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Genetic Engineering – Meaning, Applications, Advantages …
Posted: February 17, 2022 at 8:45 am
Genetic engineering, also calledgenetic modification, is the direct manipulation of an organismsgenomeusing biotechnology. It is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novelorganisms. Read important facts about Genetic Engineering in this article for the IAS Exam.
NewDNAmay be inserted in the host genome by first isolating and copying the genetic material of interest usingmolecular cloningmethods to generate a DNA sequence, or by synthesizing the DNA and then inserting this construct into the host organism.Genesmay be removed, or knocked out, using anuclease.Gene targetingis a different technique that useshomologous recombinationto change an endogenous gene and can be used to delete a gene, removeexons, add a gene, or introducepoint mutations.
Aspirants reading, GEAC can also refer to topics lined below:
Medicine, research, industry and agriculture are a few sectors where genetic engineering applies. It can be used on various plants, animals and microorganisms. The first microorganism to be genetically modified is bacteria.
Genetic Engineering Appraisal Committee (GEAC) is the biotech regulator in India. It is created under the Ministry of Environment and Forests. Read more about GEAC in the linked article.
There are five bodies that are authorized to handle rules noted underEnvironment Protection Act 1986 Rules for Manufacture, Use, Import, Export and Storage of Hazardous Microorganisms/Genetically Engineered Organisms or Cells 1989. These are:
Soybean-Herbicide tolerance,Canola-Altered fatty acid composition,Plum-Virus resistance,Corn-Insect resistance
Pros:Tackling and Defeating Diseases,Getting Rid of All Illnesses in Young and Unborn Children,Potential to Live Longer,Produce New Foods,Faster Growth in Animals and Plants,Pest and Disease Resistance.Cons:May Lead to Genetic Defects,Limits Genetic Diversity,Reduced Nutritional Value,Risky Pathogens,Negative Side Effects
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Genetic Engineering in Humans Pros and Cons List | NYLN.org
Posted: at 8:45 am
Genetic engineering in is founded on the idea of manipulating the gene pool in order to make lives better. One way of doing this is to start from the basic, from the egg cell and sperm cell. Another way is to swap bad genes in a fully formed human with good ones.
There are moral and ethical controversies surrounding genetic engineering or genetic mutation in humans. Personal convictions alone dictate people what to oppose and what to accept. However, it takes an objective inspection of this medical technology for us to draw a more acceptable conclusion and prevent pre-created biases.
1. Helps Prevent Genetic DisordersMany of the diseases today are hereditary or genetic. By manipulating the genes in humans, scientists find a way to prevent people from suffering from an otherwise hereditary health condition.
2. Helps Individual Have Better LifeGenetic engineering helps humans have a chance at a healthier, longer life with more desirable physical characteristics. By altering the genes of fetuses, there is a strong likelihood that future generations will be taller, stronger, healthier and better looking.
3. Helps Deepen Understanding of GenesPromoting genetic engineering is one way of deepening our understanding about human genetics. It helps scientists find ways to cure or prevent hereditary diseases, most especially.
4. Allows Parents to Choose Babys TraitsSome parents would want their children not to inherit their less desirable traits, if given the chance. By modifying the genes of babies, parents have a chance at designing their own babies, according to what they want gender, color of hair, etc.
5. Probes into Medical AdvancementsThere are many areas in science, which continue to be a mystery to even the most learned scientists and researchers today. Other advancements in the medical field can spring from genetic engineering.
1. Test Failure Leads to Termination of EmbryosSince genetic engineering is not a perfect science, and far from being so, there will be failures along the way, and this leads to termination of embryos with undesirable gene pool. To some people, this is tantamount to abortion.
2. Who Decides the Good and Bad GenesNo one has the right to decide or judge what specific traits are good or bad. With genetic engineering, the power likely rests on the scientists, the future parents, or the political leader. However, are these people accountable or responsible when experiments go wrong?
3. Engineered Babies Could Have Worse Imperfections When the actual results are not the outcome initially intended, society could have grave issues regarding the presence of erroneously engineered humans, specifically if they turn out to be mentally ill, psychotic, abusive, or non-responsive. How does society control these badly designed humans by murder, by further experimentation or by imprisonment?
4. It Is Very ExpensiveEngineering the genes of animals is already intricate and expensive enough, how much more an entire human being? It takes a team of skilled geneticists and researchers, plus a topnotch facility, to perform the experiment. This means that genetic engineering may only be available to the wealthy, furthering the gap in society.
5. Reduces the Individuality among HumansWhen there is a consensus as to which traits are good or bad, there is a tendency for future generations to lose their diversity and individuality. There will be no short people because being tall is more desirable. There will be no fat people because being slender is more desirable. Ultimately, the reduction of undesirable traits in humans would lead to a generation of pure breeds with very little capability of adapting to changes in the environment as in the case of pure breed animals, which are prone to disease.
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Unnatural Natural Products the story behind a photo – The John Innes Centre
Posted: at 8:45 am
Sometimes an adventure in science history begins with a chance find in a junk shop.
In the summer of 2021, a member of the public found a framed 1984 photograph of Professor David Hopwood in a shop in Surrey. We dont know how it came to be there, but we were delighted to be able to acquire it for the John Innes Centre Archives where (now Professor Sir) David Hopwoods papers are housed.
An artefact like this demands a back-story.
Although the photo has a press release pasted on the back, we wanted to know more about the circumstances behind this specially commissioned image.
The first port of call was David himself. His records were able to take us back to 1984 85 and some of the background behind the making and reproduction of this image.
The story begins with a request for David to give a 40-minute talk at the British Association for the Advancement of Science meeting in Norwich on Friday 14th September 1984.
The British Association (BA) had only visited Norwich on three previous occasions (1868, 1935 and 1961). Their fourth visit to Norwich would take place for the first time at a university venue (the University of East Anglia was by then twenty years old), with the visiting delegates able to stay on campus for the princely sum of 11 per night.
The largest scientific meeting of its kind in the UK, the BAs typical audience was described then as scientists and technologists from disciplines other than that of lecturer, representatives of industry, commerce and the professions, teachers, school students and university students, and the general public with an interest in science, its applications and implications.
At the time, in the week of the BA meeting, the national press devoted more column inches to science than at any other time of year.
David accepted the invite to the conference, and his talk appeared in the meeting programme as, Advances in genetic engineering allow the isolation of genes for antibiotic production from Streptomyces organisms, the major antibiotic producers. This will lead to more efficient antibiotic production and to useful new compounds.
However, this brief abstract doesnt reveal the breakthrough that David was about to announce. His team at the John Innes Institute had produced the first hybrid antibiotic by genetic engineering.
David had an advance publicity call from the BA press officer- could David provide 70 copies (photocopy or stencil) of his finished paper and a 1-page abstract for distribution to the journalists and broadcasters attending?
The next request was from Nexus, the UEA Student Union television service (dubbed Norwichs secret TV channel). Nexus had its own studio in the Student Union building, and a low power transmitter enabled them to broadcast to the TV viewing rooms at the end of the building and some of the halls of residence, but the audience was primarily students who would watch at lunchtimes on closed circuit TV in the foyer of the Union building.
Nexus had secured an agreement with the University to provide a TV and information service throughout the BA conference and wanted to pre-record a short informal interview with David to be shown the day before the address.
He responded to these requests by phone or post, this was before the advent of email.
So, what was the breakthrough that Davids paper was about to announce to the world and why was it important? David later described this piece of work as the closest he got to a eureka moment in his career (as recalled in 2011 in an interview with Professor Tony Maxwell for BBCs Bang goes the theory).
Davids work centred on the genetics and microbiology of a strain of Streptomyces coelicolor, an Actinomycete. This group of soil-dwelling bacteria had originally fascinated biologists because they appeared to be intermediate between bacteria and fungi.
That taxonomic question was solved in the 1950s when it was confirmed that they are true bacteria and only resemble fungi, growing in mycelial colonies with aerial hyphae and spores (to the naked eye they have a fuzzy appearance like mould when grown on an agar plate).
It was the knowledge that these microbes could provide valuable drugs for medicine that spurred many laboratories to continue studying them. Actinomycetes secrete antibiotics in nature to help them compete with other microorganisms living in the soil.
The discovery of Streptomycin in 1943 (isolated from a strain of Streptomyces griseus), the first effective treatment for TB, resulted in the setting up of drug discovery labs around the world, mostly in pharmaceutical companies.
For many years the key to finding new antimicrobial drugs was to look at the natural products of the soil. By 1984, streptomycetes had yielded more than 70 commercially important antibiotics, but it was thought that most of what the soil had to offer had been developed.
The hunt for new drugs was becoming harder and harder. David Hopwoods breakthrough offered an alternative approach the possibility of making unnatural natural products to extend the range of available compounds.
Davids lab (including Dr Francisco Malpartida and Helen Kieser at the John Innes Institute, now John Innes Centre), in collaboration with a Japanese group, had produced the worlds first hybrid antibiotic called mederrhodin A by genetic engineering.
Genes for antibiotic production were first cloned from the parent strain of Streptomyces coelicolor. The cloned genes were introduced into another streptomycete, one that usually makes a brown coloured antibiotic called medermycin.
The genetically engineered streptomycete then began to secrete a new, purple-coloured antibiotic (mederrhodin A) made by both sets of genes working in collaboration. Spectroscopic analysis by Professor Satoshi muras group at the Kitasato Institute, Tokyo confirmed its hybrid chemical structure.
The appearance of the purple colour was Davids eureka moment. The experiment had demonstrated the exciting possibility of creating new compounds by transferring genes between different organisms that make different compounds.
Scientists had already mastered the trick of getting one species of bacteria to manufacture a compound normally made by another species (by gene transfer), indeed Davids lab had first demonstrated the cloning of the complete set of actinorhodin genes in this way, but producing a totally novel compound by genetic engineering, this was new.
With this experiment the new pharmaceutical field of unnatural natural products was born. These methods also offered the prospect of cloning genes controlling rate-limiting steps in antibiotic biosynthesis, which could improve yields in antibiotic production.
This work was only possible because there had been three decades of prior research to gain a detailed knowledge of the genetics of streptomycetes (starting with Davids PhD studies in Cambridge in the 1950s), and the recent development of effective methods for the isolation and manipulation of Streptomyces genes. But thats another story.
Davids archive enables us to track some of the papers that covered the news of the discovery. The first UK clipping is from Hospital Doctor on 20th September 1984, soon followed by The Economist (22nd September), then the New Scientist (4th October) and The Scotsman (28th November).
It is likely that the UK publicity was generated directly from the BA meeting, but Davids paper also had an interesting afterlife, and the picture from the junk shop is the clue to it.
The Central Office of Information (COI) wanted to organize some publicity on the new hybrid antibiotic, and commissioned two colour photographs of David, one where he appears above a table of petri dishes (pictured here) and another where he is seen working with an array of lab flasks (the original of this second image has not yet been traced).
The COI was a government communications and marketing department (successor to the Ministry of Information) that provided publicity for other organisations in the public sector and was especially tasked with promoting British inventions and discoveries overseas.
It was after their involvement that news of a hybrid antibiotic breakthrough began to travel around the world. The COI photographs were published for the first time in January 1985 in the Arabic language press (Anwar and Amal in Beirut). Illustrated articles afterwards appeared in Trinidad, Jedda, Bogota, Montevideo, Germany, and France.
The formal announcement of the hybrid antibiotic discovery was made in Nature in April 1985 , recording the contributions of Hopwood, Malpartida, Kieser, H. Ikeda, J. Duncan, I. Fujii, B.A.M. Rudd, H.G. Floss and S. mura.
An American group, led by Professor Heinz Floss at Ohio State University, Columbus, had characterised a second hybrid antibiotic made by a strain arising when Davids group had transferred actinorhodin genes from S. coelicolor into a culture making another blue compound called dihydrogranaticin, but this did not give rise to a change in colour, so it was not so dramatic as the mederhodin case.
That 1985 landmark was the catalyst for the introduction of genetic approaches that transformed natural product chemistry (see for example, L. Katz speaking for the Society for Industrial Microbiology in 2003).
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Decoding the dangers of ticks – Cosmos
Posted: at 8:45 am
It may now be possible to reduce tick-borne diseases, such as Lyme disease, thanks to two new gene-editing methods developed by researchers in the U.S. The new tools could be used to further understand tick biology, and potentially alter parts of the tick genome involved in harbouring and transmitting pathogens.
Ticks are parasites that consume animal and human blood. There are 70 species found in Australia 16 of which have been reported to feed on humans. While there isnt evidence of locally acquired Lyme disease in Australia, ticks are nonetheless vectors of other bacteria which cause diseases such as Q fever, Queensland tick typhus, Flinders Island spotted fever, and Australian spotted fever.
Ticks are a formidable foe to public health, says Jason Rasgon, professor of entomology and disease epidemiology at Pennsylvania State University in the US. We are in desperate need of new tools to fight ticks and the pathogens they spread.
In the first study to demonstrate genetic modification in ticks, the team used two different protocols using the CRISPR/Cas9 system a gene-editing complex which allows the cutting of DNA at a targeted location in the genome to add or remove sequences of DNA.
This process is usually done by injecting the CRISPR/Cas9 into embryos, but until now this has been impossible to do in tick eggs due to their hard wax coating.
For many years, people thought it would be impossible to make a transgenic tick because tick eggs are coated in a hard wax that shattered the delicate glass needles used for injections, says Rasgon.
The researchers were able to circumvent this problem by removing the maternal organs that make this wax prior to the ticks laying their eggs. This resulted in eggs that could be injected with the complex to successfully make deletions in two different genes.
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Then, using a different protocol, they injected the CRISPR/Cas9 directly into pregnant adult female ticks and used a process called ReMOT Control to target the ovaries specifically. A small peptide (a short chain of amino acids) which binds to receptors on the ovaries of most insect species was fused to the Cas9, allowing the complex to be delivered into the developing ovaries to edit the genomes of the offspring.
This is the first research demonstrating that this peptide is functional in ticks.
They found when applied to a gene called ProbB, the gene-editing efficiencies of both protocols embryo injections (14%) and ReMOT Control (11.7%) were similar. Gene-editing efficiency is a measure of the extent to which the procedure alters the gene, and refers to the frequency of insertion/deletion mutations that occur.
The ReMOT Control protocol was just as efficient as embryo injection and significantly easier, says Rasgon.
In the United States, alone, ticks infect approximately 300,000 people with Lyme disease each year, and if left untreated, the infection can spread to joints, the heart and the nervous system, he adds. Currently, there is no vaccine, and existing treatments are not always fully effective.
In Australia, brown dog ticks are also the vector for the deadly dog disease Ehrlichiosis. First detected in May 2020, the ticks have gone on to infect dogs in northern Western Australia, the Northern Territory, and northern South Australia.
This research, published in iScience, is particularly valuable because climate change is allowing ticks to rapidly invade new areas, and putting even more people and animals at risk of infections.
The methods can be used to develop new control methods for diseases, such as Lyme disease, and also to further understand the biology of ticks, says Rasgon.
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Plant Genomics For Oilseeds And Pulses Market : Forecast by Key Products, Types, Application, Regions and Overview of History The UB Post – The UB…
Posted: at 8:45 am
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Top Key players covered in this report are: Eurofins Scientific, Illumina Inc, NRGene, Neogen Corporation, Agilent Technologies, Inc, LC Sciences.LLC, Traitgenetics GmbH, Keygene N.V, Novogene Co.Ltd, GeneWiz, BGI, Genotypic Technology and FLORAGENEX, INC
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Plant Genomics For Oilseeds And Pulses Market : Forecast by Key Products, Types, Application, Regions and Overview of History The UB Post - The UB...
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Will Global Repository for Cell Engineering improve Openness and Collaboration? – Labmate Online
Posted: at 8:45 am
An international team led by Newcastle University has developed a species and strain database that uses cell barcodes to monitor and track engineered organisms and also molecularly link that data to the associated living samples. Supporting international collaboration CellRepro also has significant safety advantages, such as limiting the impact of deliberately or accidentally released genetically modified microorganisms by enabling faster tracing of organisms lab of origin and design details.
It is built on version control, a concept from software engineering that records and tracks changes to software code and the scientists believe this will make engineering biology more open, reproducible, easier to trace and share and more trustworthy.
Lead author Natalio Kasnogor, Professor of Computer Science and Synthetic Biology atNewcastle Universitys School of Computing, said: Engineering biology is not rocket science. It is much, much harder. And because of that it is imperative that we do it more openly and more collaboratively. CellRepo, at its core, is a collaboration platform in which cell engineers can document their work and share it with others (within their own lab or more widely). By enabling more collaboration and the seamlessly sharing of engineered strains we hope to accelerate and improve synthetic biology processes and reporting for everybody. CellRepo is a community resource and as such we invite engineering biologists, synthetic biologists, biotechnologists and life scientists more generally to try it and get in touch with us so we know what works and what needs to be improved!
Dr Jonathan Tellechea, a synthetic biologist in the project added: "I have always had some misidentification issues during my projects. Fortunately I was able to find them early on and solve them but I cant imagine how many good projects have failed or stalled because of this. Some other chunk of my time as a biologist goes into retroactively building the history of the plasmids and strains I use. I may get the genetic material from someone, but who was the original author? Sometimes I am lucky and it is just one paper away, sometimes its down a rabbit hole that may end up in the 80s. CellRepo fixes these and other important problems for experimentalists."
Study co-author, ProfessorVictor de Lorenzo from the Systems and Synthetic Biology Program at theCentro Nacional de Biotecnologia in Madrid said: As a software engineer coming from industry to academia, it has been both a challenge and pleasure to work on a project where I can use my skills for the public good. Version control is a staple of software engineering and I am proud that we are now bringing these tools to engineering biology.
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Protein behind the Fate of Neural Stem Cells and Nerve Cell Production Uncovered – Genetic Engineering & Biotechnology News
Posted: at 8:45 am
Understanding how neural stem cell activity is regulated and maintained has critical implications for regenerative approaches following brain trauma and disease. Now, researchers at the University of Cologne, led by Matteo Bergami, PhD, from the CECAD Cluster of Excellence for Aging Research, have discovered that the protein YME1L is essential in coordinating the shift between cellular proliferation and quiescence.
The article, Metabolic control of adult neural stem cell self-renewal by the mitochondrial protease YME1L, was published in Cell Reports.
The transition between quiescence and activation in neural stem and progenitor cells (NSPCs) is coupled with reversible changes in energy metabolism with key implications for lifelong NSPC self-renewal and neurogenesis, the researchers wrote. How this metabolic plasticity is ensured between NSPC activity states is unclear. We find that a state-specific rewiring of the mitochondrial proteome by the i-AAA peptidase YME1L is required to preserve NSPC self-renewal.
Our results show that the activity of a single mitochondrial protease can significantly affect the fate of neural stem cells and the production rate of new nerve cells. These findings not only reveal a new layer of regulation in the biology of neural stem cells but may also have important implications for patients bearing mutated YME1L, Bergami said.
Together, our results reveal YME1L as playing a critical role in acutely shaping the mitochondrial proteome of NSPCs, adding an important layer of regulation in the mechanisms governing NSPC metabolic state transitions beyond potential changes in gene expression, concluded the researchers.
Understanding how neural stem cell activity is regulated and maintained can help pave the way for new strategies following brain trauma and disease.
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Aridis’ Pan-Coronavirus Monoclonal Antibody Cocktail AR-701 Is Protective in COVID-19 Omicron Infected Animals – PRNewswire
Posted: at 8:45 am
LOS GATOS, Calif., Feb. 17, 2022 /PRNewswire/ -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, announced today that both of its fully human monoclonal antibodies (mAbs) in the AR-701 cocktail neutralized the SARS-CoV-2 Omicron variant. Moreover, both mAbs conferred complete protection against Omicron infected animals when given either parenterally or by intranasal administration.
Aridis Pharmaceuticals has recently received a $1.9 million (USD) grant from the Bill and Melinda Gates Foundation to evaluate the prevention of influenza and SARS-CoV-2 (COVID-19) viral transmission using inhaled delivery of monoclonal antibodies.
"We believe these exciting animal efficacy results are the first of any COVID antibody program to show this level of broad reactivity and efficacy, including in Omicron infected models. Given large scale clinical data from others which showed mAbs are effective as a COVID-19 preventative treatment, we think that AR-701 is well positioned for pan-coronavirus prophylaxis" commented Vu Truong, Ph.D., CEO of Aridis Pharmaceuticals. "In addition to being broadly reactive against all COVID-19 variants, we have previously shown AR-701's effectiveness against SARS, MERS, and seasonal influenza viruses, and importantly, it is engineered for long-acting effectiveness, potentially lasting a year or more when used in humans," continued Dr. Truong. "AR-701 is just one of several exciting programs in our diverse pipeline, which includes two Phase 3 programs in bacterial pneumonia and a Phase 2 program in cystic fibrosis. We look forward to sharing further updates as we continue to move these programs forward."
About AR-701AR-701 is a cocktail of two fully human immunoglobulin G1 (IgG1) mAbs discovered from screening the antibody secreting B-cells of convalescent SARS-CoV-2 infected (COVID-19) patients. Each mAb of the AR-701 cocktail neutralizes coronaviruses using a distinct mechanism of action, namely inhibition of viral fusion and entry into human cells (AR-703) or blockage of viral binding to the human 'ACE2' receptor (AR-720). The activity of the two mAbs complement and enhance each other in a synergistic fashion, creating a potent first-in-class cocktail. AR-703 binds to the 'S2' stalk region of spike proteins from betacoronaviruses, including the SARS-CoV-2 variants (beta, gamma, delta, epsilon), and binds to the Omicron variant with no loss in affinity compared to the original Wuhan strain. Multiple animal challenge models widely used to evaluate COVID-19 treatments support the broad efficacy of AR-701 against the original Wuhan wildtype strain, the Delta variant, and the severe acute respiratory syndrome virus (SARS). The AR-701 mAbs are engineered to be active for 6-12 months in the blood. AR-701 is being developed as a long-acting intramuscular as well as a self-administered inhaled formulation for the treatment of COVID-19 patients who are not yet hospitalized. AR-701 mAbs were discovered through a collaboration with researchers at the University of Alabama in Birmingham and Texas Biomedical Research Institute (San Antonio, TX).
About Aridis Pharmaceuticals, Inc.Aridis Pharmaceuticals, Inc. discovers and developsnovel anti-infective therapies to treat life-threatening infections, including anti-infectives to be used as add-on treatments to standard-of-care antibiotics. The Company is utilizing its proprietaryPEXTMandMabIgX technology platforms torapidly identify rare, potent antibody-producing B-cells from patients who have successfully overcome an infection, and to rapidly manufacture monoclonal antibody (mAbs) for therapeutic treatment of critical infections. These mAbs are already of human origin and functionally optimized for high potency by the donor's immune system; hence, they technically do not require genetic engineering or further optimization to achieve full functionality.
The Company is advancing multiple clinical stage mAbs targeting bacteria that cause life-threatening infections such as ventilator associated pneumonia (VAP)andhospital acquired pneumonia (HAP), in addition topreclinical stage antiviral mAbs. The use of mAbs as anti-infective treatments represents an innovative therapeutic approach that harnesses the human immune system to fight infections and is designed to overcome the deficiencies associated with the current standard of care which is broad spectrum antibiotics. Such deficiencies include, but are not limited to, increasing drug resistance, short duration of efficacy, disruption of the normal flora of the human microbiome and lack of differentiation among current treatments. The mAb portfolio is complemented by a non-antibiotic novel mechanism small molecule anti-infective candidate being developed to treat lung infections in cystic fibrosis patients. The Company's pipeline is highlighted below:
Aridis'Pipeline
AR-301(VAP).AR-301 is a fully human IgG1 mAb targeting gram-positiveStaphylococcus aureus(S. aureus)alpha-toxin and is being evaluated in a global Phase 3 clinical study as an adjunctive treatment of S. aureus ventilator associated pneumonia (VAP).
AR-320(VAP).AR-320 is a fully human IgG1 mAb targeting S. aureusalpha-toxin that is being developed as a preventative treatment of S. aureus colonized mechanically ventilated patients who do not yet have VAP. Phase 3 is expected to be initiated in 2Q22.
AR-501(cystic fibrosis).AR-501 is an inhaled formulation of gallium citrate with broad-spectrum anti-infective activity being developed to treat chronic lung infections in cystic fibrosis patients.This program is currently in Phase 2a clinical development in CF patients.
AR-701(COVID-19). AR-701 is a cocktail of fully human mAbs discovered from convalescent COVID-19 patients that are directed at multiple protein epitopes on the SARS-CoV-2 virus. It is formulated for delivery via intramuscular injection or inhalation using a nebulizer. AR-701 replaces AR-712 as the company's leading COVID mAb candidate.
AR-401(blood stream infections).AR-401 is a fully human mAb preclinical program aimed at treating infections caused by gram-negativeAcinetobacter baumannii.
AR-101(HAP).AR-101 is a fully human immunoglobulin M, or IgM, mAb in Phase 2 clinical development targetingPseudomonas aeruginosa(P. aeruginosa)liposaccharides serotype O11, which accounts for approximately 22% of allP. aeruginosahospital acquired pneumonia cases worldwide.
AR-201(RSV infection). AR-201 is a fully human IgG1 mAb out-licensed preclinical program aimed at neutralizing diverse clinical isolates of respiratory syncytial virus (RSV).
For additional information on Aridis Pharmaceuticals, please visithttps://aridispharma.com/.
Forward-Looking StatementsCertain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. These statements may be identified by the use of words such as "anticipate," "believe," "forecast," "estimated" and "intend" or other similar terms or expressions that concern Aridis' expectations, strategy, plans or intentions. These forward-looking statements are based on Aridis' current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the need for additional financing, the timing of regulatory submissions, Aridis' ability to obtain and maintain regulatory approval of its existing product candidates and any other product candidates it may develop, approvals for clinical trials may be delayed or withheld by regulatory agencies, risks relating to the timing and costs of clinical trials, risks associated with obtaining funding from third parties, management and employee operations and execution risks, loss of key personnel, competition, risks related to market acceptance of products, intellectual property risks, risks related to business interruptions, including the outbreak of COVID-19 coronavirus, which could seriously harm ourfinancial condition and increase our costs and expenses,risks associated with the uncertainty of future financial results, Aridis' ability to attract collaborators and partners and risks associated with Aridis' reliance on third party organizations. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Aridis' 10-K for the year ended December 31, 2020 and Aridis' other filings made with the Securities and Exchange Commission.Forward-looking statements included herein are made as of the date hereof, and Aridis does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.
Contact:Media Communications:Matt SheldonRedChip Companies Inc.[emailprotected]1-917-280-7329
Investor RelationsDave GentryRedChip Companies Inc.[emailprotected]1-800-733-2447
SOURCE Aridis Pharmaceuticals, Inc.
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Aridis' Pan-Coronavirus Monoclonal Antibody Cocktail AR-701 Is Protective in COVID-19 Omicron Infected Animals - PRNewswire
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SpliceBio Raises EUR 50M in Oversubscribed Series A financing to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline – Yahoo Finance
Posted: at 8:45 am
- Financing co-led by UCB Ventures and Ysios Capital with participation by New Enterprise Associates, Gilde Healthcare, Novartis Venture Fund and Asabys Partners
- Unique Protein Splicing platform enables efficient delivery of large genes with adeno-associated vectors (AAV)
- Proceeds will be used to advance the lead program in Stargardt disease into the clinic and expand pipeline to other currently untreatable genetic diseases
BARCELONA, Spain, Feb. 16, 2022 /PRNewswire/ -- SpliceBio, a biotechnology company exploiting protein splicing to develop next generation gene therapies, today announced the completion of an oversubscribed 50 million series A financing. The financing was co-led by UCB Ventures and existing shareholder Ysios Capital and joined by new investors New Enterprise Associates (NEA), Gilde Healthcare, Novartis Venture Fund, and existing shareholder Asabys Partners. The Company was seeded in 2020 by Ysios Capital and Asabys Partners.
Adeno-associated viruses (AAV) are the gene therapy vector of choice for the treatment of genetic diseases. However, their small packaging capacity is a major challenge for the development of novel gene therapies. SpliceBio's Protein Splicing platform aims to address this major limitation to enable the efficient delivery of large genes using AAV vectors. The platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research. In this novel approach, engineered inteins catalyze highly efficient protein trans-splicing to reconstitute the desired full-length therapeutic protein in vivo.
The proceeds from the financing, the largest Series A round for a Spanish biotech company, will enable SpliceBio to build a pipeline of Protein Splicing gene therapy programs, while advancing the lead program in Stargardt disease to the clinic. Stargardt disease is the most common form of juvenile macular dystrophy affecting more than 80,000 people in US and EU. The disease is caused by a loss of function mutation in the ABCA4 gene, which at 6.8 kb is too large for single AAV vectors. The Company will focus its efforts on ophthalmology as well as other disease areas of significant unmet patient need. The platform has been validated in several other organs beyond the retina.
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Miquel Vila-Perell, PhD, Co-Founder and Chief Executive Officer of SpliceBio, said: "We are very pleased to attract this outstanding syndicate of institutional and corporate investors which validates our approach to developing next generation gene therapies. I am excited to lead an exceptional team as we continue to build our platform and advance our pipeline of gene therapy programs into the clinic."
Following the closing of the financing, the Board of SpliceBio chaired by Jean Philippe Combal will include: Erica Whittaker, UCB Ventures; Jol Jean-Mairet, Ysios Capital; Ed Mathers, NEA; Arthur Franken, Gilde Healthcare; Beat Steffen, Novartis Venture Fund; and Miquel Vila-Perell, CEO.
Erica Whittaker, Vice President and Head of UCB Ventures, stated: "We are delighted to support SpliceBio in the development of its innovative platform to create treatments for patients suffering from genetic diseases not currently addressable by existing gene therapy approaches."
Jol Jean-Mairet, Managing Partner at Ysios Capital, added: "We are proud to have been involved with the Company since its early days and are very impressed with the progress achieved to date. SpliceBio's platform represents an unprecedented opportunity to expand the universe of diseases that can be addressed with gene therapy. This financing is also a testament to the growing potential of the biotech hub in Barcelona."
Ed Mathers, General Partner at NEA, commented: "We are very pleased to back this team, building on the founders' early-stage research at Princeton's Muir Laboratory to develop SpliceBio into a world leading gene therapy player. We believe SpliceBio's innovative approach to maximizing the capacity of AAV vectors has the potential to make a meaningful impact in the delivery of much needed gene therapies, and we look forward to supporting the Company through its next stages of growth."
About SpliceBio
SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. The Company's proprietary platform enables efficient delivery of large genes with adeno-associated vectors (AAV), overcoming the most fundamental challenge in the quest to curing a broad range of genetic diseases. SpliceBio's platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research. For additional information, please visit http://www.splice.bio.
About Split Inteins
Inteins are auto-processing domains found in organisms from all domains of life. These proteins carry out a process known as protein splicing, which is a multi-step biochemical reaction comprised of both the cleavage and formation of peptide bonds. While the endogenous substrates of protein splicing are specific essential proteins found in intein-containing host organisms, inteins are also functional in exogenous contexts and can be used to chemically manipulate virtually any polypeptide backbone. After more than 20 years of pioneering intein research characterizing the structure-activity relationship of inteins and optimizing their properties, SpliceBio's co-founders have developed a new generation of engineered split inteins designed for therapeutic use. The Company has developed additional proprietary technologies that altogether conform its Protein Splicing platform.
About Stargardt disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration, affecting 1 in 8,000 people in the world, including children. There are no treatments currently available for Stargardt patients.
About UCB Ventures
UCB Ventures is a strategic corporate venture fund established in 2017 to further strengthen UCB's ability to create value from novel insights and technologies that can transform the lives of patients suffering from severe diseases. UCB Ventures invests in innovative therapeutics and technology platforms that are early stage and high risk, in areas adjacent to or even beyond UCB's therapeutic focus on neurology/neurodegenerative diseases, immunology and muscular skeletal/bone health. UCB Ventures takes an active role in its portfolio companies, contributing expertise in drug discovery, development, and operations. Visit http://www.UCBVentures.com to learn more.
About Ysios Capital
Ysios Capital is a leading Spanish venture capital firm that provides private equity financing to early- and mid-stage, highly innovative life science companies bringing life-changing treatments to patients, with a focus on indications with high unmet need. Our diverse international team in San Sebastin and Barcelona is driven by science, with the ambition to transform capital into medical breakthroughs. Ysios Capital was founded in 2008 and has over $450 million in assets under management through its three funds. For more information, please visit http://www.ysioscapital.com.
About New Enterprise Associates
New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With nearly $24 billion in cumulative committed capital since the firm's founding in 1977, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 230 portfolio company IPOs and more than 390 mergers and acquisitions. http://www.nea.com.
About Gilde Healthcare
Gilde Healthcare is a specialized healthcare investor with two fund strategies: Venture&Growth and Private Equity. The firm operates out of offices in Utrecht (The Netherlands), Frankfurt (Germany) and Cambridge (United States). Gilde Healthcare Venture&Growth invests in fast growing, innovative companies active in (bio)pharmaceuticals, healthtech and medtech that are based in Europe and North America. For more information, please visit: http://www.gildehealthcare.com.
About Novartis Venture Fund
Novartis Venture Fund is a financially driven corporate life science venture fund whose purpose is to foster innovation, drive significant patient benefit and generate superior returns by creating and investing in innovative life science companies at various stages of their development. For more information, go to http://www.nvfund.com.
About Asabys Partners
Asabys Partners is a venture capital manager firm specialized in the healthcare sector. With close to 120 million euros in AUM and 12 portfolio companies (including 1 exit), Asabys invests in healthcare companies that have highly innovative and disruptive technologies. The investment in SpliceBio is partly financed by its first investment vehicle, Sabadell Asabys Health Innovation Investments SCR, SA, whose anchor investor is Banc Sabadell. The fund's investment in the company benefits from the financial backing of the European Union under the European Fund for Strategic Investments ("EFSI") set up under the Investment Plan for Europe. The purpose of EFSI is to help support financing and implementing productive investments in the European Union and to ensure increased access to financing. For more information, visit: http://www.asabys.com
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SpliceBio Raises EUR 50M in Oversubscribed Series A financing to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline - Yahoo Finance
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The beating heart of a swimming robot – Ars Technica
Posted: at 8:45 am
Lee et al.
Most muscles in our bodies only act in response to incoming nerve signals, which have to trigger each individual muscle cell to contract or relax. But heart muscle is different. The impulses that trigger contraction in heart muscle are passed from one muscle cell to its neighbors, leading to a rhythmic wave of contractions. This is so thoroughly built into the system that a sheet of heart muscle cells in a culture dish will start contracting spontaneously.
Now, researchers have taken advantage of some of the unique properties of cardiac cells to build a swimming robot fish powered by nothing but sugar. And while they tried to craft the heart's equivalent of a pacemaker, it turned out not to be needed: the right arrangement of muscle cells got the fish swimming spontaneously.
In some ways, the paper describing the new robot fish is a tribute to our growing ability to control stem cell development. The researchers behind the paper, based at Harvard, decided to use cardiac muscle cells to power their robot. A couple of years ago, this would have meant dissecting out a heart from an experimental animal before isolating and growing its cardiac cells in culture.
For the robot fish, stem cells were better. That's because stem cells are easier to genetically manipulate, and they are easier to grow into a uniform population. So, the team started with a population of human stem cells and went through the process needed to direct their development so they would form cardiac muscle cells.
A thin layer of these cells was placed inside a thin slice of gelatin, which held the cells in place on the flanks of the "fish" (one slice on either side). The center of the fish was flexible, so a contraction of the muscle on the right flank would pull the tail to the right, and the same would work for the opposite side. By alternating left and right contractions, the fish would pull its tail from side to side, propelling it forward. Beyond that, the fish had a large dorsal "fin" that contained a buoyancy device to keep the beast oriented upright and prevent it from sinking. The whole thing was powered by putting it in a solution with sugar, which the cardiac muscle cells would absorb.
Perhaps because of this simplicity, the robot was so durable that it was able to swim for well over three months after its construction. Performance was decent at first but improved over the first month as the cardiac cells better integrated into a coherent muscle. Ultimately, the fish was able to travel more than a body length per second. At that pace, the robot was remarkably efficientper unit of muscle mass, its swimming speed was better than that of actual fish.
One of the things that helped enable the robot fish's efficiency is notable by its absence in the photo above: any sort of control circuitry. The researchers actually tested a number of ways of controlling the muscles but ultimately found that the simplest option was the best.
The first attempt to control the muscles relied on a bit of genetic engineering. Muscles are triggered to contract by the influx of ions, normally triggered by nerve impulses. But researchers have identified some proteins that act as light-activated ion channels, which will create an influx of ions in response to specific wavelengths of light. So, the researchers engineered the cells on one flank to be sensitive to red light and those on the other sensitive to blue. This worked well, allowing alternating flashes of red and blue light to swim the fish forward.
The second method the researchers tried was inspired by the structure of the heart, which contains a cluster of cells that acts as a pacemaker by triggering a contraction that spreads from there. The researchers formed a ball of cardiac cells to act as a pacemaker and made a bridge of cells that connected the cardiac cells to the flank muscles. The influx of ions that started in the pacemaker cells could spread to the muscles, driving a contraction.
This worked to a degree but turned out to be of secondary importance. The two muscles, the researchers discovered, paced each other's contractions.
Cardiac muscle cells also have stretch receptors. Pull on the cell too much, and the receptor will be activated and trigger a contraction. This turned out to provide a built-in coordination for the flank muscles. As one right side contracted, it caused the cells on the opposite side to stretch. Once they hit a critical point, the stretch receptors on the left side would trigger that muscle to contract, stretching the right. That stretch then restarted the cycle.
This wouldn't work indefinitely, and the two muscles would eventually go out of synch. That's when the pacemaker could help get them back into a regular cycle.
Overall, this is far more impressive than useful (unless you're the sort who's only impressed by useful things). There aren't a lot of situations that call for a robot to swim through a solution of sugar, after all. But the fact that researchers were able to figure out how to use the basic biological properties of these cells to craft an effective machine certainly fits my definition of impressive.
Science, 2022. DOI: 10.1126/science.abh0474 (About DOIs).
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The beating heart of a swimming robot - Ars Technica
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