Daily Archives: September 16, 2021

In January, his doctor, Alexis Thompson of Northwestern University, told him he no longer had sickle cell disease.

It is strange, Mr. Hubbard said, to think he has a future.

I am becoming more serious about life, he said. I didnt think I would have a life.

Helen, too, has been adjusting to life as a healthy person an alteration her pediatric hematologist, Dr. Alexander Ngwube of Phoenix Childrens Hospital, has observed in sickle cell patients cured with bone-marrow transplants.

Remember with sickle cell disease they are hospitalized a lot, he said. There are so many restrictions on their lives. They become depressed and when they are a little older they realize they have a life expectancy of 40 years. They start to think, What is the point of doing anything?

When they are cured, he said, it is almost like the world is theirs to play with.

When Dr. Esrick gave Helen the good news a year ago, Helen lay silent on the exam table, not daring to talk.

Helen and her family have since moved from Lawrence, Mass., to Mesa, Ariz. Ive followed her progress for more than two years. A lifetime of stoicism had taught her to keep her emotions to herself. Through a harrowing monthlong stay in the hospital for the gene therapy, she barely spoke to me. Even after it was completed, she tapped her mother on the arm and pointed when she wanted an ice cream cone as we walked through the Faneuil Hall Marketplace in Boston.

Freed of illness, shes become far more outgoing. On summer weekends, shes ventured to an arcade and a water park, and gone tubing.

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Pioneering Gene Therapy Freed Her of Sickle Cell. Is a Cure at Hand? - The New York Times

"Our center is home to dozens of basic science and translational researchers who investigate the biological pathways and neurological mechanisms that underlie a range of intellectual and developmental disabilities," said Sophie Molholm, Ph.D., co-primary investigator on the grant and co-director of the RFK IDDRC. "But ultimately, our sights are set on helping the children with IDDs in the Bronx and empowering their families and caregivers, a goal this new grant will help us achieve," added Dr. Molholm, who is professor of pediatrics, in the Dominick P. Purpura Department of Neuroscience, and of psychiatry and behavioral sciences at Einstein.

Investigating Gene Mutations

Previous NIH support helped establish a research program on22q11.2 Deletion Syndrome (22q11.2DS), an incurable genetic disorder associated with delayed intellectual development and psychiatric conditions. Thisnew grant's research focus involves the X chromosome'sKDM5Cgene, which plays a central role in brain development and behavior. Mutationsin theKDM5Cgene lead to intellectual disabilities and other conditions, particularly in males although females can also be affected.

In 2020, the IDDRC's annual Rare Disease Day event featured a special program in which 12 families with children who have a KDM5C variant came together from around the country and from England to meet for the first time. They learned about recent findings and the RFK IDDRCpartnerships at Montefiore and Einstein that are addressing research and care. Hayden Hatch, an M.D./Ph.D. student at Einstein, spearheaded the effort.

Julie Secombe, Ph.D., professor of genetics and in the Dominick P. Purpura Department of Neuroscience, will lead basic science studies on KDM5C, along with Bryen Jordan, Ph.D., associate professor in the Dominick P. Purpura Department of Neuroscience and associate professor of psychiatry and behavioral sciences. The translational and clinical aspects of the work will be led by Dr. Molholm and Lisa Shulman, M.D., professor of pediatrics at Einstein, interim director of the Rose F. Kennedy Children's Evaluation and Rehabilitation Center (CERC) at the Children's Hospital at Montefiore, and a developmental pediatrician at Montefiore.

Advancing IDD Research and Collaboration

Einstein is one of 15 IDDRCs funded by the NIH and was among the first such centers established in the 1960s. More than 100 researchers study neurodevelopmental conditions including autism spectrum disorders, attention-deficit hyperactivity disorder, Rett and Williams syndromes, Niemann-Pick and other lysosomal storage diseases, neurocutaneous disorders, and infantile and childhood seizures. The RFK IDDRC also has more than 20 clinical partners in neurology and pediatrics.

"We're in the unique position of having many different IDD-focused programs under one roof," said Steven Walkley, D.V.M, Ph.D., co-director of the IDDRC, co-primary investigatoron the grant,professor in the Dominick P. Purpura Department of Neuroscience, of pathology, and in theSaul R. Korey Department of Neurology."In addition to the IDDRC, the Rose F. Kennedy Center includesCERC, which is part of our University Center for Excellence in Developmental Disabilities; the NIH-funded healthcare professional training program known as LEND(Leadership Education in Neurodevelopmental and Related Disabilities); and a postdoctoral fellowship training program. We're truly at the forefront of patient-oriented science both for the Bronx community and beyond."

The new grant also funds the center's four interdisciplinary scientific cores, which support biomedical, clinical, and translational research on IDDs. The cores include resources for clinical phenotyping, epigenetic and genomic analyses, and neural cell engineering and imaging.

The grant, titled "Support for the Rose F. Kennedy IDDRC P50," is funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, part of the National Institutes of Health (1 P50 HD105352-01).

SOURCE Albert Einstein College of Medicine

http://www.einsteinmed.org

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Albert Einstein College of Medicine Awarded $5 Million for Research on Intellectual and Developmental Disabilities - PRNewswire

DUBLIN, Sept. 14, 2021 /PRNewswire/ -- The "Induced Pluripotent Stem Cell (iPSC) Global Market Opportunities and Strategies to 2030: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

This report provides the strategists, marketers and senior management with the critical information they need to assess the global induced pluripotent stem cell (iPSC) market as it emerges from the COVID-19 shut-down.

This report describes and explains the induced pluripotent stem cell (iPSC) market and covers 2015 to 2020, termed the historic period, and 2020 to 2025 termed the forecast period, along with further forecasts for the period 2025-2030. The report evaluates the market across each region and for the major economies within each region.

The global induced pluripotent stem cell (iPSC) market reached a value of nearly $2,223.5 million in 2020, having increased at a compound annual growth rate (CAGR) of 9.2% since 2015. The market is expected to grow from $2,223.5 million in 2020 to $3,362.1 million in 2025 at a rate of 8.6%. The market is then expected to grow at a CAGR of 6.2% from 2025 and reach $4,547.7 million in 2030.

Growth in the historic period resulted from increased healthcare spending, rise in funding, aid as non - animal alternatives for preclinical trials and increasing demand of personalized medicines. Factor that negatively affected growth in the historic period was lack of awareness on induced pluripotent stem cell.

Going forward, increasing prevalence of chronic diseases, growth genomic projects, development in iPSC models, increasing funding and increasing prevalence of genetic diseases. Factors that could hinder the growth of the induced pluripotent stem cell (iPSC) market in the future include risk associated with it and high costs associated with storage.

The induced pluripotent stem cell (iPSC) market is segmented by application into drug discovery and toxicity studies, academic research, cell & gene therapy, and regenerative medicine. The drug discovery and toxicity studies was the largest segment of the induced pluripotent stem cell (iPSC) market segmented by application, accounting for 48.7% of the total in 2020. Going forward, cell & gene therapy segment is expected to be the fastest growing segment in the induced pluripotent stem cell (iPSC) market segmented by application, at a CAGR of 9.82% during 2020-2025.

The induced pluripotent stem cell (iPSC) market is also segmented by end-user into hospitals and research laboratories. The research laboratories was the largest segment of the induced pluripotent stem cell (iPSC) market segmented by end-user, accounting for 68.5% of the total in 2020. Going forward, the hospitals segment is expected to be the fastest growing segment in the induced pluripotent stem cell (iPSC) market segmented by end-user, at a CAGR of 9.3% during 2020-2025.

The induced pluripotent stem cell (iPSC) market is also segmented by derived cell type into amniotic, hepatocytes, fibroblasts, keratinocytes and others. The fibroblasts was the largest segment of the induced pluripotent stem cell (iPSC) market segmented by derived cell type, accounting for 33.0% of the total in 2020. Going forward, the others segment is expected to be the fastest growing segment in the induced pluripotent stem cell (iPSC) market segmented by derived cell type, at a CAGR of 10.0% during 2020-2025.

The induced pluripotent stem cell (iPSC) market is fragmented, with a large number of players constituting the market. The top ten competitors in the market made up to 26% of the total market in 2020. Major players in the market include Fujifilm Holding Corporation, Thermo Fisher Scientific Inc Takara Bio Inc., ViaCyte, and Fate Therapeutics.

The top opportunities in the induced pluripotent stem cell (iPSC) market segmented by derived cell type will arise in the fibroblasts segment, which will gain $360.1 million of global annual sales by 2025. The top opportunities in the induced pluripotent stem cell (iPSC) market segmented by end-user industry will arise in the hospitals segment, which will gain $393.0 million of global annual sales by 2025. The top opportunities in the induced pluripotent stem cell (iPSC) market segmented by application will arise in the drug discovery and toxicity studies segment, which will gain $520.3 million of global annual sales by 2025. The induced pluripotent stem cell (iPSC) market size will gain the most in the USA at $132.6 million.

Key Topics Covered:

1. Induced Pluripotent Stem Cell (iPSC) Executive Summary

2. Table of Contents

3. List of Figures

4. List of Tables

5. Report Structure

6. Introduction6.1. Segmentation By Geography6.2. Segmentation By Derived Cell Type6.3. Segmentation By Application6.4. Segmentation By End-Use Industry

7. Induced Pluripotent Stem Cell (iPSC) Market Characteristics7.1. Segmentation By Derived Cell Type7.1.1. Hepatocytes7.1.2. Fibroblasts7.1.3. Keratinocytes7.1.4. Amniotic7.1.5. Others7.2. Segmentation By Application7.2.1. Academic Research7.2.2. Drug Discovery Toxicity Studies7.2.3. Regenerative Medicine7.2.4. Gene & Cell Therapy7.3. Segmentation By End-User7.3.1. Hospitals7.3.2. Research Laboratories

8. Induced Pluripotent Stem Cell (iPSC) Trends Strategies8.1. Use of Pluripotent Stem Cells in treating Parkinson's Disease (PD)8.2. Strategic Collaborations Partnerships8.3. Development Of Induced Pluripotent Stem Cell iPSC-Derived NK Cells8.4. Demand For Induced Pluripotent Stem Cell (iPSC) For Cell Therapy8.5. Expansion and Improvements in Drug Research8.6. Use of Pluripotent Stem Cells in treating Type 1 Diabetes

9. Impact Of Covid-19 On Induced Pluripotent Stem Cell (iPSC) Market9.1. Impact On Demand For Induced Pluripotent Stem Cells9.2. Increase In Research Development Activities

10. Global Induced Pluripotent Stem Cell (iPSC) Size Growth10.1. Market Size10.2. Historic Market Growth, 2015 - 2020, Value ($ Million)10.2.1. Drivers Of The Market 2015 - 202010.2.2. Restraints On The Market 2015 - 202010.3. Forecast Market Growth, 2020 - 2025, 2030F Value ($ Million)10.3.1. Drivers Of The Market 2020 - 202510.3.2. Restraints On The Market 2020 - 2025

11. Global Induced Pluripotent Stem Cell (iPSC) Segmentation11.1. Global Induced Pluripotent Stem Cell (iPSC) Market, Segmentation By Derived Cell Type , Historic Forecast, 2015 - 2020, 2025F, 2030F, Value ($ Million)11.1.1. Fibroblasts11.1.2. Hepatocytes11.1.3. Keratinocytes11.1.4. Amniotic11.1.5. Others11.2. Global Induced Pluripotent Stem Cell (iPSC) Market, Segmentation By Application, Historic Forecast, 2015 - 2020, 2025F, 2030F, Value ($ Million)11.2.1. Drug Discovery & Toxicity Studies11.2.2. Academic Research11.2.3. Cell & Gene Therapy11.2.4. Regenerative Medicine11.3. Global Induced Pluripotent Stem Cell (iPSC) Market, Segmentation By End-Use Industry, Historic Forecast, 2015 - 2020, 2025F, 2030F, Value ($ Million)11.3.1. Research Laboratories11.3.2. Hospitals

Companies Mentioned

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Global Induced Pluripotent Stem Cell (iPSC) Market Report 2021: Focus on Drug Discovery & Toxicity Studies, Academic Research, Cell & Gene...

CAMBRIDGE, Mass., Sept. 14, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the World Muscle Society 2021 Virtual Congress (WMS 2021), taking place Sept. 20-24, 2021. This years presentations at WMS 2021 highlight scientific leadership and innovation from across Sareptas deep, multi-platform portfolio and reflect the Companys continued commitment advancing life-changing therapies to those with rare genetic diseases.

Presented research will include data from the Companys gene therapy and RNA platforms, in addition to new research into the prevalence of pre-existing antibodies to the AAVrh74 vector, which is used in several of Sareptas gene transfer therapy programs. All posters are available on-demand throughout the Congress beginning on Monday, Sept. 20, 2021 at 7:00 am E.T. The full WMS 2021 program is available at https://www.wms2021.com/page/programme.

Poster Presentations

Presentations will be archived on the events and presentations page in the Investor Relations section of http://www.sarepta.com for one year following their presentation at WMS 2021.

About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Showcase Data from its Gene Therapy - GlobeNewswire

Introduction

Chronic obstructive pulmonary disease (COPD) is a chronic respiratory disorder that progresses slowly and is characterized by an obstructive ventilatory pattern, which is rarely reversible. The main risk factors are active smoking, genetic factors, and air pollution. In particular, COPD has been a major public health problem and will remain a challenge for clinicians in the 21st century. In recent years, COPD has been the focus of pulmonary research given its high prevalence and increased morbidity and mortality rates, which pose formidable challenges for health-care systems. The projection for 2020 indicates that COPD will be the third leading cause of death worldwide, which is responsible for early mortality, high death rates, and significant cost to health systems.1,2

Kashi city is located to the west of XinJiang province, China, and is close to Taklamakan desert which is the second largest desert in the world. The desert climate and geographic location of Kashi area are favorable for the development of COPD, because people living in this area are frequently exposed to dust storms. In these conditions, the prevalence rates of COPD increase yearly, and the Uyghur population appear most affected with COPD in the Kashi area. Furthermore, we found that the increased incidence of COPD in this area is also related to genetic factors and customs of the local people. In this study, we investigated the unique polymorphic site in the exonic region of the SREK1 gene and discuss the relationship between the SREK1 gene SNP in the rs74794265 locus and the susceptibility to COPD among the Uyghur population in Kashi, XinJiang, China.

SREK1 is a member of the SR protein superfamily with an additional glutamic acid-lysine (EK)-rich domain that regulates RNA processing by modulating multiple SR proteins. The SR proteins play significant roles in constitutive and alternative splicing of pre-mRNAs as well as in nuclear export, nonsense-mediated decay, and translation of mRNAs.3 SREK1 is a direct interactor and regulator of SRSF6 and is particularly interesting because one of its target mRNAs is the TATA-box binding protein associated factor 1 (TAF1).4 A previous study showed that SRSF6 regulates alternative splicing of genes involved in DNA damage response and DNA repair in HeLa cells.5 However, thus far, no study has shown the correlation between SREK1 and COPD. Sanger sequencing showed that only one SNP of the SREK1 gene was founded and SREK1 gene polymorphism confers protection against COPD. The role of rs74794265 in the regulation of SREK1 expression levels and the mechanisms via which it influences COPD require further investigation.

A total of 1075 subjects were included in this study: 541 patients with COPD in the case group and 534 healthy people in the control group. COPD patients were diagnosed based on the results from multiple examinations including the ratio of forced expiratory volume in 1 second/forced vital capacity (FEV1/FVC ratio, 70%) and FEV1 (80% predicted), according to the Global Initiative for Chronic Obstructive Lung Disease criteria.6 All patients and controls were matched for age, sex, and BMI; all subjects underwent pulmonary function testing and showed normal spirometry results. The exclusion criteria for the control group were: 1) other respiratory diseases such as asthma, tuberculosis, fibrosis, or lung cancer; 2) suffering from any respiratory symptoms or non-respiratory diseases such as diabetes or cardiovascular disease; 3) unable to perform the pulmonary function tests.

All subjects were required to complete a questionnaire about basic information and provide 5 mL peripheral blood for testing. Written informed consent was obtained from all participating subjects. The project was approved by the Ethics Committee of the First Peoples Hospital of Kashi. This study was conducted in accordance with the tenets of the Declaration of Helsinki.

Data analyses were performed using SPSS software (version 18.0, IBM Corporation, Armonk, NY, USA). Quantitative data are presented as meanstandard deviation or medians (interquartile ranges). Independent t-test was used to compare the difference between age and BMI, because this data showed approximately normal distribution which was supported by the QQ plots. Annual household income, FEV1%, and FEV1/FVC not conforming to normal or approximately normal distribution were compared using MannWhitney U-test. Differences in sex, smoking status, smoking index, coal combustion (Burning coal for cooking and heating), and charcoal combustion (Burning charcoal for cooking and heating) were assessed by chi-square test (2). The SNP genotypic distribution in all subjects was evaluated by HardyWeinberg equilibrium (HWE), using the Fishers exact test. Unconditional logistic regression analysis was performed to identify independent risk factors of COPD in each SNP genetic model. Odds ratio (OR), 95% confidence intervals (CI), and stratified analysis were presented by multivariable logistic regression analysis before or after adjusting for age, sex, and BMI. For all analyses, P<0.05 was considered to indicate statistical significance. The relationship between the COPD risk and the tag-SNPs under different genotypic models was designed for analysis by SNP Stats.7

The characteristics of all objects including average age, BMI, smoking, and pulmonary function (FEV, FVC) were significantly different between the patient and control groups shown in Table 1.

Table 1 Characteristics of the Study Population

The multiple logistic regression model was adjusted for age, sex, and smoking history in all subjects, and the HWE analysis was applied. Results of the HWE analysis are shown in Table 2. After adjusting for age, sex, and BMI among COPD patients, all data were analyzed using unconditional logistic regression analysis. Table 3 shows the results of the logistic regression model wherein the SNP of the rs74794265 locus SREK1 gene included two genotypes of C/C and C/T respectively; In the Additive and Dominant genetic model, the risk of COPD was significantly lower in the case group with heterozygous C/T in rs74794265 [p=0.0236, OR=0.3677 (0.15470.8742)], and the gene frequency of T was also significantly lower in the allele model in the case group [p=0.0245, OR=0.3728 (0.15770.8811)]. We speculate that the heterozygous C/T genotype can confer further protection against COPD.

Table 2 HWE Analysis

Table 3 Analysis of Genotypes of Rs74794265

Smoking is considered the main risk factor of COPD in reports;8,9 therefore, we explored the SREK1 gene SNP in the rs74794265 locus in smokers to explore the correlation the rs74794265 SNP with smoking. We evaluated the covariates related with smoking, including annual household income, coal combustion and charcoal combustion (Table 1). There were no significant differences between the cases and controls. Interestingly, there was only one SNP frequency in the smokers with heterozygous C/T in rs74794265 (Table 4). There was no significant difference in smoking status between the case and control groups in any genetic model. Table 5 showed that among the no smokers, there were significant differences with heterozygous C/T in rs74794265 in all genetic models between the cases and controls, and the frequency of the T allele was also significantly lower in the Allele model in the case group than the control group. This indicates that the SNP of rs74794265 has no correlation with smoking status.

Table 4 SNP of Rs74794265 Among Smokers

Table 5 SNP of Rs74794265 in No Smokers

COPD is a lung disease characterized by incomplete reversible airflow limitation, which progressively develops and is associated with lung damage particles and gas abnormalities of the inflammatory response. Studies10 have indicated the existence of familial clustering of COPD. A twin study showed that a higher risk of COPD was found in identical twins than in fraternal twins, and 60% of individual risks were determined by genetic factors.11 Therefore, investigation of genetic factors may be beneficial for the early prevention and diagnosis of COPD.

In our current study, we investigated 1075 subjects belonging to the Uyghur population from the Kashi region and found that the unique polymorphic site of the SREK1 gene with heterozygous C/T in rs74794265 was apparently associated with reducing COPD risk in this population who have lived here for a long term. Moreover, reports point out that tobacco smoke exposure is the primary risk factor of COPD and 1015% of smokers finally develop COPD.8,9 Therefore, we compared the SNP of rs74794265 among non-smoking COPD patients and found that there was no correlation between rs74794265 and smoking status. This indicated that the occurrence of COPD in this area is most likely related to poor air condition and genetic factors than smoking because of the special geographical location of Kashi.

Several GWAS have reported that multiple SNPs in the CXCL10,12 ADRB2,13 HHIP,14 and SERPIN215 genes contribute to COPD susceptibility. However, association of the SNPs in the SREK1 gene with COPD is still poorly understood. Our findings first revealed the rs74794265 locus on SREK1 is obviously related to COPD susceptibility. As the major risk factor for COPD, the associations of cigarette smoking with SNPs variants were evaluated. Stratified analysis showed that the rs74794265 SNP was no significant difference in smoking status between the case and control groups in any genetic model. In addition, silicosis, which is a chronic progressive fibrotic lung inflammation, may increase the risk of COPD. Research indicates that the functional variant of the CPM gene16 and the rs2609255 polymorphism in the FAM13A gene17 are associated with silicosis susceptibility in a Chinese population. In our study, we discussed the susceptibility of SREK1 rs74794265 C>T polymorphism in COPD. However, whether the rs74794265 SNP was directly associated with COPD or through silicosis increase the risk of COPD, remains unclear.

Our study has some limitations. The number of subjects was relatively small and the effects of second-hand smoke or other smoke-inhalation methods in non-smoking individuals were not considered. In summary, to our knowledge, this is the first account of the beneficial association of the rs74794265 SNP in the SREK1 gene with COPD and in Uyghur population residing in the Kashi region.

The project was approved by the Ethics Committee of First Peoples Hospital of Kashi. This study was conducted in accordance with the tenets of the Declaration of Helsinki.

Zulipikaer Abudureheman, Li Li, and XueMei Zhong contributed equally to this work as primary authors (co-first authors). Aimin Xu and Xiaoguang Zou are co-corresponding authors.

Tianshan Youth Project of Xinjiang Uyghur Autonomous Region (2019Q143).

I would like to declare on behalf of my co-authors that our paper was submitted solely to this journal and the work described is original research that has not been published previously, and not under consideration for publication elsewhere, in whole or in part.

1. Lpez-Campos JL, Wan T, Soriano JB. Global burden of COPD. Respirology. 2016;21(1):1423.

2. Raherison C, Girodet PO. Epidemiology of COPD. Eur Respir Rev. 2009;18(114):213221.

3. Li J, Barnard DC, Patton JG. A unique glutamic acid-lysine (EK) domain acts as a splicing inhibitor. J Biol Chem. 2002;277(42):3948539492.

4. Hernndez IH, Cabrera JR, Santos-Galindo M, et al. Pathogenic SREK1 decrease in Huntingtons disease lowers TAF1 mimicking X-linked dystonia parkinsonism. Brain. 2020;143(7):22072219.

5. Xiao Y, Peng Z, Shuqiang F, et al. SRSF6 regulates alternative splicing of genes involved in DNA damage response and DNA repair in HeLa cells. Oncol Rep. 2020;44(5):18511862.

6. Vogelmeier CF, Criner GJ, Martinez FJ, et al. Global strategy for the diagnosis, management, and prevention of chronic obstructive lung disease 2017 report: GOLD executive summary. Eur Respir J. 2017;49(3):1700214.

7. Sol X, Guin E, Valls J, et al. SNPS tats: a web tool for the analysis of association studies. Bioinformatics. 2006;22(15):19281929.

8. Rabe KF, Watz H. Chronic obstructive pulmonary disease. Lancet. 2017;389(10082):19311940.

9. Lareau SC, Fahy B, Meek P, et al. Chronic Obstructive Pulmonary Disease (COPD). Am J Respir Crit Care Med. 2019;199(1):P1P2.

10. McCloskey SC, Patel BD, Hinchliffe SJ, et al. Siblings of patients with severe chronic obstructive pulmonary disease have a significant risk of airflow obstruction. Am J Respir Crit Care Med. 2001;164(8 Pt 1):14191424.

11. Ingebrigtsen T, Thomsen SF, Vestbo J, et al. Genetic influences on chronic obstructive pulmonary diseasea twin study. Respir Med. 2010;104(12):18901895.

12. Yan W, Qipeng Z, Lian D, et al. The effects of CXCL10 polymorphisms on COPD susceptibility. Mol Genet Genomics. 2018;293(3):649655.

13. Li JX, Fu WP, Zhang J, et al. A functional SNP upstream of the ADRB2 gene is associated with COPD. Int J Chron Obstruct Pulmon Dis. 2018;13:917925.

14. Ortega-Martnez A, Prez-Rubio G, Ambrocio-Ortiz E, et al. The SNP rs13147758 in the HHIP gene is associated with COPD susceptibility, serum, and sputum protein levels in smokers. Front Genet. 2020;11:882.

15. Li L, Li S, Zhong X, et al. SERPINE2 rs16865421 polymorphism is associated with a lower risk of chronic obstructive pulmonary disease in the Uygur population: a casecontrol study. J Gene Med. 2019;21(9):e3106.

16. Chu M, Wu S, Wang W, et al. Functional variant of the carboxypeptidase M (CPM) gene may affect silica-related pneumoconiosis susceptibility by its expression: a multistage case-control study. Occup Environ Med. 2019;76(3):169174.

17. Wang W, Yu Y, Wu S, et al. The rs2609255 polymorphism in the FAM13A gene is reproducibly associated with silicosis susceptibility in a Chinese population. Gene. 2018;661:196201.

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SNP of the SREK1 gene is associated with COPD in Kashi | COPD - Dove Medical Press

NEW YORK The University of Saskatchewanon Friday said it has received C$709,500 ($560,771) from the provincial government to develop resources to personalize treatment for ovarian cancer patients.

The government of Saskatchewan provided the funds through Ovarian Cancer Canada's OvCAN research initiative. The project, led by Laura Hopkins, professor of oncology and gynecology at the University of Saskatchewan's College of Medicine, will establish a tumor testing and ovarian cancer drug prediction program. Around C$250,000 of the funds will support the creation of a tumor bank, which will include anonymized biological samples and linked clinical data, such as treatment and disease outcomes, from ovarian cancer patients.

The grant will specifically help researchers advance a gene panel they're developing at the university to test ovarian cancer patients for biomarkers that can help personalize their treatment. Currently, the university sends patients' samples to the US for such analysis, which costs up to C$4,000 per test.

Hopkins said in a statement that the testing program could start by January 2022, and the test could eventually become a resource for cancer patients not just in Saskatchewanbut throughout Canada. "If this is successful, this kind of project can go across other sites for breast cancer, lung cancer, prostate cancer, and other cancers," she said.

Hopkins and her team are also studying two PARP inhibitors recently approved in Canada to explore which is best at keeping ovarian cancer patients in remission.

The project involves six Saskatchewan gynecology oncologists, including Hopkins, who are collaborating with pathologists and other university scientists as well as cancer experts at the University of British Columbia, and quality-of-life specialists in Australia and California. "We will be the first to implement this type of precision medicine in Canada," Hopkins said. "Hopefully we can make a business case across the three-year duration of the grant so that we can continue the precision medicine approach."

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University of Saskatchewan to Advance Ovarian Cancer Tumor Bank, Genetic Test With Government Grant - Precision Oncology News

When developing a regulatory submission for a gene or cellular therapy, its essential to keep the basics in mind, such as how to demonstrate the products identity, purity, and potency, Haroon Hashmi, PhD, told attendees at RAPS Convergence 2021.Even when we think about these complex technologies -- new emerging technologies -- certain regulatory principles always stay in place, said Hashmi, senior vice president at Ziopharm Oncology, which has developed the Sleeping Beauty T-cell therapy platform. When you think about the CMC [its] identity, purity, potency, he added, referring to the common acronym for chemistry, manufacturing and controls.Advanced therapy sponsors will need to convince regulators that they can identify their therapeutic product based on its unique properties, show where there is the potential for impurities to be introduced during the manufacturing process or the gene transfer, and assess product potency to demonstrate that it is consistent using the manufacturing process, Hashmi explained.Developers should also keep in mind that the same regulatory pathways that are available to more traditional drug products are also used for advanced therapies. These include orphan drug designation, accelerated or conditional approval, breakthrough designation (BTD), priority medicine (PRIME) and regenerative medicine advance therapy (RMAT), depending on which health authority will be receiving the submission.Hashmi also emphasized the need for developers to communicate the story of their therapy to regulators through module 2 of the US Food and Drug Administrations investigational new drug (FDAs IND) application. Many of the questions that developers get from FDA reviewers are because they are not weaving a comprehensive story about the technology within their IND application. You have to tell the story and you have to connect the dots, he said.Hashmi also advised developers to take advantage of the opportunities for early interaction with the FDA through the INTERACT meeting and the additional pre-IND meeting that follows it. And even if the product is initially planned only for clinical trials in the US, Hashmi said its advisable to align with European regulators early in the process.Nonclinical development considerationsAs part of nonclinical development, sponsors should consider what experiments need to be conducted and shared with regulators to address the safety concerns that are specific to their investigational therapy. This is especially important in cases where there are no relevant animal models with which to conduct the toxicology assessment.The key elements to demonstrate include the products anti-tumor effect, potential toxicities (both on-target, off-tumor and on-target, on-tumor), and the potential risk of cell transformation.In the absence of an animal model, sponsors could use in silico sequence homology and in vitro data to show that the therapy is not causing off-tumor toxicities. The FDA will also be looking for data to show that on-tumor effects like cytokine release syndrome or neurotoxicity are not creating a cytokine storm, Hashmi said. (RELATED: Animal models have limitations for safety assessment of gene therapies: FDA adcomm, Regulatory Focus 02 September 2021)When it comes to the potential risk for cell transformation, sponsors need to provide information on the vector copy number (VCN), which should tie into the manufacturing data. Though FDA has not set a specific VCN in its guidance, a lower number is generally considered safer, Hashmi said, though it will depend on the technology involved.The totality of the package has to address all these safety concerns, Hashmi said.CMC considerations Drug substance, drug product, and potency are all important CMC considerations for gene and cellular therapies, Hashmi said.While some advanced therapy products do not have traditional drug substances, FDA is looking for the same level of information on plasmids and vectors that would typically be submitted for drug substances. That should include information on how the highest quality materials are being used and demonstration of lot-to-lot consistency. Sponsors should complete all the information required for a traditional drug substance, even if it is to note that the section is not applicable, Hashmi said.Cover the details on those plasmids [and] vectors at the same level of detail that you would cover in another traditional drug substance, he said.For drug products, key development considerations include:

2021Regulatory Affairs Professionals Society.

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Convergence: Regulatory considerations in advancing gene and cellular therapies - Regulatory Focus

Its a great honor to have Bob back on board. He played a pivotal role for us as an advisor beginning in 2011, before having to resign when he was appointed as CDC director. Bobs passion for public service is matched only by his devotion to the cause of finding a cure for HIV. -AGT CEO Jeff Galvin

ROCKVILLE, Md. (PRWEB) September 15, 2021

American Gene Technologies (AGT) announces the appointment of Dr. Robert R. Redfield, former director of the Centers for Disease Control and Prevention (CDC) and co-founder of the Institute for Human Virology, as special advisor to AGT CEO Jeff Galvin.

Dr. Redfield is well known for his decades of dedicated work in virology, immunology and clinical research. He has helped lead collaborative research into HIV/AIDS since the beginning of the epidemic during his years as a U.S. Army physician and at the University of Maryland School of Medicine. Dr. Redfield has held multiple advisory positions, including with the President's Advisory Council on HIV/AIDS, the National Institutes of Health and the U.S. Food and Drug Administration. He was the CDC director from March 2018 to January 2021, leading the agency through a period of historic challenges, including the onset of the COVID-19 pandemic.

Its a great honor to have Bob back on board, said AGT CEO Jeff Galvin. He played a pivotal role for us as an advisor beginning in 2011, becoming chair of the Clinical Advisory Board for our HIV Cure Program before having to resign when he was appointed as director of the CDC. Bobs passion for public service is matched only by his devotion to the cause of finding a cure for HIV. Having him on our team strengthens our capacity to achieve that dream.

Dr. Redfields expertise in virology and extensive experience in HIV research will be very helpful as AGT conducts the ongoing phase 1 trial of AGT103-T, a cell and gene therapy for HIV disease. Durable viral suppression without the use of antiretroviral therapies is the goal of AGTs phase 1, and Dr. Redfields long-standing commitment to the development of innovative treatments for HIV makes him well-suited to assist in the major milestones ahead.

This is an exciting homecoming for me, said Dr. Redfield of his appointment. I have followed AGTs development from the beginning, and am proud to do what I am able, to bring this therapy to patients. I look forward to once again collaborating with Jeff and the entire team as AGT continues to act as pioneers in gene therapy and the work for an HIV cure.

Phase 1 in the Maryland/DC Area AGTs Phase 1 trial for AGT103-T is currently underway at trial sites in the Maryland / Washington, DC area. The recruitment status of the phase 1 RePAIR (Restore Potent Antiviral Immune Responses) clinical trial along with information of the trial sites can be found on ClinicalTrials.gov. The trial is the companys first step in clinical testing of cell and gene therapies for HIV, cancer, and rare diseases.

About HIV According to UNAIDS, approximately 37.7 million people worldwide live with HIV/AIDS. In the United States, government statistics show 1.2 million people have HIV and estimate that 34,800 Americans were newly infected with HIV in 2019. Across the globe, UNAIDS estimates that approximately 1.5 million individuals were newly infected with HIV in 2020. The Washington D.C./Baltimore area is often cited as a hot spot for HIV, with Washington, D.C., having the highest rate of infection at nearly 46 cases per 100,000 population and Baltimore City having rates of 17 cases per 100,000. Maryland also ranks sixth among U.S. states and territories in HIV diagnosis rates, with more than 900 new cases in 2019 alone, according to the Maryland Department of Health.

Since the late 1980s, antiretroviral therapies have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatments offer durable remission or cure for HIV. AGT is committed to addressing this unmet medical need.

About AGT103-T AGT103-T is a genetically modified cell product made from a person's own immune cells. AGT's unique approach is focused on repairing the key immune system damage caused by HIV. AGTs goal is to develop a cell and gene therapy capable of repairing the immune system to provide natural control over HIV replication.

About American Gene Technologies AGT is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT's mission is to transform people's lives through genetic medicines that rid the body of disease. AGT has been granted four patents for the technology used to make AGT103-T and 11 patents for its unique gamma-delta () T cells to destroy a variety of solid tumors. The company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT's treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2022.

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American Gene Technologies Appoints Dr. Robert R. Redfield as Special Advisor to the CEO - PR Web

Advances in technology have helped harness the power of genomic sequencing, offering new hope in the fight against genetic diseases. Katie Hampson reports.

What was once a pipe dream to early genetics researchers is now a reality as the genomic age brings new ways to detect and treat disease, and offers families with devastating inherited illnesses a helping hand against fate.

We humans have never known more about our genetics than now, says Professor Nigel Laing, an internationally respected genetics expert from Perths Harry Perkins Institute of Medical Research.

It is all about giving information to people that they can use to make decisions with.

The complex science of genomics isnt exactly sexy stuff but genetics experts say the benefits to medicine, in particular, have the potential to be huge.

Already, Australian scientists have been relying heavily on genomic sequencing to trace infection outbreaks and, more recently, to understand how COVID-19 is spread in an effort to contain the virus.

Sequencing the genome of viruses allow us to quickly identify the emergence of new strains, explains Timo Lassmann, head of the genetics and rare diseases program at Telethon Kids Institute.

Other powerful information that has emerged from sequencing concerns recessive genes for certain diseases.

Most of us are carriers of mutations that are harmless. But Professor Laing says that this can change if we start a family with a partner who has a mutation in the same gene.

Now, couples can undergo genomic sequencing, which he says will identify if they have recessive genes for certain diseases, as well as their risk of having a child affected by a genetic disease. This means couples no longer have to find out they are at risk of having a child with a recessive disease by having an affected child they will be informed beforehand, he explains.

All this is possible because scientists are given a comprehensive picture of a persons genetic information when they sequence their genes.

Professor Laing says exciting discoveries about which genes are responsible for certain life-limiting rare or undiagnosed disorders are occurring more and more, thanks to the sophistication of todays sequencing technology.

Research suggests rare conditions affect one in 50 Australians, he says.

The genome contains the complete set of instructions to build a human being, Professor Lassmann says. Understanding what is written in the genome, and how the information is read and used, is highly relevant to understanding human disease and health.

When many people talk about their DNA, what they are really referring to is their genome.

Each of us has a genome made up of more than 3 billion letters of DNA, found in nearly every cell in the body, and errors can trigger a vast array of disorders.

The first whole human genome to be sequenced began in 1990 and took 13 years.

It was not without controversy and not every promise has materialised but it produced some great strides in medicine.

And, thanks to technological advances, it can now be done in a day for a fraction of the cost, leading to an explosion in genetic research.

Professor Laing says genomic sequencing has already offered tremendous benefits to many people.

For example, scientists have used sequencing to hunt for genes linked to different types of disease, such as breast cancer, and to learn more about inherited disorders, including Huntingtons disease and cystic fibrosis. Families can then find out if they might pass the conditions on to the next generation.

Professor Laing, who has been involved in the discovery of more than 30 disease genes, says that when a diagnosis can be made immediately, it transforms the lives of families.

Take Duchenne muscular dystrophy. This life-limiting condition, which mainly affects young boys, causes every muscle in the body, including the heart, to deteriorate.

One of the problems with DMD is that the early signs are often vague and confusing, which means children are put through a battery of tests in the first years of their life as doctors try their best to work out the cause. These tests can be uncomfortable, invasive, costly and will not necessarily be effective.

Now, thanks to genomic sequencing, Professor Laing says this condition can be pinpointed quickly and some boys with it can now be treated immediately.

The great thing about genetic testing is that now, for a large number of diseases, the first test a clinician will order is a genetic test and that can get the answer and save a whole lot of other different tests, he explains.

Furthermore, when we do a genetic test, we find the variant and that can lead to switching which medication the patient is on from something that isnt going to work to something that is going to work and that is just joyful.

Australia has had a long history of excellence in genetics research and our scientists continue to help discover new disease genes and world-first treatments.

A team of scientists, led by Perth professors Steve Wilton and Sue Fletcher, discovered the first treatment in the world to drastically improve the health of many young people with DMD.

Whats more, Dr David Chandlers Perth research was crucial in helping to discover a new disease gene responsible for a condition that causes facial deformity. The condition can be devastating, with some babies needing breathing support and a tracheostomy, and the deformity often continues to worsen as the child grows.

One WA family has 12 members who are affected. However, a breakthrough like this meant they finally had the answers they longed for, and so did other families living with the same condition in other parts of the world, including in the US. For the first time, those families are also able to find out if they will pass the condition on to the next generation and now have reproductive choices.

Professor Laing, who jointly supervised Dr Chandlers research, says hunting for disease genes is like putting together an international jigsaw puzzle.

For a start, DNA from families across the globe needs to be cross-referenced.

And, back when Dr Chandler started hunting for this disease gene some 20 years ago, he could only take his research so far because the technology at the time was limited.

The latest genetic sequencing technology provides scientists with a goldmine of information, which can help researchers achieve the kind of eureka moment experienced by Dr Chandler and the US team of scientists he collaborated with.

In some ways, I keep feeling it is the people who have invented the new technologies that deserve all the credit here, Professor Laing says. Because before the next-generation sequencing came on board, it was just not financially possible to do all the good as a researcher that you would like to do.

He says as sequencing has become easier and cheaper to do, more diseases have opened up.

Professor Lassmann agrees the field of genomics is full of promise, adding: I can see the potential across many diseases and (more) people will benefit from genetic screening when treatments and strategies for disease management become available for the majority of possible diseases.

Genome sequencing is used in many contexts, says Telethon Kids Institute Professor Timo Lassmann, including for finding the cause for rare and undiagnosed diseases and in cancer. Broadly speaking, however, genome sequencing can currently be used for:

Screening: Detecting conditions and risk factors early before disease occurs, allowing for early intervention and changes in lifestyle decisions.

Diagnosis: Identifying the exact cause of a disease.

Treatment: Identifying the most effective treatment option, thus minimising adverse side-effects while improving patient outcomes.

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Cracking the code - The West Australian

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 15 September 2021 15:53Hits: 294

If Approved, Vutrisiran will Provide a New, Subcutaneously Administered, Once-Quarterly Treatment Option for Patients with hATTR Amyloidosis

CAMBRIDGE, MA, USA I September 13, 2021 IAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with polyneuropathy.

hATTR amyloidosis is a rare, rapidly progressive, debilitating and fatal condition and we are delighted that the EMA has agreed to review the regulatory submission for vutrisiran based on the positive 9-month data from the HELIOS-A Phase 3 study, said Rena Denoncourt, Vice President, TTR Franchise Lead. If approved, we believe that vutrisiran will provide an important new subcutaneously administered, once-quarterly treatment option for patients, with the potential to reverse manifestations of the disease in hATTR amyloidosis patients with polyneuropathy. We look forward to working closely with the EMA to bring vutrisiran to the hATTR amyloidosis community in Europe.

Positive 9-month results from the HELIOS-A Phase 3 study of vutrisiran were presented in April 2021 at the American Academy of Neurology (AAN) Virtual Annual Meeting. At 9 months, vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy, quality of life, and gait speed, and demonstrated an encouraging safety profile, relative to the external placebo group of the APOLLO study of patisiran. As aligned with the EMA, the results of the 18-month analysis from the HELIOS-A study will be provided to the Agency during its evaluation of the MAA.

Vutrisiran has been granted Orphan Drug Designation in the European Union (EU) and U.S. for the treatment of ATTR amyloidosis. In June 2021, the U.S. Food and Drug Administration (FDA) accepted the Companys New Drug Application (NDA) submission for review for vutrisiran, with an action date set for April 14, 2022 under the Prescription Drug User Fee Act (PDUFA). The Company also plans to submit regulatory filings in Brazil and Japan in 2021.

About Vutrisiran

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary ATTR (hATTR) and wild-type ATTR (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylams Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the European Medicines Agency, the U.S. Food and Drug Administration, or any other health authority.

About the HELIOS-A Phase 3 Study

HELIOS-A (NCT03759379) is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran. The study enrolled 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries. Patients were randomized 3:1 to receive either 25mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (as a reference comparator) for 18 months. The efficacy results of vutrisiran in HELIOS-A are compared to the placebo group from the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in a patient population similar to that studied in HELIOS-A. The primary endpoint is the change from baseline in mNIS+7 score at nine months. Secondary endpoints at 9 months are the change from baseline in the Norfolk QoL-DN score and the timed 10-MWT. Changes from baseline in NT-proBNP were evaluated as an exploratory endpoint at nine months. Additional secondary endpoints at 18 months will be evaluated in the HELIOS-A study, including change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI), Rasch-built Overall Disability Scale (R-ODS), and serum transthyretin (TTR) levels. Additional exploratory cardiac endpoint data at the 18-month time point will be evaluated, including NT-proBNP, echocardiographic measures and cardiac amyloid assessments with technetium scintigraphy imaging. Following the 18-month treatment period, all patients are eligible to receive vutrisiran for an additional 18 months as part of the randomized treatment extension where they will receive either 25mg vutrisiran once quarterly or 50mg vutrisiran once every six months.

About hATTR Amyloidosis

Hereditary transthyretin-mediated (hATTR) amyloidosis is an inherited, progressively debilitating, and fatal disease caused by variants (i.e., mutations) in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Variants in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis, represents a major unmet medical need with significant morbidity and mortality affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as a major scientific breakthrough that happens once every decade or so, and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylams RNAi therapeutic platform, function upstream of todays medicines by silencing messenger RNA (mRNA) the genetic precursors that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the potential treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylams commercial RNAi therapeutic products are ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran) and Leqvio (inclisiran) being developed and commercialized by Alnylams partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its Alnylam P5x25 strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

SOURCE: Alnylam Pharmaceuticals

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Alnylam Submits Marketing Authorization Application to the European Medicines Agency for Investigational Vutrisiran for the Treatment of Hereditary...