target immune cells and heart tissue, further expanding the application of RNA-targeted therapies.
Dr. Kimberly Smith
Image source: ViiV Healthcare official website
Dr. Kimberly Smith is the Head of Research and Development at Viv Healthcare. Cabenuva (cabotevir and repivirine, injectable formulations) and Apretude (cabotevir extended-release injections) developed by the company were approved by the FDA early last year and late last year, respectively, becoming the first long-acting HIV injection therapy and pre-exposure prophylaxis injection therapy. These two therapies are unique in that they only need to be injected once every two months, which facilitates the control and prevention of HIV infection.
Prior to joining ViiV Healthcare, Dr. Smith had been in the field of HIV for more than 20 years, bringing a patient-centric perspective to new drug development as a physician and researcher. This is especially important in the fight against HIV. "We are developing long-acting therapies that potentially require only one injection a year, which is the direction of research and development driven by Dr. Smith." Dr. John Pottage, the former head of research and development at ViiV, said, "This is because she has experience caring for patients and understands their needs. She was telling the whole company that this was the direction and goal we were heading for. ”
Dr. Smith had a hunch when he was in medical school that he would devote his life to HIV infection. After graduating, she worked at Rush University Medical Center, where she published hundreds of papers in the field of HIV. Dr. Pottage believes she can provide a key ingredient that is often missing for drug development. "In our industry, there are a lot of scientists, and there are a lot of business-oriented people who want to do good, but they don't have a deep understanding of patient care." Dr Pottage said, "That empathy with patients is what Dr. Smith brings to the company, and to the field as a whole." ”
Following the approval of Cabenuva and Apedude, Dr. Smith said ThatViV's near-term research and development goals are to expand the drug interval to 3 months and develop products that allow patients to self-administer. Her years of experience as a doctor have taught her that this is something patients need.
"I think as an industry, we need to improve the lives of patients the way they want to. That means listening to their voices. "In addition to treating, researching HIV, developing HIV drugs, I think I'm also a patient advocate." The energy of these different identities has helped me lead our R&D organization to success. ”
Title Image Source: References[1]
Resources:
[1] Most influential people in biopharma—the scientists. Retrieved May 9, 2022, from https://www.fiercepharma.com/pharma/most-influential-people-biopharma-scientists
[2] Avidity Biosciences. Retrieved May 9, 2022, from https://www.aviditybiosciences.com/about/management/
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