Category Archives: Genome

MEXICO CITY, Sept. 27, 2022 /PRNewswire/ -- Population genomics startup omica.bio proudly announces its investment in web3 data management platform GenoBank.io. This new partnership is the next phase in both companies' work to promote fair and equitable genomic research in Latin America.

With more than 600 million inhabitants and 700 ethnic groups, Latin America is one of the most diverse regions in the world. However, less than 1% of all genomic data is of Latinx origin. "Not only does the lack of genomic data from Latin America exacerbate health disparities for the Latinx community, but it leads to missed scientific opportunities for the global population," said Victor Angel-Mosti, CEO and founder of omica.bio. "Our goal, however, is not only to add genetic diversity to genomic research, but to do so with forward-thinking bioethical standards."

As part of this partnership, genobank.io will provide omica.bio with web3 capabilities for the deployment of the first tokenized consent protocol. "The aim of this collaboration is to offer research participants unprecedented transparency and traceability over the use of their genomic and clinical data," said Daniel Uribe, Founder and CEO of genobank.io. The technology will be deployed as part of a omica.bio's effort to sequence 10,000 whole-genomes from rare disease patients throughout Mexico and Central America.

"We believe the promise of personalized health will only be fulfilled through the power of community. GenoBank.io's platform offers a stepping stone towards a trusted bioethics standard across the globe." said Angel-Mosti. "We invite the research community to follow along at http://www.omica.bio."

CONTACT: Victor Angel Mosti, [emailprotected]

SOURCE OMICA VM REGENERATIVA SAPI DE CV

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Omica.bio and GenoBank.io Partner to Drive Transparency in Genomic Research in Latin America - PR Newswire

The National Institutes of Health on Thursday announced more than $600 million in fresh funding for an expansive and ongoing push to unravel the mysteries of the human brain, bankrolling efforts to create a detailed map of the whole brain, and devise new ways to target therapeutics and other molecules to specific brain cell populations.

Scientists across the country are involved, from teams at the Salk Institute to Duke University to the Broad Institute of MIT and Harvard, among other places. If successful, they will help answer fundamental questions about the bodys most complex organ. What are all the cell types in the brain? How are they connected to one another? How do the workings of the brain change during disease, and what can we do about that?

So far, those questions have proven easier to ask than to answer, with researchers gleaning bits of information from individual studies, but the hope is that a broad-based effort will jump-start new revelations.

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The fresh funding adds to $2.4 billion that the NIH has already invested in related projects. By 2026, the agency will have spent $5 billion. The scientists who will be spearheading the research openly compare its scale and scope to the push to sequence the first human genome in the 1990s and early 2000s.

I really do view this as like the Human Genome Project. We have the ability now to define cells like we were able to define genes, said Ed Lein, a neuroscientist at the Allen Institute in Seattle. This is the foundation to start to understand a lot of other aspects of biology and disease.

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The latest announcement is part of a continuing effort known as Brain Research Through Advancing Innovative Neurotechnologies (BRAIN), which was unveiled by the Obama administration in 2013 and kicked off in 2014. Its goal: to better understand the 86 billion cells that populate the brain and the trillions of connections they form with one another.

Its arguably the most sophisticated computer that we know, said John Ngai, director of the BRAIN Initiative. Its a highly complex organ whose connections and organizational principles we do not come close to understanding, and there was a realization that we needed better tools.

Since then, BRAIN-related grants have funded around 1,200 studies and led to 5,000 research publications. Ngai also measures the programs success in its life-changing impact on some patients. In 2021, researchers at the University of California, San Francisco deciphered brain signals from a paralyzed man who hadnt spoken in over 15 years and used his attempts to talk to generate words that appeared on a screen. Last November, researchers at Baylor College of Medicine launched a clinical trial for patients with depression that is testing the benefits of deep brain stimulation, an approach that uses electrical jolts to stimulate brain circuits and which has proven helpful for disorders such as Parkinsons disease.

These were experiments based on concepts that were literally science fiction 10 years ago maybe even five years ago, Ngai said.

The new round of funding, dubbed BRAIN 2.0, seeks to build on this progress. Eleven grants are going to groups that are building a comprehensive atlas of the brain, a sort of parts list and 3D map of what cells are there and how they are organized. The Allen Institute will lead one key piece of this project: Mapping the whole brains of humans as well as marmosets and macaques, two monkey species often used in neuroscience research.

We know that the neuron types in the front of the brain cortex [are] very different from the ones in the back of the brainstem, said Hongkui Zeng, who will be leading the Allen Institutes efforts along with Lein. But we dont know how theyre different. We also dont know the extent of the diversity.

BRAIN-funded scientists have successfully mapped the mouse brain, Zheng says, and plan to publish those findings soon. And researchers will rely on many of the same cutting-edge experimental tools to study primates and people.

One technique, known as spatial transcriptomics, allows researchers to understand where different cells are located. To do so, researchers first break up brain tissue, isolate cell nuclei, and use sequencing to understand what genes are active in that cell. By doing this across many cells, scientists find groups of cells that tend to use the same set of genes. They can then look at thin slices of brain tissue and search for activation of those genes to find where certain cells are present.

Its an audacious task our brains are about 3,000 times bigger than a mouses, Lein says. To start, his team will use autopsy tissue from roughly six people to build an initial map, which they plan to make publicly available. Thats enough to build a basic atlas, though understanding person-to-person variation will require looking at many more samples in the future.

Lein says that even a preliminary map could help researchers find cell types that are damaged by a certain neurological disorder or that may be responsible for a disease. For instance, his group has already been studying the brains of people with Alzheimers disease and has identified cell types that die off during disease as well as others that become more abundant.

Researchers at the Salk Institute in San Diego will focus on 50 brain regions to understand how they change with age. The plan is to use roughly 30 samples from infants all the way to people in their 70s and 80s, according to Joseph Ecker, head of the Salk-led effort.

The team will focus on so-called epigenetic changes. These are changes that dont alter a cells genetic code but control the activation of genes in other ways, often through small chemical modifications of DNA and changes in how the genome is packed and organized.

At each one of these stages during the lifespan, there are diseases that probably impact those cell types, Ecker said. We want to be able to understand how the normal brain develops so we can compare it to various disease states.

He adds that understanding the rules behind gene regulation in the brain could allow researchers to precisely target specific cell types. Thats the aim of another aspect of BRAIN 2.0, with seven grants going to developing experimental tools that can reach specific regions of the brain. Many of these efforts are focused on adeno-associated viruses, a class of viruses that are already popular in gene therapy.

And theres more work to come. Ngai says that a third pillar of BRAIN 2.0 wont launch until early 2023: Understanding the dizzying array of connections that brain cells in one area make with cells in other far-flung regions.

One of the basic challenges facing researchers will be how to process and present their findings in a clear, intelligible way to the public. Case in point: The Salk group alone will likely generate 11 petabytes of data, which is enough to fill up nearly 172,000 USB drives.

I think thats going to be our greatest challenge, Zeng said. Its not just a matter of collecting data its also the matter of conveying.

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NIH launches the next stage of its 'human genome project' for the brain - STAT

View Larger Image David Ussery, Ph.D.

Sept. 26, 2022 | LITTLE ROCK The rapid spread of monkeypox is unlike the virus past outbreaks and may be a result of genetic mutations identified by University of Arkansas for Medical Sciences (UAMS) researchers.

Led by UAMS David Ussery, Ph.D., the UAMS team published its findings this month in the Journal of Applied Microbiology.

The team compared the genomes of the 2022 virus to monkeypox genomes from a 2017 outbreak in Nigeria, plus sequenced genomes from localized outbreaks in 1965 and 1970. None of the previous monkeypox variants spread beyond their place of origin in Africa.

The UAMS teams bioninformatics analysis using advanced genomic sequencing methods revealed 25 mutations, 14 of which appear to change protein function and bear further research, said Ussery, a professor in the College of Medicine Department of Biomedical Informatics and director of the Arkansas Center for Genomic and Epidemiology Medicine at UAMS.

At least one of the differences we found could be responsible for why the current virus is causing a pandemic while past strains of monkeypox viruses did not, he said.

The teams article notes that the current monkeypox virus outbreak is not only the largest known outbreak to date, the infections result in much different clinical and epidemiological features compared to previous outbreaks.

In July, the World Health Organization declared the monkeypox outbreak a global health emergency.

While the virus is not usually lethal, its genetic makeup is strikingly similar to smallpox, Ussery said, so health officials and researchers are monitoring it closely. Smallpox killed an estimated 300500 million people in the 20th century before a vaccine campaign eradicated the virus by 1979.

Monkeypox is 99.5% identical to smallpox, Ussery said. It is so closely related that if you are old enough to have been vaccinated for smallpox, you are likely protected against monkeypox.

The research teams findings are a starting point for additional investigation in the lab, he said. A follow-up study will be needed to identify the changed properties of the monkeypox virus and to test which mutations are responsible for the virus increased ability to spread.

Co-authors on the publication are:

Usserys work is supported in part by the National Institutes of Health (NIH), grant 1P20GM121293; the UAMS Translational Research Institute, which is funded by the NIH National Center for Advancing Translational Sciences, award UL1 TR003107; the National Science Foundation, award OIA-1946391; and the Arkansas Research Alliance.

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UAMS Researchers Find Changes in Monkeypox Genome That May Explain Its Recent Rapid Spread - UAMS News

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Improved pea reference genome and pan-genome highlight genomic features and evolutionary characteristics - Nature.com

EQS-News: BRAIN Biotech AG / Key word(s): Personnel/Strategic Company DecisionChange in the Executive Management Board of BRAIN Biotech AG, genome editing activities established under the Akribion Genomics brand 27.09.2022 / 07:30 CET/CESTThe issuer is solely responsible for the content of this announcement.

Lukas Linnig stepping down as CFO. Michael Schneiders to become new CFO of BRAIN Biotech AG as of October 1st, 2022

Lukas Linnig and Dr. Michael Krohn to jointly lead Akribion Genomics activities

Akribion Genomics established as new brand for the proprietary BRAIN genome editing platform technology

Zwingenberg, September 27, 2022 BRAIN Biotech AG has announced that current Chief Financial Officer Lukas Linnig is stepping down as CFO to lead Akribion Genomics, the newly established brand for the highly promising genome editing platform technology of BRAIN Group. Michael Schneiders, currently Head of Investor Relations & Sustainability, will be appointed as the new Chief Financial Officer of BRAIN Biotech AG.

Michael Schneiders has joined the company in May 2020 after more than twenty years in investment banking. He has also been involved in the initial public offering of BRAIN. The personnel changes in the executive management board of BRAIN Biotech AG will become effective as of October 1st, 2022. Linnig will be joined at Akribion Genomics by Dr. Michael Krohn, currently Head R&D BRAIN Biotech. Linnig and Krohn will jointly lead the activities under the brand name to fully capitalize on the great promise that genome editing represents to BRAIN and its customers. It is BRAINs stated intention to establish a separate legal entity for Akribion in the next year and to attract significant external growth funding for this venture.

Adriaan Moelker, CEO of BRAIN Biotech AG, says: Lukas Linnig and Michael Krohn have been essential in leading our genome editing activities over the last years, driven by the great scientific progress made by the R&D team. It is only logical that they will now become the appointed leaders to drive Akribion Genomics towards its stated goals. Michael Krohn will assume his leadership role as soon as his successor takes over the R&D leadership at BRAIN. I am also looking forward to working together with Michael Schneiders on the executive board of BRAIN Biotech AG. Michael has proven IR, financial and business development skills and a well-established capital market track record.

Dr. Georg Kellinghusen, Chairman of the Supervisory Board of BRAIN Biotech AG, says: "It is a very strong sign of confidence in the business potential of Akribion Genomics that the envisaged spin-out will be led by the high caliber team of Lukas Linnig and Dr. Michael Krohn. I am also happy that we have been able to capitalize on our internal succession planning with Michael Schneiders as the designated CFO of BRAIN Biotech."

Lukas Linnig states: It has been a great honor for me to serve BRAIN as its CFO. Leading Akribion, I will be focusing my attention on accelerating one of BRAINs most important value drivers with transformational potential. I am thrilled to take on this challenge of building a successful business in the genome editing market under the Akribion Genomics brand.

Dr. Michael Krohn, adds: I am convinced about the potential of our proprietary genome editing technology, especially since our unique mode of action enables us to offer a very valuable addition to the existing genome editing toolsets.

About BRAIN

BRAIN Biotech AG (BRAIN) is a leading European specialist in industrial biotechnology with a focus on nutrition, health and the environment. As a technology and solution provider, the company supports the biologization of industry with biobased products and processes. From contract research and development with industrial partners to the development of own disruptive incubator projects and customized enzyme products: BRAIN's broad, innovative biotech know-how and its agile teams are the key to success.

The German BRAIN Biotech AG is the parent company of the international BRAIN Group, which distributes B2B specialty products, including enzymes and bioactive natural products. The BRAIN Group has its own fermentation or production facilities in continental Europe, the UK and the USA, which complete the value chain within the Group with the associated biotechnological production know-how.

As a participant in the United Nations Global Compact, BRAIN Biotech AG is committed to aligning its strategies and activities with the universal principles on human rights, labor, environment and anti-corruption, and to actively promote common social goals. Our products and services directly target at least five of the UN SDGs.

Since its IPO in 2016, BRAIN Biotech AG has been listed in the Prime Standard of the Frankfurt Stock Exchange (ISIN DE0005203947 / WKN 520394).

Contact Investor Relations

Michael Schneiders

Head of Investor Relations & Sustainability

Tel.: +49 6251 9331-86

E-mail: mis@brain-biotech.com

Contact media

Dr. Stephanie Konle

PR & Corporate Communications

Tel.: +49 6251 9331-70

E-mail: stk@brain-biotech.com

Follow @BRAINbiotech on Twitter (https://twitter.com/BRAINbiotech) and on LinkedIn (https://www.linkedin.com/company/brainbiotech)

Disclaimer

This press release contains forward-looking statements. These statements reflect the current views, expectations and assumptions of the management of BRAIN Biotech AG and are based on information currently available to management.

Forward-looking statements are not guarantees of future performance and involve known and unknown risks and uncertainties. The actual future results of BRAIN Biotech AG and the BRAIN Group and developments concerning BRAIN Biotech AG and the BRAIN Group may therefore differ materially from the expectations and assumptions expressed herein due to various factors. These factors include, in particular, changes in the general economic situation and the competitive situation. In addition, developments on the financial markets and exchange rate fluctuations, as well as national and international legislative changes, particularly with regard to tax regulations, and other factors may have an impact on the future results and developments of BRAIN Biotech AG.

BRAIN Biotech AG assumes no obligation to update the statements contained in this release.

27.09.2022 CET/CEST Dissemination of a Corporate News, transmitted by EQS - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

The EQS Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases.Archive at http://www.eqs-news.com

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Change in the Executive Management Board of BRAIN Biotech AG, genome editing activities established under the Akribion Genomics brand - Yahoo Finance...

Washington [US], September 27 (ANI): Mutations can drastically help or hurt the odds of an organism surviving and reproducing. Researchers have created a computer program called ExtRaINSIGHT that tracks the history of harmful mutations throughout human evolution. Theyve discovered several regions of the genome are especially vulnerable to mutations, meaning any mutations there could lead to severe or lethal consequences.

Siepels program is called ExtRaINSIGHT. It finds harmful mutations by looking for their absence. By random chance, every part of the human genome should have mutations but some have none. Siepel calls these places ultraselected. When they occur, the mutations there can be deadly or drastically hurt the odds of reproducing. Siepel explains:

If we look across a panel of a hundred thousand humans and we never see a mutation at a particular gene, that suggests that any mutation that did occur was so harmful, that anyone carrying that mutation died out from the population.

The team analyzed over 70,000 human genomes with ExtRaINSIGHT. They discovered that three parts of the genome have been extremely sensitive to mutations over generations. Of these, splice sites are the most sensitive. Splice sites help produce correct instructions for making proteins. Mutations here can have a huge impact on the odds of passing down genes, also known as fitness. Theyre linked to several diseases including spinal muscular atrophy, the leading genetic cause of death in infants and toddlers. Siepel says:

If you see a mutation in a splice site, you better take it seriously. That mutation alone would reduce your fitness by 1 or 2%. That doesnt sound like very much, but thats a huge fitness effect. And if you had multiple of these, pretty soon your chance of passing on your genes might be close to zero.

Molecules called miRNA and central nervous system genes are also sensitive. If you find a mutation in miRNA theres a good chance its responsible for a genetic disease, Siepel says. And because the nervous system is so complex and interconnected, it seems particularly sensitive to mutation.

The origins of many genetic diseases and conditions remain a mystery. Siepel hopes technology like ExtRaINSIGHT will help reveal their origins and guide diagnoses and future treatments. He also hopes his work will help further illustrate how mutations continue to shape the evolution of the human genome. (ANI)

This report is auto-generated from ANI news service. ThePrint holds no responsibility for its content.

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Exposing the evolutionary weak spots of the human genome: Research - ThePrint

When is a pandemic over? Did biotech over-SPAC? And what cant be CRISPRd?

We cover all that and more this week on The Readout LOUD, STATs biotech podcast. Heidi Tworek, a professor at the University of British Columbia and expert on public health communication, joins us to discuss President Bidens declaration that the pandemic is over and how leaders around the world are talking about Covid-19 as it enters its third year. Well also discuss the latest news in the life sciences, including the twilight of the SPAC boom, the coming evolution of genome editing, and the next big trial in Alzheimers disease.

For more on what we cover, heres the reaction to Bidens remarks; heres more on CRISPR; heres a look at the next Alzheimers readout; heres where you can find episodes of Color Code; heres where you can subscribe to the First Opinion Podcast;and heres our complete coverage of the Covid-19 pandemic.

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Be sure to sign up on Apple Podcasts, Spotify, Stitcher,Google Play, or wherever you get your podcasts.

And if you have any feedback for us topics to cover, guests to invite, vocal tics to cease you can email [emailprotected].

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Biden's Covid declaration, twilight of the SPAC, & genome editing 2.0 - STAT

The Genome Engineering Market Industry research forecast to 20222028 offers in-depth market information to help companies develop growth strategies and make better business decisions based on forecasts and market trends. The studys marketing variables include the dynamic market structure, key players product offerings, their difficulties, technical innovation, roadblocks and hurdles, data on communication and sales, sales by country, risk, prospects, competitive landscape, growth strategy, and others. It delves extensively into the situation of the market, both now and in the future. The study examines several elements, such as levels of development, technical advances, and the various business models employed by the markets current top players.

The Genome Engineering market study is divided into several sections, including product type, application, end-user, and geography. Each segment is assessed based on its CAGR, market share, and growth potential. The study emphasizes the prospective region in the regional analysis, which is projected to generate chances in the worldwide Genome Engineering market in the next years. This segmented study will absolutely prove to be an invaluable tool for readers, stakeholders, and industry participants seeking a comprehensive view of the global Genome Engineering market and its growth prospects in the future years.

Global genome engineering market is estimated to be valued atUS$ 5,205.60 millionin2022and is expected to exhibit aCAGRof14.3%during the forecast period(2022-2030).

@ https://www.coherentmarketinsights.com/insight/request-sample/1262

Major Key playersare : Thermo Fisher Scientific Inc., CRISPR Therapeutics AG, Intellia Therapeutics, Inc., Editas Medicine, Inc., Sangamo Therapeutics, Inc., Bluebird Bio, Inc., Cellectis S.A., and Merck Group.

SWOT Analysis of Global Genome Engineering Market

In addition to market share analysis of companies, in-depth profile, product/service, and business overview, the study focuses on revenue analysis, as well as SWOT analysis, to better correlate market competitiveness.

Information source and Research Methodology:

Our researchers compiled the study utilizing primary (surveys and interviews) and secondary (industry body databases, reliable paid sources, and trade magazines) data collection methods. The research includes a thorough qualitative and quantitative analysis. The study examines growth trends, micro- and macroeconomic data, legislation, and government efforts.

Market Dynamics

Increasing strategic collaboration for genome engineering technologies by key players is expected to drive market growth over the forecast period. Key players in market are focusing on strategic collaborations, in order to increase their product offerings. For instance, in February 2018, Kite Pharma, Inc., a Gilead Sciences, Inc. company, collaborated with Sangamo Therapeutics Inc. for developing engineered cell therapies to treat cancer. As per the agreement, Kite Pharma, Inc. would use Sangamo Therapeutics zinc finger nuclease (ZFN) gene-editing technology for developing next-generation ex vivo cell therapies for treatment of cancer. Furthermore, in 2017, Synthego and Thermo Fisher Scientific collaborated to manufacture and distribute synthetic guide ribonucleic acid (RNA) products for CRISPR genome engineering.

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Table of Contents:

Purchasing the Genome Engineering Market for the Following Reasons:

The study analyses emerging market trends as well as the likelihood that different trends will have an impact on expansion.The analysis also covers the factors, challenges, and opportunities that will significantly affect the worldwide Genome Engineering industry.Technological tools and benchmarks that mirror the projected growth of the Genome Engineering industry.In order to provide futuristic growth estimates, the research includes a detailed analysis of market statistics and historical and present growth conditions.The research includes a comprehensive analytical overview of the competitive environment, as well as highlights on fundamental capabilities and growth plans of the profiled businesses.

What are the goals of the report?

The predicted market size for the Genome Engineering Market Industry at the conclusion of the forecast period is shown in this market report.The paper also analyses market sizes in the past and present.The charts show the year-over-year growth (percent) and compound annual growth rate (CAGR) for the given projected period based on a variety of metrics.The research contains a market overview, geographical breadth, segmentation, and financial performance of main competitors.The research evaluates the current situation of the industry in North America, Asia Pacific, Europe, Latin America, the Middle East, and Africa,as well as future growth opportunities.The study examines the future periods growth rate, market size, and market worth.

What our reports offer:

Market share assessments for the regional and country-level segments Strategic recommendations for the new entrants Covers market data for 2021, 2022, till 2028 Market trends (drivers, opportunities, threats, challenges, investment opportunities, and recommendations) Strategic recommendations in key business segments based on the market estimations Competitive landscaping mapping the key common trends Company profiling with detailed strategies, financials, and recent developments Supply chain trends mapping the latest technological advancements

Examine market data, tables, and figures in detail. The most recent independent research report on a wide range of market development strategies and business approaches, including product and service development, joint ventures, partnerships, mergers and acquisitions, and so on. Market company profiles comprise Business Overview, Product / Service Offerings, SWOT Analysis, Segment & Total Revenue, Gross Margin, and% Market Share in order to provide a more complete picture. This Genome Engineering study examines market definitions, an overview, a classification, and segmentation, including market type and applications, before moving on to product details, production plans, pricing schemes, raw material sourcing, and supply chain analysis.

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Genome Engineering Market Report 2022 with Leading Key Players and Regional Analysis 2028 | Thermo Fisher Scientific Inc., CRISPR Therapeutics AG,...

NEW YORK Researchers have uncovered shared genetic liability between autism spectrum disorder (ASD) and attention-deficit hyperactivity disorder (ADHD), but also genetic distinctions between the conditions.

ASD and ADHD are common neurodevelopmental disorders affecting children that may last into adulthood. They are also highly heritable and polygenic, and previous studies have suggested the two conditions are genetically correlated.

To look into just which genetic variants are shared by the conditions, an international team of researchers led by Aarhus University's Anders Brglum conducted genome-wide association studies using the Psychiatric Genomics Consortium (PGC) and the Lundbeck Foundation for Integrative Psychiatric Research (iPSYCH) cohorts. As they reported in Nature Genetics on Monday, they found seven loci shared by ASD and ADHD but also five loci that differentiate them.

"[W]e have disentangled the shared and differentiating genetic liability underlying ASD and ADHD, identifying shared and disorder-specific risk variants providing information on pathophysiology," Brglum, director of the iPSYCH program, and colleagues wrote in their paper.

The researchers conducted a combined genome-wide association study of diagnosed ASD or ADHD in a cohort of 34,462 cases and 41,201 controls. After identifying more than 260 SNPs in seven distinct loci, the researchers conducted a transcriptome-wide association study to home in on putative causal shared genes. This identified five genes or isoforms that were differentially expressed between the case and control groups. Further gene-based analysis using the tool MAGMA confirmed those TWAS findings and identified two additional shared loci, for a total of seven genetic loci shared between ASD and ADHD.

These shared loci tended to be pleiotropic and had been identified in previous GWAS of related disorders or cross-disorder studies, the researchers noted.

Meanwhile, to identify loci that differ between ASD and ADHD, the researchers performed an ADHD versus ASD GWAS using 9,315 ASD-only and 11,964 ADHD-only cases from the iPSYCH cohort. This analysis uncovered five genome-wide significant loci, three of which had not previously been tied to either ASD or ADHD.

Those loci, though, have been linked to related disorders or traits, particularly cognitive traits. Four of the loci, for instance, have been associated with cognitive ability or neuroticism, and two with educational attainment. The lead variants further exhibited opposite direction of effect in the two conditions.

In a TWAS, the top gene or isoform identified that differentiated the disorders was HIST1H2BD-201, located on chromosome 6. Deleterious or de novo variants in histone-modifying or histone-related genes have previously been linked to autism and developmental delay with features of autism. Here, the researchers found that the expression of HIST1H2BD-201 was lower in ASD compared to ADHD. Further, the ASD risk allele of the lead SNP there was linked to better educational performance and increased volume of the left globus pallidus in the brain.

The researchers proposed that the ADHD-ASD differentiating locus on chromosome 6 affects the expression of HIST1H2BD-201 and left globus pallidus volume and, in turn, traits like educational performance, social interaction, and motor impairments. This pushes the disorder presentation toward ASD or ADHD.

The researchers additionally conducted a GWAS of 2,304 individuals diagnosed with both ASD and ADHD, as well as a polygenic risk score-based analysis. They found that these comorbid cases had an ASD-PRS load similar to that of ASD-only cases as well as an ADHD-PRS load similar to that of ADHD-only cases, indicating they are "double-loaded" with genetic predispositions for both disorders. This provides support, they noted, for the recent change in diagnostic guidelines allowing for the diagnosis of both ASD and ADHD in the same individual.

"The results advance our understanding of the complex etiologic basis of ASD and ADHD and the relationship between the two disorders, toward the long-term goals of better diagnosis and treatment of these disorders," the authors wrote.

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Autism, ADHD Found to Have Overlapping and Distinct Genetic Contributions - GenomeWeb

ALAMEDA, Calif.--(BUSINESS WIRE)--Scribe Therapeutics Inc., a molecular engineering company pioneering a CRISPR by Design platform for genetic medicine, today announced a strategic collaboration with Sanofi for the use of Scribes CRISPR genome editing technologies to enable genetic modification of novel natural killer (NK) cell therapies for cancer.

The agreement grants Sanofi non-exclusive rights to Scribes proprietary CRISPR platform of wholly owned enzymes to create ex vivo NK cell therapies. Scribes suite of custom engineering genome editing and delivery tools called CasX-Editors (XE), based on novel foundations such as the CasX enzyme, will support Sanofis expanding pipeline of NK cell therapeutics for oncology.

Were pleased to provide Sanofi with access to Scribes proprietary and enhanced gene editing technologies for use in ex vivo oncology applications distinct from our current pipeline, said Benjamin Oakes, Ph.D., co-founder and CEO of Scribe. Scribe is proud to expand the use of our XE CRISPR technologies with the team at Sanofi, whose commitment to deep scientific rigor and clinical development experience will enable the rapid advancement of novel ex vivo cell therapies for patients in need.

At Sanofi, we are pushing the boundaries of science by developing a diverse range of next-generation therapies based on natural killer (NK) cells, which could have broad applications across solid tumors and blood cancers, said Frank Nestle, Global Head of Research and Chief Scientific Officer, Sanofi. This collaboration with Scribe complements our robust research efforts across the NK cell therapy spectrum and offers our scientists unique access to engineered CRISPR-based technologies as they strive to deliver off-the-shelf NK cell therapies and novel combination approaches that improve upon the first generation of cell therapies."

Deal Terms

Under the terms of the agreement, Scribe will receive $25 million in upfront payment and be eligible to potentially receive more than $1 billion in payments based on development and commercial milestones, as well as tiered royalties on net future sales on any products that may result from this research agreement.

About Scribe Therapeutics

Scribe Therapeutics is a molecular engineering company focused on creating best-in-class in vivo therapies that permanently treat the underlying cause of disease. Founded by CRISPR inventors and leading molecular engineers Benjamin Oakes, Brett Staahl, David Savage, and Jennifer Doudna, Scribe is overcoming the limitations of current genome editing technologies by developing custom engineered enzymes and delivery modalities as part of a proprietary, evergreen CRISPR by Design platform for genetic medicine. The company is backed by leading individual and institutional investors including Andreessen Horowitz, Avoro Ventures and Avoro Capital Advisors, OrbiMed Advisors, Perceptive Advisors, funds and accounts advised by T. Rowe Price Associates, Inc., funds managed by Wellington Management, RA Capital Management, and Menlo Ventures. To learn more about Scribes mission to engineer the future of genetic medicine, visit http://www.scribetx.com.

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Scribe Therapeutics Announces Research Collaboration with Sanofi to Accelerate Breakthrough CRISPR-based Cell Therapies for Cancer - Business Wire