Category Archives: Transhuman News

An international team of researchers, led by scientists at University of California San Diego and Shiley Eye Institute at UC San Diego Health, has broadened and deepened understanding of how inherited retinal dystrophies (IRDs) affect different populations of people and, in the process, have identified new gene variants that may cause the diseases.

The findings published in the October 18, 2021 issue of PLOS Genetics.

IRDs are a group of diseases, from retinitis pigmentosa to choroideremia, that result in progressive vision loss, even blindness. Each IRD is caused by at least one gene mutation, though mutations in the same gene may lead to different IRD diagnoses.

IRDs are rare, but they affect individuals of all ages, progressing at different rates, even within families afflicted with the same disease. Specific diagnosis depends on finding the genetic causative mutations.

The U.S. Food and Drug Administration has approved gene therapy for treating one form of IRD involving the gene RPE65, but for other IRDs caused by mutations in more than 280 different genes, there are no cures or treatments proven to slow disease progression.

The researchers conducted whole-genome sequences (WGS) of 409 persons from 108 unrelated family lineages, each with a previously diagnosed IRD. WGS is a process of determining the entirety, or near-entirety, of the DNA sequence of an individual. It provides a comprehensive portrait of the persons entire genome, including mutations and variants, which can be used for broad comparative purposes.

Study participants were recruited from three different geographic regions: Mexico, Pakistan and European Americans living in the United States. Genomic analyses were conducted from blood samples taken from all participants, which revealed causative variants in 62 of the 108 lineages. A total of 94 gene variants were found in the 62 families: 52 variants had previously been identified as causative and 42 had not. Surprisingly, more than half of the new variants were not listed in the Genome Aggregation Database, an international compilation of genomic data.

Overall, causative variants were detected in 63 percent of Mexican participants, 60 percent of Pakistani, and 48 percent of European American.

The study also identified a large proportion of new IRD causative mutations specific to the populations studied and revealed the types of mutations contributing to inherited retinal dystrophies. Approximately 13 percent of the families displayed atypical or unexpected changes in the genome. Five of the family lineages had mutations in more than one gene in all affected individuals; one family carried mutations in different genes in different affected members and a de novo mutation was found in one patient that was not present in both parents.

An additional 8 percent of families had large changes in the structure of their genome causing the inherited retinal disease and the initial clinical diagnosis in four families was re-classified based on their genotype.

The authors said the new findings boost understanding of the distribution of IRD causative mutations in these three diverse populations, which will further understanding of disease variation and presentation. That, in turn, will help design more efficient genetic testing strategies and therapies applicable to global populations.

The research team was led by Radha Ayyagari, PhD, professor of ophthalmology and pathology, and Kelly A. Frazer, PhD, professor of pediatrics and director of the Institute for Genomic Medicine, both at UC San Diego School of Medicine; and S. Amer Riazuddin, PhD, associate professor of ophthalmology at John Hopkins University, in collaboration with institutions in India, Mexico, Canada, Brazil, Pakistan and the United States.

Co-authors include: Pooja Biswas, UC San Diego and REVA University, India; Adda L. Villanueva, Retina and Genomics Institute, Mexico and Hpital Maisonneuve Rosemont, Canada; Angel Soto-Hermida, Hiroko Matsui, Shyamanga Borooah, Berzhan Kumarov, Bonnie Huang, John Suk, Jason Zhao, Sindhu Devalaraja, Andrew Huynh, Akhila Alapati and Qais Zawaydeh, UC San Diego; Jacque L. Duncan, UCSF; Gabriele Richard, GeneDx; Shahid Yar Khan, Johns Hopkins University School of Medicine; Kari Branham, Naheed W. Khan and John R Heckenlively, University of Michigan; Benjamin Bakall, University of Arizona; Jeffrey L. Goldberg, Byers Eye Institute; Luis Gabriel, Genetics and Ophthalmology, Genelabor, Brazil; Pongali B Raghavendra, REVA University and Manipal University, Brazil India; Richard G Weleber, Oregon Health & Science University; J. Fielding Hejtmancik, National Institutes of Health; Sheikh Riazuddin, University of Punjab and Allama Iqbal Medical College, Pakistan; and Paul A. Sieving, National Eye Institute and UC Davis.

Funding for this research came, in part, from the National Institutes of Health (grants EY031663, EY13198, EY21237, EY002162 and P30EY022589) the Foundation Fighting Blindness; Research to Prevent Blindness; The Claire Giannini Foundation; The L.L. Hillblom Foundation and That Man May See, Inc.

Link:
Genomic Study Revealing Among Diverse Populations with Inherited Retinal Disease - UC San Diego Health

image:ALRs are essential for the patients response to radiation and chemotherapy: A schematic depiction of how by impeding the repair of DNA breaks, ALRs enhance the killing effects of radiation and chemotherapy. Schematic prepared by Hui Jiang and Nelson Gekara. view more

Credit: Schematic prepared by Hui Jiang and Nelson Gekara.

Researchers at Stockholm University have identified key genetic factors important for the efficacy of radiation and chemotherapy. These proteins called AIM2-like receptors (ALRs) are possible biomarkers for predicting the patients response to treatment and could be targeted to achieve optimal outcome radiation and chemotherapy.

Radiation and chemotherapy are not only the most common treatments for cancer but also essential preparative procedures for bone marrow transplantation. These treatments are however highly unspecific and often cause severe harm to the patients. The outcomes of radiation and chemotherapy varies from individual to individual, mostly due to inherent genetic differences in patients. This makes it difficult to prescribe an optimal patientspecific dose. Hence, the fixeddose prescribed for the majority is limited because of the severe toxicity witnessed in a minority of individuals when higher doses are used. The molecular determinants of patient response to radiation and chemotherapy remain unclear. Understanding these is key for optimal individualized treatments and is required for the development of therapies against tissue injury caused by these treatments, explains Dr. Nelson Gekara, Stockholm University, the senior investigator of the study published in Advanced Science.

The bone marrow is the most radiosensitive tissue and bone marrow failure is the major cause of suffering and death upon exposure to irradiation.

When we exposed mice deficient in ALRs to a high dose of irradiation, we found that such mice were resistant, and their bone marrows were less affected compared the control mice. We also found that cancer cells deficient in ALRs were resistant but when genetically engineered to express ALRs, such cells became sensitive to the killing effect of irradiation and chemotherapy. Notably, our analysis of cancer patient data revealed that patients with lower levels of ALRs had poorer survival following chemotherapy, describes Dr. Hui Jiang a research engineer at Stockholm University and the first author of the study.

ALRs were originally identified as proteins that become highly expressed under inflammatory conditions, such as during viral infections. Until now ALRs were thought to act as innate immune receptors that detect the presence of foreign or misplaced DNA inside cells and initiate inflammation. Here, the authors found that the ability of ALRs to potentiate the effects of radiation and chemotherapy is independent of their classical function in innate immune activation.

The most toxic effect of radiation or chemotherapy is linked to their ability to damage the genome. DNA damage often results in cell death, or if incorrectly repaired, causes genetic alterations that may lead to cancer or hereditary disorders. The authors found that when exposed to irradiation or chemotherapy, mouse and human cells deficient in ALRs repair DNA breaks faster, indicating that ALRs are inhibitors of DNA repair hence accelerate the killing effects of radiation and chemotherapy.

How do the ALRs inhibit the repair of the genome? The estimated length of DNA in a single mammalian cell is about 2 meters. In order to accommodate it within the nucleus of an average size of 6 m, DNA is folded and densely packed within the chromatin. However, when the genome incurs damage, the chromatin undergoes decompaction to allow DNA repair factors to be recruited to damaged sites.

We show that ALRs are in fact chromatin-bound proteins and that by virtue of their ability to undergo self-self interaction, these proteins hinder chromatin decompaction vital for the efficient repair of DNA breaks, explains Dr. Jiang.

The discovery of ALRs as key drivers of host response to radiation and chemotherapy is significant and could lead to more optimized treatment. For example, devising safe means to modulate the expression of ALRs would be one way of manipulating the patients response to achieve the desired therapeutic effects of radiation and chemotherapy. Further, determining the expression level of these proteins could be used to predict how the patients are likely to respond and therefore help in adjusting the treatment to suit the specific patient, concludes Dr. Gekara.

About the study:Nuclear AIM2-Like Receptors Drive Genotoxic Tissue Injury by Inhibiting DNA Repair by Hui Jiang, Patrycja Swacha, and Nelson O. Gekara was published in Advanced Science, October 2021, DOI: 10.1002/advs.202102534

For more information, please contactNelson Gekara, Department of Molecular Biosciences, The Wenner-Gren Institute, Stockholm University. Phone: +46 8-16 41 89, e-mail: nelson.gekara@su.se

Experimental study

Animals

Nuclear AIM2-Like Receptors Drive Genotoxic Tissue Injury by Inhibiting DNA Repair

18-Oct-2021

The authors declare no conflict of interest.

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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The DNA sensors ALRs regulate genome repair and are targets for improve radiation and chemotherapy - EurekAlert

LEXINGTON, Mass., Oct. 18, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company, today announced clinical trial results demonstrating the first-ever in vivo genome editing in children. Early data from the company's Phase 1/2 SUNRISE clinical trial showed measurable levels of albumin-2A, a technology-related biomarker indicating site-specific gene insertion and protein expression. The SUNRISE trial is evaluating the safety, tolerability and preliminary efficacy of LB-001, the company's investigational, single-administration genome editing therapy, in pediatric patients with methylmalonic acidemia (MMA).

These results follow a recommendation from the independent Data Safety Monitoring Board (DSMB) overseeing the SUNRISE trial to continue the study without modification. The DSMB's recommendation was based on an evaluation of the safety data from the first two patients enrolled in the trial. Per the FDA-cleared protocol, albumin-2A detection together with the DSMB continuation recommendation enables LogicBio to begin enrolling two patients in the higher dose (1 x 1014 vg/kg) cohort (with ages ranging three to twelve years old) and two patients in the lower age (six months to two years old) cohort at the lower dose (5 x 1013 vg/kg) of LB-001.

"We are very excited to have achieved this significant milestone in the field of genetic medicine," said Fred Chereau, president and chief executive officer of LogicBio. "These early data indicate that we can precisely edit hepatocytes in vivo to treat a genetic liver disease with a single intravenous infusion using our proprietary GeneRide technology. Today's announcement is a demonstration that homologous recombination genome editing without the use of nucleases is a potential alternative to genome editing technologies in development that use nucleases, such as CRISPR. The ability to insert the correct version of a gene in a cell's genome without nucleases is an important step to unlocking the potential of GeneRide to treat a larger number of genetic diseases."

SUNRISE is a first-in-human, open-label, multi-center, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with MMA. LB-001 is designed to non-disruptively insert a corrective copy of the MMUT gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver. LB-001 is based on the company's proprietary GeneRide technology, which uses homologous recombination, a natural DNA repair process, to enable precise editing of the genome without the need for exogenous nucleases and promoters that have been associated with an increased risk of immune response and cancer.

"MMA is a rare, life-threatening genetic disorder for which there are no treatments addressing the underlying cause of the disease. By demonstrating for the first time ever that in vivo, nuclease-free genome editing in pediatric patients is achievable, we are one step closer to bringing a safe and effective genetic medicine to children suffering from MMA and, potentially, other early onset genetic diseases where early intervention is critical to achieve optimal health outcomes," said Daniel Gruskin, MD, chief medical officer of LogicBio. "I would like to thank the patients, their families and the investigators who are participating in this landmark trial. We look forward to continuing to progress the clinical study to better understand the biochemical and clinical effect of this genome editing therapy."

The Company remains on track to present additional interim data by the end of 2021.

About the SUNRISE Trial

The SUNRISE trial is an open-label, multi-center, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with methylmalonic acidemia (MMA) characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. Seven leading centers in the United States and one in Saudi Arabia are expected to participate in the trial. With the aim of evaluating LB-001 at an early age, the SUNRISE trial initially enrolled 3-12 year old patients and, following a recommendation from the trial's independent Data Safety Monitoring Board and detection of a biomarker indicating site-specific gene insertion, is permitted to enroll infants as young as 6 months old. The SUNRISE trial is designed to enroll up to 8 patients and evaluate a single administration of LB-001 at two dose levels.

About LB-001

LB-001 is an investigational, first-in-class, single-administration, genome editing therapy for early intervention in methylmalonic acidemia (MMA) using LogicBio's proprietary GeneRide drug development platform. GeneRide technology utilizes a natural DNA repair process called homologous recombination that enables precise editing of the genome without the need for exogenous nucleases and promoters that have been associated with an increased risk of immune response and cancer. LB-001 is designed to non-disruptively insert a corrective copy of the methylmalonyl-CoA mutase (MMUT) gene into the albumin locus to drive lifelong therapeutic levels of MMUT expression in the liver, the main site of MMUT expression and activity. LB-001 is delivered to hepatocytes intravenously via liver-targeted, engineered recombinant adeno-associated virus vector (rAAV-LK03). Preclinical studies found that LB-001 was safe and demonstrated transduction of hepatocytes, site-specific genomic integration, and transgene expression. LB-001corrected hepatocytes in a mouse model of MMA demonstrated preferential survival and expansion (selective advantage), thus contributing to a progressive increase in hepatic MMUT expression over time. LB-001 resulted in improved growth, metabolic stability, and survival in MMA mice. TheU.S. Food and Drug Administration(FDA) granted fast track designation, rare pediatric disease designation and orphan drug designation for LB-001 for the treatment of MMA. In addition, theEuropean Medicines Agency(EMA) granted orphan drug designation for LB-001 for the treatment of MMA.

About Methylmalonic Acidemia (MMA)

Methylmalonic acidemia (MMA) is a rare and life-threatening genetic disorder affecting approximately 1 in 50,000 newborns in the United States. In the most common form of MMA, a mutation in a gene called methylmalonyl-CoA mutase (MMUT) prevents the body from properly processing certain fats and proteins. As a result, toxic metabolites accumulate in the liver, in muscle tissue and in the brain. Symptoms include vomiting, lethargy, seizures, developmental delays and organ damage. There is no approved medical therapy addressing the underlying cause of the disease. To manage the symptoms, patients go on a severely restrictive, low-protein, high-calorie diet, often through a feeding tube. Even with aggressive management, these patients often experience life-threatening metabolic crises that can require recurrent hospitalizations and cause permanent neurocognitive damage. Because of this risk for irreversible damage, early intervention is critical and newborns are screened for MMA in every state in the United States.

AboutLogicBio Therapeutics

LogicBio Therapeuticsis a clinical-stage genetic medicine company pioneering genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. The Company's genome editing platform, GeneRide, is a new approach to precise gene insertion harnessing a cell's natural DNA repair process potentially leading to durable therapeutic protein expression levels. The Company's gene delivery platform, sAAVy, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues. The Company is based inLexington, MA.For more information, visitwww.logicbio.com, which does not form a part of this release.

Forward-Looking Statements

Statements in this press release regarding LogicBio's strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding early clinical results and the significance and interpretation thereof; homologous recombination genome editing without the use of nucleases as a potential alternative to genome editing technologies in development that use nucleases, such as CRISPR; the potential of the GeneRide platform, including the potential for genetic medicines based on the platform to be treatment options for genetic diseases; progressing the SUNRISE trial; the expected timing of announcing additional interim clinical data in the SUNRISE trial; the potential benefits of LB-001; and the sites expected to participate in the SUNRISE trial. The terms "demonstrating," "indicate," "look forward," "on track," "potential" and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the risk that existing preclinical and clinical data, including early clinical data from a trial, may not be predictive of the results of ongoing or later clinical trials; the risk that clinical trials may not be successful or may be discontinued or delayed for any reason; the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators and vendors operate; manufacturing risks; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize product candidates, including for safety, tolerability, enrollment, manufacturing or economic reasons; the timing and content of decisions made by regulatory authorities; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape; changes in the economic and financial conditions of LogicBio; and LogicBio's ability to obtain, maintain and enforce patent and other intellectual property protection for LB-001 and any other product candidates. Other risks and uncertainties include those identified under the heading "Risk Factors" in LogicBio's Annual Report on Form 10-K for the year ended December 31, 2020 and other filings that LogicBio may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and LogicBio does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Investor Contacts:

Laurence Watts Gilmartin Group (619) 916-7620 [emailprotected]

Stephen Jasper Gilmartin Group (858) 525-2047 [emailprotected]

Media Contacts:

Jenna Urban Berry & Company Public Relations W: 212-253-8881 C: 203-218-9180 [emailprotected]

Bill Berry Berry & Company Public Relations W: 212-253-8881 C: 917-846-3862 [emailprotected]

SOURCE LogicBio Therapeutics, Inc.

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LogicBio Therapeutics Announces Early Clinical Trial Results Demonstrating First-Ever In Vivo Genome Editing in Children - PRNewswire

NEW YORK, Oct. 19, 2021 (GLOBE NEWSWIRE) -- Cellectis S.A. (NASDAQ: CLLS EURONEXT GROWTH: ALCLS) (the Company), a gene-editing company with clinical-stage immuno-oncology programs using allogeneic chimeric antigen receptor (CAR)-T cells and gene therapy programs for genetic diseases, in collaboration with Professor Toni Cathomen, scientific director at the Center for Chronic Immunodeficiency Medical Center at the University of Freiburg, Germany, will present two oral presentations at the European Society of Gene and Cell Therapy (ESGCT) Congress to be held virtually from October 19-22, 2021.

Professor Cathomens team at University of Freiburg will be presenting encouraging pre-clinical data that supports further evaluation of Cellectis .HEAL platform, an innovative gene therapy platform that uses a genome editing approach based on TALEN , for two product candidates targeting primary immunodeficiencies: RAG1 for Severe Combined Immunodeficiency (SCID) and STAT3 for Hyper IgE syndrome.

The data accepted for presentation at ESGCT reflects our ongoing commitment to finding new ways to treat and potentially provide a cure to patients that have failed to respond to standard therapies. Utilizing Cellectis TALEN technology, which we believe to be the most precise, versatile, and effective gene editing tool currently available, we demonstrate our potential to precisely correct RAG1 and STAT3 deficient genes and restore functionalities of the gene. These new milestones bring us one step closer to our goal of unlocking the full potential of our gene editing platform and bringing new therapies to patients with unmet medical needs. said Philippe Duchateau, Ph.D, Chief Scientific Officer of Cellectis.

Last May, during Cellectis Innovation Days, the Company revealed its new .HEAL platform, a novel hematopoietic stem cell gene therapy that aims to address debilitating genetic diseases. .HEAL leverages the power of TALEN gene editing technology to perform genome surgery, resulting in highly efficient and precise gene inactivation, insertion, and correction in hematopoietic stem cells (HSCs). Cellectis has announced ongoing programs targeting sickle cell disease, lysosomal storage disorders and primary immunodeficiencies.

Story continues

Presentations details

Data presentation on preclinical development of a TALEN based genome editing therapy for RAG1 deficiency

Newborns with RAG1 SCID have extremely low levels of B and T cells and a severe risk of recurrent, life-threatening infections. RAG1 is an essential enzyme specifically and temporarily expressed in the early development of T and B cells, making traditional gene therapy approaches to treat the disease challenging due to the need for tight and precise spatio-temporal expression control.

Previous attempts to treat the RAG1 deficiency via conventional gene therapy have produced unsatisfactory results.

These results highlight the need for tight spatio-temporal control of RAG1 expression as key for functional restoration and the use of a gene editing tool.

Using Cellectis TALEN technology and .HEAL, Professor Cathomen engineered HSCs with a corrected copy of RAG1 that replaced the existing, mutated copy of RAG1. The precise replacement of the mutated gene enabled the corrected RAG1 gene to be expressed at its natural timing and stage of cell development.

30% of gene correction was achieved within the long-term HSC population.

The presentation titled Preclinical development of a TALEN based genome editing therapy for RAG1 deficiency will be made on October 21 (9-11AM CET) by Manuel Rhiel, Ph.D University of Freiburg, and can be found on the ESGCT website.

Presentation Details:

Data presentation on a preclinical development of a TALEN based genome editing in T-cells for the treatment of Hyper-IgE- Syndrome

Hyper IgE syndrome is a rare primary immunodeficiency disease that clinically manifests as skin inflammation and recurrent skin and lung infections. Mutations in the transcription factor STAT3 have been associated with Hyper IgE. Alternative splicing gives rise to two STAT3 isoforms, STAT3 and STAT3 that display distinct functions.

The / ratio needs to be tightly regulated, which represents a major challenge for traditional gene therapy approaches.

Cellectis has developed a strategy applicable in HSCs and T-cells to insert a corrected version of the STAT3 gene into the patients genome to restore its functionality.

In T-cells isolated from patients, 60% integration was achieved. More importantly, the / isoforms ratio was restored.

The presentation titled Preclinical development of a TALEN based genome editing in T cells for the treatment of Hyper-IgE-Syndrome' will be made on October 20 (9-11AM CET) by Viviane Dettmer, Ph.D, University of Freiburg, and can be found on the ESGCT website.

About Cellectis Cellectis is a gene editing company, developing first of its kind therapeutic products. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 21 years of expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. As part of its commitment to a cure, Cellectis remains dedicated to its goal of providing lifesaving UCART product candidates for multiple cancers including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) and multiple myeloma (MM). .HEAL is a new platform focusing on hemopoietic stem cells to treat blood disorders, immunodeficiencies and lysosomial storage diseases. Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).

For more information, visit http://www.cellectis.com Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

For further information, please contact:

Media contacts: Margaret Gandolfo, Senior Manager, Communications, +1 (646) 628 0300 Pascalyne Wilson, Director, Communications, +33776991433, media@cellectis.com

Investor Relation contact: Eric Dutang, Chief Financial Officer, +1 (646) 630 1748, investor@cellectis.com

Forward-looking Statements

This presentation contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as at this time, anticipate, believe, expect, on track, plan, scheduled, and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management, include statements about our research and development projects and priorities, our pre-clinical project development efforts and the timing of our presentation of data. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development as well as the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2020 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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Cellectis Presents Initial Preclinical Data on Two Novel Gene Therapies for Patients with RAG1 Severe Combined Immunodeficiency (SCID) and Hyper IgE...

This article has been sponsored by Intel.

Every successful startup or business begins with an idea that has the potential to inspire and create impact. But aside from the eureka moment, the idea alone cannot drive the impact it aims. Understandably, it takes countless days, months or even years of work to translate it into something substantial.

In addition to the big idea, the methodology involved to create a tangible product is what sets the stage for progress. And, in this situation technology becomes a prominent enabler.

Time and again through numerous startup success stories, we have been reminded of the transformational power of technology. When leveraged in the optimal way, it can bridge the gap between availability and accessibility with an efficiency like no other.

In this sphere, Intel has been facilitating this change as a pioneer in disruptive technologies. From its AI-driven technology solutions that are employed in a variety of sectors like security, education and healthcare to its mentorship program called Intel Startup Program, the company has been paving the way for tech startups and businesses who are about to change the world for the better.

Intels technical assistance, mentorship and high-impact collaboration with the industry, academia and government, has helped several companies/individuals realise their maximum potential and here are some of those:

Motivated to help a friend, Jagadish K Mahendran and his team designed a unique AI-powered voice-activated visual assistance system that could bridge the gap of accessibility and help the visually impaired perceive the world around them efficiently.

Equipped with OpenCVs Artificial Intelligence Kit with Depth (OAK-D) and powered by Intel, this innovation comes in a backpack that helps the wearer effectively navigate and detect common accessibility challenges like, changing elevations, traffic signs, moving objects, crosswalks, etc.

The backpack comes with a host computing unit that looks like a laptop, a Luxonis OAK-D spatial AI camera and a pocket-size battery pack with a capacity to power the device for at least 8 hours. The wearer can easily navigate with the help of this interactive device by connecting it with a pair of Bluetooth-enabled earphones. Through voice commands and responses, the system continuously guides the wearer through the journey.

This innovation can potentially help more than 285 million visually-impaired individuals across the globe.

The technology exists, we are only limited by the imagination of the developer community. Its incredible to see a developer take Intels AI technology to the edge and quickly build a solution to make their friends life easier, says Hema Chamraj, director, Technology Advocacy and AI4Good at Intel.

Dr Tinku Acharya started Videonetics in 2008, with a vision to create a homegrown Unified Video Computing platform that can revolutionise security and surveillance across the globe. Today, almost 12 years later, this company has used the same technology to aid in securing public health and safety during the pandemic.

To do so, their technologists developed SAJAG, an innovative video analytics-based Pandemic Management Suite that enables 247 monitoring of cities, hotspots, IT parks, office complexes, educational institutions, hospitals, etc. Operating in collaboration with several private and public stakeholders, SAJAG monitors a vast array of COVID relevant parameters and ensures the detection of anomalies with accuracy.

From ensuring compliance with all the government lockdown guidelines to monitoring social distancing to maintaining law and order it has played a pivotal role in helping authorities tackle the pandemic, says Avinash Trivedi, VP Business Development of Videonetics.

An ISV (Independent Software Vendor) partner of Intel, Videonetics has been leveraging technical insights, and computing infrastructure powered by Intel Xeon Scalable Processors and Intel Core i7 processors through the collaboration with the technology giant since its inception. SAJAG is the most recent example of this collaboration.

Much before COVID-19, Assams rural population was battling a deadly health condition hypertension. More than 50% of the hypertension cases in Assam lead to hemorrhagic strokes, as compared to a 20% margin in the rest of the country.

As a solution to this problem, Mumbai-based artificial intelligence (AI) startup, Qure.ai came forward during the pandemic with an innovation that sided in efficient management and care of stroke victims. They deployed an FDA-approved, CE-certified software qER, which can detect 12 critical abnormalities including strokes, clots and fractures. They also developed qXR, an AI-driven solution, which employs deep learning to provide comprehensive Chest X-ray screening to assist in the identification, treatment and management of asymptomatic COVID-19 symptoms in patients. With this technology in place doctors can now detect abnormalities like intracranial hemorrhage, atrophy, hydrocephalus, etc within just 1-2 minutes of a CT scan.

The most critical impact of this technology can be felt in the reduction of the time taken for diagnosis during emergencies like a stroke or trauma even in the absence of a specialist. The AI tech accurately highlights the need for intervention in critical areas and provides physicians with all the necessary information to make crucial decisions in time, adds Dr Pooja Rao, Qure.ais co-founder.

Working towards empowering the healthcare sector, the founders share that they were able to amass this impact with Intels help. With its cloud servers based on Intel processors and its x86 technology, they add that Qure.ai was able to successfully install its AI gateway on even lower-end basic computers because of Intels technology.

Boston-based social entrepreneur and cognitive scientist of Indian origin, Venkat Srinivasan along with Sanjay Gupta started English Helper, a tech-based learning solution for students in government schools. In 2013, they launched an initiative called RightToRead that introduced a multisensory reading and comprehension software that helps read out English textbooks using Artificial Intelligence.

Even before we start to speak, our brain begins to associate and relate everyday objects and situations with words, through sound and sight. It is through this visual and aural exposure that language learning develops into the spoken and written form making the multisensory approach the ideal one, says Vineet Mehra, Vice President and Chief Operating Officer (K12) of English Helper.

Available in three formsthe ReadToMe School Edition, ReadToMe Virtual Classroom and ReadToMe Student Edition softwaresthis innovation has helped bridge the gap in education, even during the pandemic. From a few hundred schools, today they have expanded to thousands of schools impacting millions of students. Utilising key capabilities of AWS instances powered by Intel Xeon Processors, they have managed to deploy the RightToRead program seamlessly across 28,000 schools in urban and rural India.

Started in 2017, Vacus Tech developed a unique technology that ensures a comprehensive and accurate monitoring in public places like offices, educational institutions and hospitals. Specialising in indoor positioning and tracking technology for safety purposes, Vacus Tech has leveraged its technology to ensure accurate monitoring of social distancing and following of COVID-19 protocols while indoors.Prior to the pandemic, our focus was to create smart buildings and workplaces that ensured safety and security of the employees working inside. But when the pandemic struck, a lot of automobile companies reached out to help track factory workers with respect to the following of COVID protocols, says co-founder Pratik Magar, who spent two years to build this technology along with co-founder Venugopal.Installed in employee ID cards, this innovation has not only encouraged employees to follow COVID guidelines at all times, but also simplified contact tracing in case of infection.

We participated in this startup accelerator programme before COVID. The platform helped us finalise our architecture and scale up the company. At the time we were able to produce only 3,000 to 4,000 tags and to scale beyond 10,000 tags we needed an efficient gateway that could handle such a big load. Thats when Intel helped us further with the AAEON UP Squared developer board. One of the fastest, this also comes with several security options that allow us to encrypt the data received, adds Pratik. With Intels help, they managed to source the gateway UP Squared board in record time, despite challenges posed by the lockdown.

India has the largest population of diabetic patients and a projection suggests it will hit almost 98 million people by 2030. An outcome of this is a condition called diabetic retinopathy, which is a leading cause of blindness and loss of vision in adults. Although the damage done due to diabetic retinopathy is serious and irreversible, early diagnosis and treatment can prevent loss of vision.Realising the potential need for a reliable solution, Sankara Eye Foundation, Leben Care and Intel technologies came together to develop a powerful innovative device called NETRA.AI that uses deep learning technology to efficiently diagnose retinal conditions, in less than a minute.NETRA.AI is built on Amazon EC2 C5 and M5 instances and powered by Intel Xeon Scalable processor. It also leverages Intels AI, DL Boost and Vector Neural Network to ensure accurate, accessible and affordable AI detection of retinal illnesses, to a large population, even with limited infrastructure, resources or an overburdened healthcare system. With the help of this technical backing, NETRA.AI has screened 3,293 patients and identified 812 at-risk patients so far.

In order to understand virus transmission and tackle the COVID-19 pandemic better, a healthcare startup called HaystackAnalytics devised a unique genome sequencing dashboard that was incubated by Indian Institute of Technology (IIT), Bombay and deployed for Brihanmumbai Municipal Corporation (BMC) in June 2021.Incubated under the Society for Innovation and Entrepreneurship (SINE) at IIT-B, Haystack created an analytical tool that aids public health officials and epidemiologists, better understand data that is retrieved from genome sequencing of ribonucleic acid (RNA) samples collected from Covid-19 patients.

Divided in four prominent stepssample processing, creation of a DNA library, sequencing the data from the genomes and analysing datathe process of genome sequencing is usually long and tedious.

Generally, it takes between 72 hours to 15 days to get the complete results of genome sequencing. Powered by Intel Xeon processors, Haystack is now able to complete the entire process and get results in less than 36 hours, with the help of assistance provided at the Intel Startup Program.

With Intels assistance we were able to reduce the turnaround time considerably. Initially when we started our analysis time would take around an hour but when we finished the programme, we were able to bring it down to 15 minutes. Essentially, through this technology, we are reducing a patients diagnostic journey and the cost. For instance, to identify a condition, instead of having to do multiple tests and therapies, the patient is now able to find the most likely cause and solution in a matter of just a few hours, says co-founder Anirvan Chatterjee.

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From Genome Sequencing to Helping the Visually-Impaired: 7 AI Innovations Changing Lives - The Better India

An extinct Japanese wolf appears to be the closest known wild relative of dogs, New Scientist reports.

It notes that the Japanese wolf, Canis lupus hodophilax, a subspecies of the gray wolf, went extinct in the early 1900s but that there are a number of museum specimens that researchers led by Yohey Terai at the Graduate University for Advanced Studies in Japan studied. As they report in a preprint posted to BioRxiv, the researchers analyzed the whole genomes of nine Japanese wolves and 11 Japanese dogs to find that Japanese wolves are the closest among gray wolves to dogs.

The Eurasian gray wolf lineage and the dog lineage split about 20,000 to 40,000 years ago, but the researchers uncovered some introgression from the ancestor of the Japanese wolves into the ancestor of East Eurasian dogs that occurred about 10,000 years ago.

Terai tells New Scientist that even if the dog ancestor lived in East Asia, that does not necessarily mean dogs were domesticated there. "It is not possible to determine when the dogs began to have a relationship with humans from the genome data," Terai tells it. New Scientist notes that archaeological evidence is needed to make that determination.

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Closest to the Dog | Genomeweb - GenomeWeb