▎ WuXi AppTec content team editor
Today, Endpoints News released The 20 under 40 in Biopharma, a total of 21 young scientists and entrepreneurs. The Endpoints article notes that they are chasing lofty ideals that promise to help reshape the pharmaceutical field. In today's article, WuXi AppTec's content team will introduce the 21 young talents on the list.
Omar Abudayyeh, Jonathan Gootenberg (MIT Fellow, Co-Founder of Sherlock Biosciences)
Dr. Omar Abudayyeh and Jonathan Gootenberg, who studied under CRISPR gene editing pioneer Professor Feng Zhang, discovered a new Cas enzyme called Cas13 based on data from NIH partners. Unlike Cas9, which is commonly used in DNA gene editing, it targets RNA and, after recognizing the RNA sequence, shreds all the RNA around it.
Cas13 became the basis for innovative molecular detection methods. Most viral genomes are made up of RNA, and when Cas13, which targets a viral sequence, binds to a specific sequence in the viral genome, it activates and cuts off the reporter RNA, releasing a fluorescent signal.
Building on this molecular testing, Sherlock Biosciences was born, which has raised nearly $130 million to develop molecular tests for cancer and infectious diseases.
Theonie Anastassiadis (Co-Founder and Chief Innovation Officer, Alltrna)
Dr. Theonie Anastassiadis came across tRNA biology while working at the investment firm Flagship. tRNA is an important component of the protein synthesis mechanism in cells, which synthesizes polypeptide chains by recognizing the code on the mRNA and transporting the corresponding amino acids into the ribosome. Many rare diseases arise because of a miscoding error on the mRNA that gives the protein synthesis the wrong termination signal. One strategy for treating these diseases is to use mRNA or gene therapy to replace the wrong code.
Another strategy is to replace the tRNA, allowing the tRNA to recognize the prematurely occurring stop codon and add the correct amino acids. Its advantage is that since many different diseases are due to the same type of codon mutation, altering tRNA promises to treat multiple diseases with one drug.
Dr. Theonie Anastassiadis co-created Alltrna within Flagship dedicated to developing a platform for tRNA technology. Last November, the company announced the completion of a $50 million Series A funding round. Dr. Anastassiadis said the company's main goal is to engineer tRNA to correct senseless mutations that lead to premature termination of protein synthesis, but the company's vision is to use different tRNAs to alter the translation results of mRNA and modify biological processes that are dysregulated in cancer and other diseases.
Sam Bakhoum (Co-Founder of Volastra)
Dr. Sam Bakhoum, who has his own lab at Memorial Sloan Kettering Cancer Center, has found that chromosomal instability (CIN) in tumor cells is an important factor in driving cancer metastasis. CIN is due to tumor cells making errors when performing mitosis, causing chromosomal fragments to drift outside the nucleus. This DNA located in the cytoplasm will not only promote cancer metastasis by stimulating the NF-κB signaling pathway, but also stimulate tumor cells to release cytokines with immunosuppressive ability, preventing tumor cells from being discovered by the body's immune system, thereby further promoting the metastasis of tumor cells.
Based on this finding, Dr. Bakhoum co-founded Volastra, a company dedicated to developing ways to stop cancer cell metastasis by targeting chromosomal instabilities. The company has partnered with Microsoft and Bristol-Myers Squibb.
Jake Becraft (Co-Founder and CEO of Strand Therapeutics)
Dr. Jake Becraft is a synthetic biologist who, along with Dr. Tasuku Kitada of the Massachusetts Institute of Technology (MIT), co-founded Strand Therapeutics. While working at MIT's Center for Synthetic Biology, he led the development of a synthetic biology language for mRNA programming.
Strand Therapeutics' goal is to use synthetic biology to engineer mRNA to produce programmable, long-acting mRNA therapies. Not only do they self-expand, but they are also delivered precisely into cells, providing the potential to treat multiple diseases with a single dose of therapy. The company reached an R&D partnership with BeiGene last year to jointly develop immunotherapies to treat solid tumors.
Seemay Chou (Co-Founder and CEO of Arcadia Science)
Dr. Seemay Chou was a professor in the Department of Biochemistry and Biophysics at the University of California, San Francisco. Her lab studies the mechanical forces that drive interactions between animals and microbes. In her research, she used a model organism not commonly found in biological studies— ticks. In her view, scientific research using non-traditional model organisms could open up new avenues for innovative scientific discoveries. Ticks, for example, have evolved compounds that paralyze human skin perception so that they go undetected when they bite the skin. Its saliva could be a potential treasure trove of skin-related research and therapy development.
The goal of Arcadia Science, co-founded by Dr. Chou, is to drive researchers to conduct open-ended, curiosity-driven research, with a particular focus on the study of species that are less studied. The company believes that nature may have found answers to many medical problems through millions of years of evolutionary processes, and scientists may discover biological innovations and develop new technologies and products by studying a wider range of species.
Abasi Ene-Obong (Founder and CEO of 54Gene)
Today, although scientists have completed the sequencing of the human genome and constructed a complete genome map. However, due to the bias of sampling when sequencing, the diversity of the world's population in genome sequences is not fully represented. African populations, for example, have the highest genomic diversity in the world. However, in genomic studies, the genome sequences of african populations accounted for less than 3% of the study data.
Dr. Abasi Ene-Obong is the co-founder and CEO of 54Gene. The company's goal is to increase the use of genomic data from African populations in genomic research. The company expects to have access to more than 100,000 samples by the end of the year, generating large amounts of genomic data from African populations. These data will not only increase the diversity of genomic sequences, but may also provide clues to the development of innovative therapies.
Nicholas Flytzanis (Co-Founder and Chief Scientific Officer, Capsida)
Nick Goeden (Capsida Co-Founder and CTO)
Adeno-associated virus (AVV) is one of the important vectors for delivery gene therapy, and although gene therapy using AAV delivery has been approved by the FDA and widely used in clinical trials, it still has a series of limitations. These include limited payloads, inability to efficiently deliver GMos to specific tissues, and potential toxicity. The goal of Capsida Biotherapeutics, co-founded by Dr. Nicholas Flytzanis and Dr. Nick Goeden, is to use its unique AAV engineered platform to generate viral capsids that target specific tissues or cells associated with disease organs, while limiting the transduction of gene therapy in unrelated tissues, thereby improving the efficacy and safety of gene therapy. It completed a $140 million Series A funding round last year and entered into a strategic partnership with CRISPR Therapeutics to develop in vivo gene-editing therapies delivered using engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedrich ataxia.
Isaac Kinde (Co-Founder of Thrive)
Dr. Isaac Kinde studied for his Ph.D. at Johns Hopkins University under the tutelage of Bert Vogelstein, a pioneer in cancer research. The liquid biopsy he helped develop analyze genomic mutations in circulating tumor DNA (ctDNA) and cancer-associated protein markers in plasma to find common features in many different cancer types. Thrive Earlier Detection, which was born on this test, raised $110 million in Series A funding in 2019 to develop a liquid biopsy that can detect multiple cancer types at an early stage of cancer. A 2020 study published in the journal Science showed that the company's CancerSEEK test, added to routine cancer screening, was able to allow doctors to detect 52 percent of cancers, nearly twice as much as routine screening (26.1 percent).
In 2020, Exact Sciences announced the acquisition of Thrive. Dr. Kinde is currently at Exact Sciences evaluating emerging technologies that make liquid biopsy easier and faster, and expand accessibility.
Najat Khan (Chief Data Science Officer, Johnson & Johnson Drug Discovery)
While pursuing his Ph.D. in organic chemistry at the University of Pennsylvania, Dr. Najat Khan also took a computer science course to learn how to use data to predict which molecules bind more strongly to specific receptors on the surface of cancer cells. While the use of AI for molecular design and optimization is already being used in many biotech and pharmaceutical companies today, it seemed like an unpopular decision more than a decade ago.
Eventually, Dr. Najat Khan's research project blossomed into a way that developed algorithms that continually learned and iterated from failures to continuously optimize candidate molecules. The success of this project became the starting point for her career, and she was eventually hired by Johnson & Johnson as chief data science officer in the pharmaceutical research and development department, leading about 150 scientists and engineers to integrate machine learning and other data science tools into the drug discovery process.
Isaac Klein (Chief Scientific Officer, Dewpoint Therapeutics)
Since 2017, Dr. Isaac Klein has been working on biomolecular condensates at the Whitehead Institute. In cells, in addition to the well-known mitochondria, endoplasmic reticulum and other organelles with cell membranes, there are also a variety of organelles without cell membranes, these membrane-free organelles are formed by liquid-liquid phase separation (LLPS), which are widely present in the cell, which can gather proteins, RNA and other macromolecules together, and isolate unrelated molecules, accelerating the occurrence of chemical reactions. These membraneless organelles are also known as biomolecular condensates.
Dr. Klein's addition of Newpoint Therapeutics aims to discover disease-associated aggregate biology and then develop small molecule drugs that target these pathways. The company has reached cooperation with pharmaceutical companies such as Merck, Pfizer and Bayer. Dr Klein said biomolecular aggregates could offer a "whole new world and a whole new way" for cancer drug development.
Tim Knotnernus (CEO of Agomab)
Mr. Tim Knotnernus previously worked for venture capital firm Aescap Venture, where he was responsible for investing in European medical companies. However, he prefers to take a more active role in drug development. That led him to become CEO of Agomab in 2019. The company is working on developing agomAbs, an agonist antibody drug that mimics hepatocyte growth factor (HGF).
Hepatocyte growth factor (HGF) is a polypeptide growth factor with a variety of biological activities produced by various organs in the human body, HGF plays a vital role in cell growth and differentiation, angiogenesis, embryonic development, wound healing and repair through paracrine or autocrine mechanisms. More than three decades of research have shown that HGF has shown effective healing potential in preclinical models of organ fibrosis, inflammation, autoimmune diseases, and degenerative diseases.
AgomAb's agonistic monoclonal antibody agumbAbs against the MET receptor can bind to MET with high affinity, promote receptor dimerization and activation, and achieve the molecular effect of replacing HGF. AgomAbs not only retains the potential for HGF treatment, but is also more stable, less toxic and safer.
Recently, the company acquired the Spanish biotech company Origo, further expanding the company's R&D pipeline.
Claire Mazumdar (CEO of Bicara)
Dr. Claire Mazumdar received her Ph.D. in cancer biology from Stanford University, then worked for Editas Medicine and then venture capital firm Third Rock. Early on, she decided to devote herself to the work of translating science into therapies, rather than working in academia like her parents.
In 2020, she became CEO of Beacon Therapeutics. The company's flagship research therapy, BCA101, is a bispecific antibody. One end of it can bind to EGFR receptors on the surface of cancer cells, and at the other end is the TGF-β trap. TGF-β plays an important role in building an immunosuppressive tumor microenvironment, and by capturing TGF-β, this therapy may alter the tumor microenvironment and help enhance the anti-cancer activity of immune cells. It is currently being tested in a Phase 1b clinical trial and is expected to have results by the end of this year.
Dina Radenkovic (CEO of Gameto)
Women have about 2 million eggs at birth, but after the age of 35, the number of eggs decreases dramatically, and by the age of 45, most women are almost unable to conceive. Although people's life expectancy is constantly increasing, women's reproductive ability has not changed, one of the main reasons is that the ovaries age 5 times faster than the rest of the body!
This is the problem that Gameto, founded by Ms. Dina Radenkovic, aims to solve. The company's goal is to synchronize the pace of ovarian aging with the rate of aging of other organs by developing an ovarian treatment platform. Some initial studies conducted in professor George Church's lab at Harvard Medical School have shown that Gameto made breakthroughs in solving the problem of accelerating ovarian aging, generating the first human ovarian reprogramming cells (granulocytes and oocyte precursors). The platform will initially be used to improve the assisted reproductive process and hopefully eventually develop cell therapies that address the medical burden of menopausal women.
Aaron Ring (Professor at Yale University and Founder of Simcha)
Yale Professor Dr. Aaron Ring represents an emerging force in the field of translational research. Since 2015, he has helped launch several biotech companies, including ALX Oncology, Ab Initio Biotherapeutics. Most recently, a company was Simcha Therapeutics. Research in Professor Ring's lab has shown that protein-engineered cytokine IL-18 has a strong anti-tumor effect in animal models. The study was published in the journal Nature in 2020. Simcha Therapeutics, which was created based on this research, completed a $40 million Series B round earlier this year, and its main potential "first-in-class" IL-18 variant is currently in Phase 1/2 clinical trials.
Lex Rovner (Co-Founder and CEO of 64x Bio)
Dr. Lex Rovner, while a postdoc in The George Church lab at Harvard University, co-founded synthetic biology company 64x Bio based on the lab's technology. The company's goal is to use high-throughput screening to discover cell lines that can dramatically increase the productivity of viral vectors. Its unique genetic barcode system links information on viral vector productivity to the cell lines that produce the vector, allowing for simultaneous screening of millions of candidate cell lines. The company completed a $55 million Series A funding round this year, and Dr. Rovner said the technology could revolutionize the economics of gene therapy and expand the patient base reached by this treatment model.
Nabiha Saklayen (Co-Founder and CEO of Cellino)
Dr. Saklayen is a Ph.D. in physics graduate from Harvard University, and during her studies, she began to dabble in the life sciences. In 2017, she co-founded Cellino Biotech with two other partners. The company's technology platform combines labelless imaging, high-speed laser editing, and intelligent algorithm (AI) technologies to automate cell reprogramming, amplification, and differentiation. This breakthrough approach benefits more patients by being able to process thousands of patient samples in parallel in a single facility. It completed a $80 million Series A funding round in January.
Dr. Saklayen has also been featured on Forbes' "30under30" list and MIT Tech Review's 35 Under 35StechNologists list.
Sharif Tabebordbar (Co-Founder and Chief Scientific Officer, Kate Therapeutics)
Dr. Sharif Tabebordbar's father gradually lost the ability to walk due to a muscular dystrophy disease, which led him to develop therapies to treat muscular dystrophy. Gene therapy has shown good promise in treating such diseases, but how to make the viral vector that delivers gene therapy target muscles, rather than being swallowed up by the liver, is a problem that scientists need to solve.
Dr. Tabebordbar's strategy is to generate millions of different variants of the virus and then inject them into mice or monkeys. By sequencing animal tissue, he could determine which variant of the virus was able to transfect muscles more efficiently. His findings have spawned a biotech company called Kate Therapeutics. He hopes the study will lead to safer and more effective treatments.
Pamela Ting (Novartis, scientist)
Dr. Pamela Ting is a scientist at the Novartis Institute for Biomedical Research. The work she leads aims to develop a small molecule drug to treat sickle cell anemia. Sickle cell anemia affects thousands of patients in Africa, and while gene therapies for the disease are expected to be approved to bring potentially curative therapies to patients, their development and manufacture require significant resources and may only be used to treat the most severe patients. A small molecule therapy is expected to benefit a wide range of patients.
So far, Dr. Ting's team has discovered small molecules that can activate fetal hemoglobin. Fetal hemoglobin is no longer expressed after birth, and if drugs can be used to re-express them, it may achieve the same effect as gene therapy.
Vasanthi Viswanathan (Co-Founder and Head of Discovery Biology, Kojin)
Dr. Viswanathan conducts cutting-edge research into how cancer cells evade drug treatment through reprogramming at Columbia and Harvard. Together with her mentor, Dr. Stuart Schreiber of Harvard University, she co-founded Kojin. The platform developed by Kojin focuses on measuring the state a cell is in and developing targeted therapies based on the specific state the cell is in. The cells in which lesions occur function and behave differently than healthy cells, so by understanding the state of the cells it is possible to distinguish them from healthy cells.
The company's initial target of a cell state is an iron death-sensitive state, and cells in this state are prone to iron death. Iron death is a form of cell death, and the chain reaction caused by lipid peroxidation can destroy cell membranes like rapidly expanding wildfires. By targeting iron death-sensitive states, Kojin is able to selectively cause iron death in cells while protecting beneficial cells. Cells that cause fibrosis and immune diseases, as well as cancer cells that develop drug resistance, are often susceptible to iron death. The company completed a $60 million Series A funding round last year.