Category Archives: California Stem Cells

Redding, California, Aug. 01, 2022 (GLOBE NEWSWIRE) -- According to a new market research report, Cell Culture Media Market by Product (Serum-free, Chemically Defined, Classical, Xeno-free/Animal Component Free), Application (Monoclonal Antibody, Diagnostics, Cancer and Stem Cell Research), End User (Pharma & Biotech, Academic)- Forecast to 2029, published by Meticulous Research, the cell culture media market is expected to grow at a CAGR of 13.2% from 20222029 to reach $14.64 billion by 2029.

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Cell culture media, also known as growth media, isany gel or liquid created to support cellular growth in an artificial environment. When scientists remove cells, organs, or tissues from living creatures, they must keep them in an appropriate artificial environment. Cell culture media contains compounds required to support and regulate the growth of cells. These cells are used in the production of biopharmaceuticals or for research purposes. Growing demand for monoclonal bodies, rising funding for cell-based research, and increased R&D spending in the life science sector are some factors driving the cell culture media market.

COVID-19 Pandemic Impact: Cell Culture Media Market

During the pandemic, major pharmaceutical and biotech companies collaborated to find vaccines and treatment methods for containing the virus. The idea behind the collaboration was to come together and find a solution as quickly as possible during the pandemic. Collaborations also took place between companies and universities to speed up the research & production process. Outsourcing pharmaceutical and biotechnological research to contract research organizations (CROs) also increased during the pandemic. Below are some of the instances,

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The global cell culture media market is segmented based on product (serum-free media, chemically defined media, classical media, animal component-free media, and other cell culture media), application [bioproduction (monoclonal antibody production, therapeutic protein production, vaccine production, and cell & gene therapy)], diagnostics, cancer research, drug screening & development, and stem cell research), end user (pharmaceutical & biotechnology companies, academic & research institutes, and diagnostic laboratories) and geography. The study also evaluates industry competitors and analyzes the regional and country markets.

On the basis of product, in 2022, the serum-free media segment is estimated to account for the largest share of the cell culture media market. The major contributing factor for the large share of this segment is the higher use of cell culture techniques in companies and research institutes, which demand media to grow cells. Some of the major benefits offered by this media include reduced risk of serum borne agents and increase in cell culture productivity. Several serum-free media have been developed and are generally formulated to support the culture of a single cell type. The segment is also expected to grow at the highest CAGR during the forecast period.

On the basis of application, the bioproduction segment is expected to grow at the highest CAGR during the forecast period. The increased production of biotherapeutics for treating diseases like cancer, diabetes, rheumatoid arthritis, and other non-communicable diseases has created an unprecedented demand for cell culture media, which, in turn, is expected to drive the growth of this segment in the forecast period.

Based on the end user, the pharmaceutical & biotechnology companies segment is expected to grow at the highest CAGR during the forecast period. The major contributing factor is the increasing demand for antibody therapeutics and emerging biosimilars in the coming years.

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Geographically, North America is estimated to dominate the overall cell culture media market in 2022, followed by Europe and Asia-Pacific. Increased demand for cell-based diagnosis and treatments, the presence of major players, the availability of skilled professionals, and the presence of major research institutes are major contributing factors to the regional market's large share. However, Asia Pacific is expected to grow at the highest CAGR during the forecast period. The growth is attributed to the rising technological advancements in biotechnology & pharmaceutical centers, growing focus on developing vaccines & clinical research activities, the presence of major research organizations, low manufacturing & labor costs, and developing healthcare infrastructure.

The report also includes an extensive assessment of the product portfolio, geographic analysis, and key strategic developments adopted by leading market participants in the industry during 20192022. In recent years, the cell culture media market has witnessed several new product launches, partnerships, agreements and collaborations, expansions, and acquisitions. For instance, in January 2022, Cytiva (U.S.) signed an agreement with Nucleus Biologics, LLC (U.S.) to develop custom cell media for cell and gene therapies. Also, in November 2021, Sartorius AG (Germany) opened its Cell Culture Technology Center in Germany.

Some of the key players operating in this market are Merck KGaA (Germany), Thermo Fisher Scientific Inc. (U.S.), PromoCell GmbH (Germany), Cytiva (U.S.), Lonza Group Ltd (Switzerland), Corning Incorporated (U.S.), Becton, Dickinson and Company (U.S.), HiMedia Laboratories (India), Sartorius AG (Germany), FUJIFILM Holdings Corporation (Japan), Agilent Technologies, Inc. (U.S.), Miltenyi Biotec B.V. & Co. KG (Germany), BIOLOGOS (U.S.), STEMCELL Technologies Inc. (Canada), and Cell Applications, Inc. (U.S.).To gain more insights into the market with a detailed table of content and figures, click here:https://www.meticulousresearch.com/product/cell-culture-media-market-5318

Scope of the Report:

Cell Culture Media Market, by Product

Cell Culture Media Market, by Application

Cell Culture Media Market, by End User

Cell Culture Media Market, by Geography

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About Meticulous Research

Meticulous Research was founded in 2010 and incorporated as Meticulous Market Research Pvt. Ltd. in 2013 as a private limited company under the Companies Act, 1956. Since its incorporation, the company has become the leading provider of premium market intelligence in North America, Europe, Asia-Pacific, Latin America, and the Middle East & Africa.

The name of our company defines our services, strengths, and values. Since the inception, we have only thrived to research, analyze, and present the critical market data with great attention to details. With the meticulous primary and secondary research techniques, we have built strong capabilities in data collection, interpretation, and analysis of data including qualitative and quantitative research with the finest team of analysts. We design our meticulously analyzed intelligent and value-driven syndicate market research reports, custom studies, quick turnaround research, and consulting solutions to address business challenges of sustainable growth.

Contact:Mr.Khushal BombeMeticulous Market Research Inc.1267WillisSt,Ste200 Redding,California,96001, U.S.USA: +1-646-781-8004Europe : +44-203-868-8738APAC: +91 744-7780008Email-sales@meticulousresearch.comVisit Our Website:https://www.meticulousresearch.com/Connect with us on LinkedIn-https://www.linkedin.com/company/meticulous-researchContent Source: https://www.meticulousresearch.com/pressrelease/542/cell-culture-media-market-2029

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Cell Culture Media Market Worth $14.64 Billion by 2029 - Exclusive Report by Meticulous Research - GlobeNewswire

image:Haifan Lin assumes the role of ISSCR president. view more

Credit: Yale University School of Medicine, USA

TheISSCR is pleased to announce Haifan Lin, PhD, Yale University School of Medicine, USA, as its President. Dr. Lins one-year term of office leading the global Society begins today.

It is a great honor for me to assume the ISSCR presidency during the 20th anniversary year of the Societys founding, Dr. Lin said. In the past 20 years, the ISSCR has embodied our shared passion for stem cell research. The remarkable growth of the Society is driven by our members exciting research and discoveries, many of which are being translated into lifesaving treatments and new approaches in drug development for the benefit of humanity.

Dr. Lin urged, In a time challenged by the persisting pandemic as well as by polarizing geopolitical forces and a devastating war, there is more need and more urgency for ISSCR to serve as a haven for stem cell researchers from all over the world to work together synergistically for the benefit of all mankind, regardless of race, color, religion, gender, and national origin. He pledged, to work closely with members, the board, and the staff to actively expend our global engagement in both academia and industry during my presidency.

Haifan Lin is the Eugene Higgins Professor of Cell Biology and founding Director of Yale Stem Cell Center at Yale University School of Medicine, a Member of US National Academy of Sciences, a Member of American Academy of Arts and Sciences, and a Foreign Member of Chinese Academy of Sciences.

Dr. Lin studies the self-renewing mechanism of stem cells, stem cell-related cancers, and germline development, using Drosophila germline stem cells, mouse germline and embryonic stem cells, Hydra stem cells, and human cancer cells as models. He has made key contributions to the demonstration of stem cell asymmetric division and the proof of the stem cell niche theory. He discovered the Argonaute/Piwi gene family and their essential function in stem cell self-renewal and germline development and demonstrated their crucial function in breast and colon cancers. He is a discoverer of PIWI-interacting RNAs (piRNAs), a discovery hailed by the Science magazine as one of the 10 Scientific Breakthroughs in 2006. Recently, he proposed and demonstrated the crucial roles of the Piwi-piRNA pathway in epigenetic programming and in post-transcriptional regulation of mRNA and lncRNA.

Amander T. Clark, PhD, University of California, Los Angeles, USA is President-Elect and will serve as President in 2023. Valentina Greco, PhD, Yale Stem Cell Center, USA is the new Vice President and Clive Svendsen, PhD, Cedars-Sinai Regenerative Medicine Institute, USA, is the Societys new Treasurer.

Helen Blau, PhD, Stanford University School of Medicine, USA, and Kathy Niakan, PhD, University of Cambridge, UK were elected as new Board Members and begin their three-year terms 1 July 2022. Arnold Kriegstein, MD, PhD, University of California, San Francisco, USA and Kenneth Zaret, PhD, University of Pennsylvania School of Medicine, USA were re-appointed to another term.

Learn more.

About the International Society for Stem Cell Research (www.isscr.org)With more than 4,400 members from more than 70 countries, the International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. The ISSCR mission is to promote excellence in stem cell science and applications to human health. Additional information about stem cell science is available at A Closer Look at Stem Cells, an initiative of the Society to inform the public about stem cell research and its potential to improve human health.

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Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Haifan Lin takes on new role as President of the ISSCR - EurekAlert

Irvine, Calif., June 30, 2022 University of California, Irvine-led researchers have discovered that a signaling molecule called SCUBE3 potently stimulates hair growth and may offer a therapeutic treatment for androgenetic alopecia, a common form of hair loss in both women and men.

The study, published online today in Developmental Cell, determined the precise mechanism by which the dermal papilla cells specialized signal-making fibroblasts at the bottom of each hair follicle promote new growth. Although its well known that dermal papilla cells play a pivotal role in controlling hair growth, the genetic basis of the activating molecules involved has been poorly understood.

At different times during the hair follicle life cycle, the very same dermal papilla cells can send signals that either keep follicles dormant or trigger new hair growth, said Maksim Plikus, Ph.D., UCI professor of developmental & cell biology and the studys corresponding author. We revealed that the SCUBE3 signaling molecule, which dermal papilla cells produce naturally, is the messenger used to tell the neighboring hair stem cells to start dividing, which heralds the onset of new hair growth.

The production of activating molecules by the dermal papilla cells is critical for efficient hair growth in mice and humans. In people with androgenetic alopecia, dermal papilla cells malfunction, greatly reducing the normally abundant activating molecules. A mouse model with hyperactivated dermal papilla cells and excessive hair, which will facilitate more discoveries about hair growth regulation, was developed for this research.

Studying this mouse model permitted us to identify SCUBE3 as the previously unknown signaling molecule that can drive excessive hair growth, said co-first author Yingzi Liu, a UCI postdoctoral researcher in developmental & cell biology.

Further tests validated that SCUBE3 activates hair growth in human follicles. Researchers microinjected SCUBE3 into mouse skin in which human scalp follicles had been transplanted, inducing new growth in both the dormant human and surrounding mouse follicles.

These experiments provide proof-of-principle data that SCUBE3 or derived molecules can be a promising therapeutic for hair loss, said co-first author Christian Guerrero-Juarez, a UCI postdoctoral researcher in mathematics.

Currently, there are two medications on the market finasteride and minoxidil that are approved by the Food and Drug Administration for androgenetic alopecia. Finasteride is only approved for use in men. Both drugs are not universally effective and need to be taken daily to maintain their clinical effect.

There is a strong need for new, effective hair loss medicines, and naturally occurring compounds that are normally used by the dermal papilla cells present ideal next-generation candidates for treatment, Plikus said. Our test in the human hair transplant model validates the preclinical potential of SCUBE3.

UCI has filed a provisional patent application on the use of SCUBE3 and its related molecular compounds for hair growth stimulation. Further research will be conducted in the Plikus lab and at Amplifica Holdings Group Inc., a biotechnology company co-founded by Plikus.

The study team included health professionals and academics from UCI, San Diego, China, Japan, Korea and Taiwan.

This work was supported by LEO Foundation grants LF-AW-RAM-19-400008 and LF-OC-20-000611; Chan Zuckerberg Initiative grant AN-0000000062; W.M. Keck Foundation grant WMKF-5634988; National Science Foundation grants DMS1951144 and DMS1763272; National Institutes of Health grants U01-AR073159, R01-AR079470, R01-AR079150, R21-AR078939 and P30-AR075047; Simons Foundation grant 594598; the National Natural Science Foundation of China; the NNSFCs Major Research Plan training program; and Taiwans Ministry of Science and Technology.

About UCIs Brilliant Future campaign:Publicly launched on Oct. 4, 2019, the Brilliant Future campaign aims to raise awareness and support for UCI. By engaging 75,000 alumni and garnering $2 billion in philanthropic investment, UCI seeks to reach new heights of excellence instudent success, health and wellness, research and more. The School of Biological Sciences plays a vital role in the success of the campaign. Learn more by visitinghttps://brilliantfuture.uci.edu/school-of-biological-sciences.

About the University of California, Irvine:Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities and is ranked among the nations top 10 public universities byU.S. News & World Report. The campus has produced five Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 224 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $7 billion annually to the local economy and $8 billion statewide.For more on UCI, visitwww.uci.edu.

Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.

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UCI-led team discovers signaling molecule that potently stimulates hair growth - UCI News

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy, characterized by progressive muscle wasting and weakness and caused by abnormally repetitive DNA segments that are transcribed into toxic molecules of RNA. Instead of ferrying a gene's instructions for translation into proteins, these RNA molecules accumulate in cells, disrupting cellular machinery.

Rett syndrome (RS) is a rare genetic neurological disorder that affects the way the brain develops, resulting in progressive loss of motor skills and language early in life.

Writing in the June 29, 2022 online issue of Science Translational Medicine, researchers at University of California San Diego School of Medicine used three-dimensional brain organoids self-organized tissue grown from stem cells that mimics neurological functions to discover fundamental similarities between DM1 and RS, and perhaps therapeutic opportunities.

"We turned to 3D brain organoids that simulate the developing human cortex to study the effects of the CTG repeat expansion on neuronal processes," said first author Kathryn Morelli, PhD, a fellow in the lab of senior author Gene Yeo, PhD, professor of cellular and molecular medicine at UC San Diego School of Medicine.

"It's a model that can be made from induced pluripotent stem cell lines from real DM1 patients that carry these toxic RNA aggregates. It mimics cortical development in utero."

Unlike other types of muscular dystrophy, patients with DM1 often exhibit progressive neurocognitive symptoms, with learning and social impediments that can appear similar to autism spectrum disorders. Recent clinical data has shown that the higher the number of inherited DNA repeats, the earlier the onset of symptoms and the greater the impact of the disease on the central nervous system.

Modern DM1 treatments target only skeletal and heart muscle defects. Research by Yeo and colleagues has shown that RNA-targeting CRISPR/Cas proteins can bind to repetitive RNA in live human cells and reverse markers of disease in the skeletal muscle of mouse models of DM1.

Still, the absence of a cellular model of the human brain limited our understanding of how toxic RNA can cause cognitive symptoms, and hindered efforts to develop an effective holistic therapy, said Morelli.

In the latest study, researchers packaged a compact RNA-targeting CRISPR/Cas protein into viral vectors, then added them to DM1 brain organoids. They found that the proteins destroyed toxic RNA aggregates, with scientists able to observe and control the cascade of events.

The team has focused on a model in which toxic RNA traps a special class of proteins called RNA binding proteins or RBPs. "In cortical organoids, we were surprised to find that another RBP called CELF2 protein was dysregulated in glutamatergic neurons, which are responsible for excitatory signaling in the brain," said Yeo.

Using the enhanced cross-linking and immunoprecipitation technologies pioneered in the lab, Morelli and colleagues discovered that CELF2 did not bind its normal targets: genes in the methyl-CpG binding protein 2 (MECP2) pathway that are crucial for neuron function. Mutations that result in the loss of MECP2's normal function cause RS.

The findings, said the authors, suggestion a possible convergence in neurodevelopment defects in DM1 and RS. Morelli noted that clinical trials are underway to evaluate the therapeutic potential of N-methyl-d-aspartic acid (NMDA) antagonists for treating patients with RS. NMDA receptors are believed to be important in controlling synaptic plasticity and mediating learning and memory functions.

In DM1 organoids, Morelli found that NMDA antagonists reversed key features of the disease, suggesting that targeting NMDA receptors might ameliorate cognitive impairments in young patients with DM1, and substantially improve their quality of life.

Co-authors include: Wenhao Jin, Shashank Shathe, Assael A. Madrigal, Krysten L. Jones, Joshua L. Schwartz, Tristan Bridges, Jasmine R. Mueller, Archana Shankar, Isaac A. Chaim, all at UC San Diego; and John W. Day, Stanford University.

# # #

Science Translational Medicine

29-Jun-2022

Yeo is a co-founder, member of the board of directors, equity holder and paid consultant for Locanabio and BioInnovations, and a Scientific Adviser and paid consultant to Jumpcode Genomics. He is also a Distinguished Visiting Professor at the National University of Singapore.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Organoids reveal similarities between myotonic dystrophy type 1 and Rett syndrome - EurekAlert

LA JOLLA, Calif.--(BUSINESS WIRE)--Calidi Biotherapeutics, Inc., a clinical-stage biotechnology company that is pioneering the development of stem cell-based delivery of oncolytic viruses, today announced the appointment of W.K. Alfred Yung, M.D., Professor, Neuro-Oncology at the MD Anderson Cancer Center, to its Medical Advisory Board.

We are inspired by clinician-scientists like Dr. Yung who have dedicated their careers to advancing care for patients with deadly cancers, such as glioblastoma, for which there are few effective treatments, said Allan J. Camaisa, Chief Executive Officer and Chairman of Calidi. As clinical trials studying the safety and efficacy of Calidis oncolytic virus-based therapies progress, the expertise of seasoned clinicians like Dr. Yung will help us further our understanding of the benefits of these drugs to patients.

Dr. Yung is a fixture in the treatment of brain cancers with extensive experience studying glioblastoma, the deadliest form of brain cancer. He built the neuro-oncology department at MD Anderson Cancer Center, served as co-chair of the National Cancer Institute Brain Malignancy Steering Committee, and advised President Bidens Cancer Moonshot Initiative. Dr. Yung has published more than 350 peer-reviewed articles and served as the editor-in-chief of Neuro-Oncology.

Despite many promising new advancements in precision medicine for many cancers, glioblastoma remains one of the deadliest cancers with limited treatment successes and poor patient quality of life, said W. K. Alfred Yung, M.D. I see great promise in the potential of Calidi Biotherapeutics therapeutic vaccine which shields cancer-fighting oncolytic viruses in stem cells, helping protect the virus from a patients immune system until it reaches the cancer cell. If this approach proves successful in human trials, it could be a game changer not just for the treatment of brain cancers, but for other solid tumor cancers too.

About Calidi Biotherapeutics

Calidi Biotherapeutics is a clinical-stage immuno-oncology company with proprietary technology that is revolutionizing the effective delivery of oncolytic viruses for targeted therapy against difficult-to-treat cancers. Calidi Biotherapeutics is advancing through the FDA approval process a potent allogeneic stem cell and oncolytic virus combination for use in multiple oncology indications. Calidis off-the-shelf, universal cell-based delivery platform is designed to protect, amplify, and potentiate oncolytic viruses currently in development leading to enhanced efficacy and improved patient safety. Calidi Biotherapeutics is headquartered in La Jolla, California. For more information, please visit http://www.calidibio.com.

Forward-Looking Statement

This press release contains forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Terms such as anticipates, believe, continue, could, estimate, expect, intends, may, might, plan, possible, potential, predicts, project, should, would as well as similar terms, are forward-looking in nature. The forward-looking statements contained in this discussion are based on the Calidis current expectations and beliefs concerning future developments and their potential effects. There can be no assurance that future developments affecting Calidi will be those that it has anticipated. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Calidis control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: the occurrence of any event, change or other circumstances that could give rise to the termination of negotiations and any subsequent definitive agreements with respect to the business combination (the Business Combination) with Edoc Acquisition Corp. (Edoc); the outcome of any legal proceedings that may be instituted against Edoc, Calidi, the combined company or others following the announcement of the Business Combination, the private placement financing proposed to be consummated concurrently with the Business Combination (the PIPE), and any definitive agreements with respect thereto; the inability to complete the Business Combination due to the failure to obtain approval of the shareholders of Edoc, the possibility that due diligence completed following execution of the principal definitive transaction documents for the Business Combination and PIPE will not be satisfactorily concluded, the inability to complete the PIPE or other financing needed to complete the Business Combination, or to satisfy other conditions to closing; changes to the proposed structure of the Business Combination that may be required or appropriate as a result of applicable laws or regulations or as a condition to obtaining regulatory approval of the Business Combination; the ability to meet stock exchange listing standards following the consummation of the Business Combination; the risk that the Business Combination disrupts current plans and operations of Calidi as a result of the announcement and consummation of the Business Combination; the ability to recognize the anticipated benefits of the Business Combination or to realize estimated pro forma results and underlying assumptions, including with respect to estimated shareholder redemptions; costs related to the Business Combination; changes in applicable laws or regulations; the evolution of the markets in which Calidi competes; the inability of Calidi to defend its intellectual property and satisfy regulatory requirements; the ability to implement business plans, forecasts, and other expectations after the completion of the proposed Business Combination, and identify and realize additional opportunities; the risk of downturns and a changing regulatory landscape in the highly competitive pharmaceutical industry; the impact of the COVID-19 pandemic on Calidis business; and other risks and uncertainties set forth in the section entitled Risk Factors and Cautionary Note Regarding Forward-Looking Statements in Edocs preliminary prospectus dated March 16, 2022, in the Registration Statement on Form S-4 filed with the Securities and Exchange Commission (SEC) on March 16, 2022.

Important Information About the Business Combination Transaction and Where to Find It

This press release relates to a proposed business combination between Edoc Acquisition Corp. a Cayman Islands exempted company, EDOC Merger Sub Inc., a Nevada corporation and Calidi Biotherapeutics, Inc., a Nevada corporation. A full description of the terms and conditions Agreement and Plan of Merger constituting the business combination is provided in the registration statement on Form S-4 filed with the U.S. Securities and Exchange Commission (SEC) by Edoc Acquisition Corp., that includes a prospectus with respect to the securities to be issued in connection with the merger, and information with respect to an extraordinary meeting of Edoc Acquisition Corp. shareholders to vote on the merger and related transactions. Edoc Acquisition Corp. and Calidi Biotherapeutics, Inc. urges its investors, shareholders and other interested persons to read the proxy statement and prospectus as well as other documents filed with the SEC because these documents will contain important information about Calidi Biotherapeutics, Inc., Edoc Acquisition Corp., and the business combination transaction. After the registration statement is declared effective, the definitive proxy statement and prospectus to be included in the registration statement will be distributed to shareholders of Edoc Acquisition Corp. and Calidi Biotherapeutics, Inc., as of a record date to be established for voting on the proposed merger and related transactions. Shareholders may obtain a copy of the Form S-4 registration statement, including the proxy statement and prospectus, and other documents filed with the SEC without charge, by directing a request to: Edoc Acquisition Corp. at 7612 Main Street Fishers, Suite 200, Victor, New York 14564. The preliminary and definitive proxy statement and prospectus included in the registration statement can also be obtained, without charge, at the SECs website (www.sec.gov).

Participation in the Solicitation

Edoc Acquisition Corp., Calidi Biotherapeutics, Inc., and their respective directors and executive officers may be deemed to be participants in the solicitation of proxies or consents from Edoc Acquisition Corp. and Calidi Biotherapeutics, Inc. shareholders in connection with the proposed transaction. A list of the names of the directors and executive officers of Edoc Acquisition Corp. and Calidi Biotherapeutics, Inc. and information regarding their interests in the business combination transaction is contained in the proxy statement and prospectus. You may obtain free copies of these documents as described in the preceding paragraph.

No Offer or Solicitation

This press release will not constitute a solicitation of a proxy, consent or authorization with respect to any securities or in respect of the proposed business combination. This press release will also not constitute an offer to sell or the solicitation of an offer to buy any securities of Calidi Biotherapeutics, Inc., nor will there be any sale of securities in any states or jurisdictions in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities will be made except by means of a prospectus meeting the requirements of section 10 of the Securities Act of 1933, as amended, or an exemption therefrom.

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Calidi Biotherapeutics Announces Appointment of W.K. Alfred Yung, M.D., to its Medical Advisory Board - Business Wire

Its open season on fundamental rights, now that the Supreme Court has reversed Roe v. Wade and other landmark abortion rights cases, despite more than 60% of Americans saying abortion should be legal in all or most cases.

For those in favor of this ruling, were not hearing them cheer on the decision from a constitutional position. Instead, its based on religious and moral beliefs. Weve prayed and lobbied for this for 50 years, they say. As states scramble to regulate or eliminate abortion, we see more fallout to come.

One being, the high courts decision is disastrous for science.

The vast world of medical research, procedures and the development of critical medicines and vaccines will be severely impacted. This advanced work relies on human fetal tissue.

Fetal tissue obtained from elective abortions is uniquely adaptable and valuable to medical researchers. The tissue has been crucial in understanding normal fetal development and studies of neurological and infectious diseases, including HIV, heart disease, diabetes, Parkinson's and COVID-19. Common vaccines, such as chicken pox, rubella and shingles, were created using human fetal tissue.

Lawrence Goldstein, a distinguished professor at the University of California San Diego School of Medicine, told NPR that because fetal cells are not fully developed, they are useful in, for example, developing replacement organs.

If you're trying to make a kidney from stem cells, you'd like to know that as the cells begin going down the kidney development path that they're doing it normally," Goldstein said. Comparison to early fetal kidney cells that are doing it normally tells you that you're on the right track or not."

In 2021, the Biden administration reversed restrictions on fetal tissue research put in place by former President Trump in 2019. In the throes of the pandemic, Trump basically ended highly meritorious research projects that had already been through multiple layers of scientific and ethical reviews.

In March 2020, research institutions and medical foundations appealed to the Trump administration to lift restrictions to enable COVID-19 studies. We can only speculate how many lives may have been saved, schools kept open, and businesses and economies left to thrive if researchers had the opportunity to do the work directly in front of them. The World Health Organization estimates the number of global deaths attributable to the COVID-19 pandemic in 2020 alone is at least 3 million.

After restrictions were lifted, the U.S. National Institutes of Health no longer had to adhere to both a ban on studies and an ethical review from conservative board members opposed to abortion. Proponents of human fetal tissue research argue this endeavor is morally separate from abortion.

Longstanding, required ethical processes were already in place before the Trump administration stacked this board. The board became the place where federal funding applications went to die. Board members prevented work based on religious and moral grounds.

Conservative judges acted in this same way. The dissent opinion (by Justices Stephen Breyer, Elena Kagan and Sonia Sotomayor) said it plainly: The majority makes radical change too easy and too fast, based on nothing more than the new views of new judges. The majority has overruled Roe and Casey for one and only one reason: because it has always despised them, and now it has the votes to discard them. The majority thereby substitutes a rule by judges for the rule of law.

Giving a subject in a human trial or patient a drug thats already been tested on human tissue in a Petri dish is less likely to cause harm. This is socially responsible and for the greater good.

Consequences from the reversal of Roe v. Wade will continue to creep into our lives. The hard hit to scientific research is just one of them.

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Our View: Roe reversal hard hit to science - The Durango Herald

Scientists from the University of California, Irvine have discovered that an injury to one part of the brain changes the connections between nerve cells across the entire brain. The new research was published this week inNature Communications.

Every year in the United States, nearly two million Americans sustain a traumatic brain injury (TBI). Survivors can live with lifelong physical, cognitive and emotional disabilities. Currently, there are no treatments.

One of the biggest challenges for neuroscientists has been to fully understand how a TBI alters the cross-talk between different cells and brain regions.

In the new study, researchers improved upon a process called iDISCO, which uses solvents to make biological samples transparent. The process leaves behind a fully intact brain that can be illuminated with lasers and imaged in 3D with specialized microscopes.

With the enhanced brain clearing processes, the UCI team mapped neural connections throughout the entire brain. The researchers focused on connections to inhibitory neurons, because these neurons are extremely vulnerable to dying after a brain injury. The team first looked at the hippocampus, a brain region responsible for learning and memory. Then, they investigated the prefrontal cortex, a brain region that works together with hippocampus. In both cases, the imaging showed that inhibitory neurons gain many more connections from neighboring nerve cells after TBI, but they become disconnected from the rest of the brain.

Weve known for a long time that the communication between different brain cells can change very dramatically after an injury, saidRobert Hunt, PhD, associate professor ofanatomy and neurobiologyand director of theEpilepsy Research Centerat UCI School of Medicine whose lab conducted the study, But, we havent been able to see what happens in the whole brain until now.

To get a closer look at the damaged brain connections, Hunt and his team devised a technique for reversing the clearing procedure and probing the brain with traditional anatomical approaches.

The findings surprisingly showed that the long projections of distant nerve cells were still present in the damaged brain, but they no longer formed connections with inhibitory neurons.

It looks like the entire brain is being carefully rewired to accommodate for the damage, regardless of whether there was direct injury to the region or not, explained Alexa Tierno, a graduate student and co-first author of the study. But different parts of the brain probably arent working together quite as well as they did before the injury.

The researchers then wanted to determine if it was possible for inhibitory neurons to be reconnected with distant brain regions. To find out, Hunt and his team transplanted new interneurons into the damaged hippocampus and mapped their connections, based on the teamsearlier researchdemonstrating interneuron transplantation can improve memory and stop seizures in mice with TBI.

The new neurons received appropriate connections from all over the brain. While this may mean it could be possible to entice the injured brain to repair these lost connections on its own, Hunt said learning how transplanted interneurons integrate into damaged brain circuits is essential for any future attempt to use these cells for brain repair.

Our study is a very important addition to our understanding of how inhibitory progenitors can one day be used therapeutically for the treatment of TBI, epilepsy or other brain disorders, said Hunt. Some people have proposed interneuron transplantation might rejuvenate the brain by releasing unknown substances to boost innate regenerative capacity, but were finding the new neurons are really being hard wired into the brain.

Hunt hopes to eventually develop cell therapy for people with TBI and epilepsy. The UCI team is now repeating the experiments using inhibitory neurons produced from human stem cells.

This work takes us one step closer to a future cell-based therapy for people, Hunt said, Understanding the kinds of plasticity that exists after an injury will help us rebuild the injured brain with a very high degree of precision. However, it is very important that we proceed step wise toward this goal, and that takes time.

Reference:Frankowski JC, Tierno A, Pavani S, Cao Q, Lyon DC, Hunt RF. Brain-wide reconstruction of inhibitory circuits after traumatic brain injury. Nat Commun. 2022;13(1):3417. doi:10.1038/s41467-022-31072-2

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Connection Map Reveals Changes in the Injured Brain - Technology Networks

image:Maike Sander has been selected to direct the Max Delbrck Center for Molecular Medicine (MDC). view more

Credit: Peter Himsel / MDC

On November 1, 2022, Prof. Maike Sander will take the reins as Scientific Director and Chair of the Board of the Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC). The Supervisory Board of the MDC formally appointed her to the post on Thursday, June 16, 2022. The MDC, which is celebrating its 30th anniversary this year, is one of five Health Centers in the Helmholtz Association of German Research Centers. The internationally renowned researcher and experienced science manager Maike Sander will be succeeding Prof. Thomas Sommer, who has directed the MDC on an interim basis since 2019. That will make Sander the first woman to head one of the Helmholtz Health Centers.

The MDC has distinguished itself as an internationally renowned center for highly innovative biomedical research, says Maike Sander. Work at the MDC lays the foundation for better medicine of the future. The MDC provides on outstanding environment for research and attracts talent from around the globe. I had the opportunity to experience this first-hand as a visiting professor at the MDC. As Scientific Director, my goal will be to further strengthen the MDCs role as a leading biomedical research center and to deepen partnerships with other institutions in Berlin and beyond, so that our discoveries can be rapidly turned into medical innovations. Sander emphasizes that medical innovation needs strong basic science, clinical science and industry partners components that are all part of the vibrant Berlin biomedical ecosystem, she points out. The Berlin region is developing into a flourishing biotech pharma hub and I see the MDC as a principal driver of innovation in this landscape. I very much look forward to working with all stakeholders across Berlin.

Maike Sanders research focuses on identifying novel therapeutic approaches for diabetes. To this end, Sander studies the molecular mechanisms that underlie the formation and function of the different cell types in the pancreas, in particular the insulin-producing beta cells. Her goal is to identify strategies for replacing beta cells in diabetes using beta cells derived from human pluripotent stem cells.

Since 2012, Sander has served as the Director of the Pediatric Diabetes Research Center at the University of California, San Diego (UC San Diego), where she is also a Professor in the Departments of Pediatrics and Cellular & Molecular Medicine. In Berlin, Maike Sander will be appointed as Professor at the Charit Universittsmedizin.

Maike Sander is an outstanding scientist with a track record of innovation in biomedical research, says Bettina Stark-Watzinger, Germanys Federal Minister of Education and Research. I am delighted we have been able to bring her back to Germany after many years in the United States and to win her as the new Scientific Director of the Max Delbrck Center. It demonstrates the attractiveness of Berlin as a hub for biomedical research. As a scientist and administrator, Prof. Sander is the perfect match for the MDC with its mission to improve human health through transformative biomedical research. Also, having a female leader is an important signal. Prof. Sanders appointment represents a significant gain for German research.

Berlins Senator for Higher Education and Research, Health, Long-Term Care and Gender Equality, Ulrike Gote, says: In Prof. Maike Sander, the Max Delbrck Center has gained an internationally renowned scientist as its new Scientific Director. I warmly welcome her to the science and healthcare metropolis Berlin. Prof. Sanders expertise and experience provide the ideal background for future development of the MDC and for increasing the international visibility of the vibrant life sciences community at the MDC and in Berlin. As the senator in charge of higher education, research, and gender equality, I am delighted to see a woman at the helm of a Helmholtz Health Center.

I got to know Maike Sander as an expert in diabetes and stem cells when she was a visiting professor at the MDC, says OtmarD.Wiestler, President of the Helmholtz Association. With her high scientific standing and international experience, she is the ideal person to determine the future direction of the MDC as Scientific Director and Chair of the Board. With Prof. Sander we are gaining an excellent scientist whose expertise will be of tremendous benefit to the Helmholtz Association. A critical focus area is the development of precision medicine approaches. The MDC is at the forefront of advancing research in this important area. I look forward to working with Prof. Sander and to a vivid exchange of ideas.

Maike Sander, a native of Gttingen, is 54 years old. After graduating with a medical degree from the University of Heidelberg Medical School in 1994, she conducted research at the University of California, San Francisco. Before moving to UC San Diego in 2008, she held faculty positions at Hamburg Medical School and the University of California, Irvine. An expert on insulin-producing pancreatic beta cells, she has nearly 30 years of experience in medicine and diabetes research.

Sander is an elected member of the German National Academy of Sciences Leopoldina, the Association of American Physicians, and the American Society of Clinical Investigation. In addition, she is a member of two NIH consortia: The Human Islet Research Network and the NIH Impact of Genomic Variation on Function Consortium, which seeks to define basic mechanisms of gene regulation.

She is a recipient of the Grodsky Award of the Juvenile Diabetes Research Foundation, the 2022 Albert Renold Prize of the European Association for the Study of Diabetes, and the Alexander von Humboldt Foundation Research Award. Since 2019, Sander has been an Einstein Visiting Fellow at the Berlin Institute of Health at Charit (BIH).

30 Years MDC

Sander Laboratory and Publications at University of California, San Diego

German Federal Ministry of Education and Research (BMBF)Division for Press; Social Media; InternetKapelle-Ufer 110117 Berlin+49-(0)30-1857-5050presse@bmbf.bund.de

Jutta Kramm Head of the Staff Unit CommunicationsMax Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC)+49-(0)30-9406-2140jutta.kramm@mdc-berlin.de or presse@mdc-berlin.de

The Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC) is one of the worlds leading biomedical research institutions. Max Delbrck, a Berlin native, was a Nobel laureate and one of the founders of molecular biology. At the MDCs locations in Berlin-Buch and Mitte, researchers from some 60 countries analyze the human system investigating the biological foundations of life from its most elementary building blocks to systems-wide mechanisms. By understanding what regulates or disrupts the dynamic equilibrium in a cell, an organ, or the entire body, we can prevent diseases, diagnose them earlier, and stop their progression with tailored therapies. Patients should benefit as soon as possible from basic research discoveries. The MDC therefore supports spin-off creation and participates in collaborative networks. It works in close partnership with Charit Universittsmedizin Berlin in the jointly run Experimental and Clinical Research Center (ECRC), the Berlin Institute of Health (BIH) at Charit, and the German Center for Cardiovascular Research (DZHK). Founded in 1992, the MDC today employs 1,600 people and is funded 90 percent by the German federal government and 10 percent by the State of Berlin.

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Maike Sander named to lead the Max Delbrck Center - EurekAlert

Genes are like Egyptian hieroglyphs. Thanks to advances in whole-genome sequencing, its increasingly easy to read each DNA letter. But the strings of A, T, C, and G bring up a second puzzle: what, if anything, do they mean?

Its a problem that has haunted biologists since the completion of the Human Genome Project. By tapping into our genetic base code, the project assumed, wed be able to master control of inherited diseases, edit them at will, and easily predict the consequences of any gene that laid the foundation for our bodies, functions, and lives.

The vision didnt exactly work out. DNA sequences, while capturing extremely powerful genetic information, dont necessarily translate to indicating how our bodies behave. Genes can turn on or off in different tissues depending on the cells need. Reading a DNA sequence for any gene is like parsing the base code of a cells internal program. Theres the raw genetic codethe genotypewhich determines the phenotype, lifes software that controls how cells behave. Linking the two has taken decades of painstaking experiments, slowly building up an encyclopedia of knowledge that decodes the influence of a gene on biological functions.

A new study ramped up the effort. Led by Drs. Thomas Norman and Jonathan Weissman at Memorial Sloan Kettering Cancer Center in New York and the University of California, San Francisco, respectively, the team built a Rosetta Stone for translating genotypes to phenotypes, with the help of CRISPR.

They went big. Changing gene expression in over 2.5 million human cells, the tech, dubbed Perturb-seq, comprehensively mapped how each genetic perturbation alters the cell. The technology centers around a sort of CRISPR on steroids. Once introduced into cells, Perturb-seq rapidly changes thousands of genesa brutal shakeup at the genomic scale to see how single cells respond.

In other words, Perturb-seq is a large-scale tool that can help scientists translate DNA code to functiona Rosetta Stone for uncovering our cells inner workings. Years in the making, the dataset is open for anyone to explore.

I think this dataset is going to enable all sorts of analyses that we havent even thought up yet by people who come from other parts of biology, and suddenly they just have this available to draw on, saidNorman.

Whats the function of a gene? Its easy to think that genes are your destiny but thats far from the truth. Environmental factors, such as a massive bowl of spaghetti or a walk along the beach, can easily change gene expression, bodily functions, and potentially your body and mind.

If thats the case, whats the point of sequencing whole genomes if the outcome is always in flux? A central goal of genetics is to define the relationships between genotype and phenotype, the authors said. In other words, what does any gene actually do?

Scientists have long sought to build a bridge between genotype and phenotype. Its a painstaking process. One method, for example, perturbs genes that may be related to a disorder one by one and observes the cells behavior. Dubbed forward genetics, the idea is gene-focused rather than focusing on the phenotype. An alternative approach, reverse genetics, dives deep into how a body or mind changes with a specific genetic edit.

Each method is an uphill struggle. With over 20,000 genes in our bodies and every cell behaving slightly differently (even with the same genetic changes), deciphering a genes function often takes years, if not decades.

Is there any way to speed the process up?

Enter CRISPR. Long revered as a genetic editing multitool, the method has further blossomed into a biological translator. At its heart is a technology dubbed Perturb-seq, first published in 2016 to dissect the expression of genes. Perturb-seq makes it possible to follow the consequences of turning a gene on or off in a single cell. The method rapidly rose to fame in 2020 for its efficiency at altering multiple genes at once.

Its a huge win for cell biology, said the team. While scientists have readily chipped away at the massive web connecting genes and proteins, nailing down the role of individual genes has been a struggle. We often take all the cells where gene X is knocked down and average them together to look at how they changed, said Weissman. But sometimes when you knock down a gene, different cells that are losing that same gene behave differently, and that behavior may be missed by the average.

The idea behind Perturb-seq is pretty simple. Imagine a toddler breaking stuff and realizing what hes done after seeing the consequences. Perturb-seq uses CRISPR-Cas9 to silence multiple genes at once, which may sometimes change a cells behavior. While powerful, the tool has been hard to scale, studying at most a few hundred genetic perturbations at once for pre-defined biological questions.

So why not expand the method to the whole genome?

The advantage of Perturb-seq is it lets you get a big dataset in an unbiased way, said Norman. No one knows entirely what the limits are of what you can get out of that kind of dataset. Now, the question is, what do you actually do with it?

In the new study, the team first found the magic sauce for making genome-wide changes in human cells with CRISPR. A major point was to optimize a library of guide RNAs (sgRNAs), the bloodhounds that track down a gene. Next, they captured cells infected with CRISPR and analyzed their gene expression. Overall, the team focused on nearly 2,000 genes. Cross-referencing changed genes with each cells phenotype, they then clustered genes into networks that linked to a cellular outcome.

One enigmatic gene stood out: C7orf26. Nixing it with CRISPR changed how a cell builds a huge molecular complex, dubbed the Integrator, which helps make molecules that control gene activity. Before Perturb-seq, C7orf26 had never been associated with the complex before.

In another analysis, the team found a subset of genes that changes how daughter cells inherit the parent genome. For example, removing some genes altered the distribution of chromosomes as a cell divides. Adding or removing a chromosome can fundamentally change our biology, such as by leading to Down Syndrome.

To Norman, this aspect is the most interesting part of Perturb-seq. It captures a phenotype that you can only get using a single-cell readout. You cant go after it any other way.

This database is just the start. The team is looking to use Perturb-seq on other human cell types, and all the data is available for collaboration. With the rise of Ultima Genomics, an ultra-low-cost genomic sequencing solution, single-cell CRISPR screens are likely to play an even bigger role in biotechnology, such as in analyzing the genomes of iPSCs (induced pluripotent stem cells).

To Weissman, it may even spark a shift in how we approach cellular mysteries. Rather than defining ahead of time what biology youre going to be looking at, you have this map of the genotype-phenotype relationships, and you can go in and screen the database without having to do any experiments, he said.

Image Credit: Jen Cook/Chrysos Whitehead Institute

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Scientists Used CRISPR to Trace Every Human Gene to Its Function - Singularity Hub

An appealing alternative to the Streptococcus pyogenes CRISPR nuclease SpyCas9, are Type V CRISPR Cas12a nucleases, commonly isolated from Acidaminococcus (Asp) and Lachnospiraceae (Lba). These Cas12a nucleases embody several desirable attributes that SpyCas9 lacks: they exhibit greater editing precision, recognize a thymine-rich PAM (protospacer adjacent motifa two-to-six base sequence following the nuclease target), use a single CRISPR-RNA to detect its target, cut DNA in a staggered fashion generating overhangs, process CRISPR arrays, and have been shown to function in diverse organisms ranging from plants to mammals. However, Cas12a nucleases exhibit lower editing rates than SpyCas9 in primary cells.

In a study published in GENmagazines sister journal,GEN Biotechnology (Optimization of Nuclear Localization Signal Composition Improves CRISPR-Cas12a Editing Rates in Human Primary Cells), Scot Wolfe, PhD, professor of molecular, cell and cancer biology at the University of Massachusetts Chan Medical School and his team, increased Cas12as on-target gene editing rate to nearly 100% by engineering the configuration of the enzymes nuclear localization signal (NLS). These advancements to the Cas12a editing framework could improve the use of this nuclease to uncover functions of new genes and develop new CRISPR-based treatments.

Previous work by our laboratories and others indicated that the efficiency of Cas12a editing in CD34+ hematopoietic stem and progenitor cells could potentially be improved by increasing the efficiency of its nuclear import, said Wolfe.

In earlier studies, Wolfes team had enhanced SpyCas9 gene editing in primary cells by optimizing the NLS sequence composition and number. They had found adding one NLS at the amino-terminus and two at the carboxy-terminus of the nuclease markedly improved SpyCas9s (3xNLS-SpyCas9) editing efficiency in hematopoietic stem and progenitor cells (HSPCs). They had then added two NLSs to the carboxy-terminus of Cas12a but did not achieve the same efficiency of targeted mutagenesis as the engineered SpyCas9 with three NLSs.

Ben Kleinstiver, PhD, assistant professor of pathology at Massachusetts General Hospital and Harvard Medical School, said, Genome editing efficiency is impacted by many different variables, including the concentration of a CRISPR-Cas enzyme in the nucleus where it performs its function. Researchers have previously dedicated substantial effort to improve CRISPR nuclease expression and nuclear localization for SpyCas9, but comparatively fewer optimizations have been performed for Cas12a. (Kleinstiver was not involved in the current study).

In the current study, Wolfes team developed three NLS C-terminus variants of Cas12a where they substituted the previously used simian virus NLS (SV40) with a more efficient NLS of a proto-oncogene (c-Myc). In addition, they added a third NLS to the carboxy end to achieve an editing platform at par with 3xNLS-SpyCas9 in editing efficiency. The researchers observed increased knockout efficiency in all three Cas12a orthologs (Asp, Lba, and engineered-Asp) they tested, which suggests this triple NLS strategy could be effective in improving the activity of other members of the Cas12a family, without decreasing the enzymes inherent specificity.

The study used standard electroporation to deliver the engineered Cas12a ribonucleoproteins (RNPs) into transformed human cells lines (HEK293T, Jurkat, and K562 cells) and into primary cells (natural killer cells and CD34+ HSPCs) to improve indel frequencies.

We believe that the improved NLS sequence architecture described in this paper will increase the efficiency of genome editing by Cas12a in primary cells, thus leading to increased levels of therapeutic genome editing in a variety of applications, said Wolfe. The researchers claim this strategy of enhancing the NLS sequence can be widely applied to other Cas12a orthologs and variants with similar outcomes.

The Wolfe lab and collaborators had previously demonstrated increased activity with a new NLS framework for SpyCas, so it is exciting that they demonstrated success with a new NLS for Cas12a in this publication. It is important to have additional NLSs to test in the growing list of nucleases and cell types, said Thomas Cradick, PhD, CSO at Excision BioTherapeutics. (Cradick was not involved in the current study.)

Kleinstiver said, Luk et al., demonstrated that the efficiency of editing with various Cas12a enzymes can be improved by using a more optimal configuration of NLSs. The effect of this optimization was most striking in lipid-based transfections (nucleofections) in transformed cell lines, with a more modest improvement in primary cells, the latter of which due to already high levels of editing in primary cells.

This study resurfaces a really important consideration, that you can only edit cells as efficiently as your enzyme is designed to. There are lots of knobs to turn to optimize and improve editing efficiency, and the NLS architecture clearly plays a key role in regulating the nuclear concentration, and thus the potency, of the editor, added Kleinstiver.

Nicole Gaudelli, PhD, director and head of gene editing platform technologies at Beam Therapeutics, who was not involved in the current study, said, In addition to advancing Cas12a gene editing applications, these learnings may potentially be evaluated for other gene editing tools to further increase editing efficiencies and provide greater therapeutic benefit if higher levels of gene correction or modification can be achieved.

This study was rigorously done in multiple cell types that show the robustness of the data. I liked how they delivered Cas12 as an RNP, as this is therapeutically relevant and greatly reduces off-target editing, said Alexis Komor, PhD, assistant professor of chemistry and biochemistry at the University of California, San Diego, who was not involved in the study.

I also liked this work because it uses a very universal approach to improve editing (the modifications they made to the system can be applied to any genome editing agent), and they demonstrated its utility with multiple Cas12 enzymes (which have slightly different PAMs, which is nice). Overall, its a useful and practical study, Komor continued.

As we continue the deployment of diverse CRISPR-Cas effectors in the clinic, it is important to individually engineer each molecular machine for optimal efficiency and specificity. Here, the authors show how NLS can be optimized for enhanced activity in medically relevant human primary cells, said Rodolphe Barrangou, PhD, professor of food, bioprocessing, and nutrition at North Carolina State University (NCSU), editor-in-chief of The CRISPR Journal, and CEO of TreeCo, a company that uses CRISPR to produce genetically enhanced trees. Barrangou was not part of the current study.

Optimizing on-target mutagenesis rates whilst maintaining specificity is key for successful translation to the clinic, reaffirmed Jennifer Harbottle, PhD, a senior scientist at Horizon Discovery, who was not part of this study. The Cas12a NLS variant developed by Scot Wolfes lab holds the potential to lower dosage whilst exerting therapeutic effect.

It will be of interest to see this strategy expanded to other Type V systems, and track efficiency of delivery in a wider range of cell types and tissues, added Harbottle. Comprehensively evaluating the genomic integrity of edited cells, particularly the occurrence of structural variants and chromosomal rearrangements compared to editing by canonical Cas9 systems, will be critical to push the optimized Type V variants towards in vivo use in humans.

In future studies, Wolfe intends to continue refining Cas12a nucleases to edit specific therapeutic targets. He said, We are particularly interested in applications for certain hematopoietic disorders and muscular dystrophies.

Continued here:
CRISPR-Cas12a Editing Rates Improve with Better Directions to the Nucleus - Genetic Engineering & Biotechnology News