Monthly Archives: March 2021

A pioneering gene therapy innovation centre at the University of Sheffield is set to advance scientific discoveries into promising treatment options for millions of patients with life-threatening diseases.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC), led by Professor Mimoun Azzouz, is one of three pioneering hubs announced today (18 March 2021) in a new 18 million network funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC).

Gene therapy is a promising treatment option for more than 7,000 rare diseases that currently have no cure. It aims to treat these conditions, by engineering another gene to replace, silence or manipulate the faulty one.

The UK has a world-class genetics research base however, to date, academics have found it difficult to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials.

Professor Mimoun Azzouz, Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield, said: The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases.

Gene therapies are pioneering medical advances that have the potential to offer much-needed, novel, effective treatments for many rare and incurable diseases that cannot be treated by conventional drug compounds.

This is a momentous milestone for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the UK.

Sheffield has emerged as one of the leading players in the cell and gene therapy and this national network of partners, facilities and training programmes will allow us to keep pace with translational discoveries for new and potentially life changing treatments

Professor Mimoun Azzouz

Director of the GTIMC and Chair of Translational Neuroscience at the University of Sheffield

The new centre, which will be the first in the north of England, builds on the University of Sheffields strong history of translational research and its reputation as an international centre of excellence for gene therapeutics.

Professor Dame Pam Shaw, Director of the NIHR Sheffield Biomedical Research Centre and co-applicant on the GTIMC application said: This exciting development and partnership will speed up the pull through of new gene therapies into early phase clinical trials and offer hope to patients with neurological and other rare diseases that can be addressed in this way.

The support given to this initiative will greatly accelerate the translational potential of genetic therapies in the UK and bring benefits in key areas of unmet medical need.

The state-of-the-art centre will bring together academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands and Wales enabling academic-led clinical trials of novel gene therapies. The GTIMC will deliver essential translational and regulatory support alongside extensive training and skills programmes to enable upskilling and address shortage of skills in Good Manufacturing Practice (GMP) manufacturing.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: At the University of Sheffield we focus our research on finding real-world solutions to some of the biggest global challenges. The Gene Therapy Innovation and Manufacturing Centre will unlock development pathways for new treatments for people affected by devastating genetic disorders, many of which have no cure.

We are delighted that our University is at the forefront of research in this pioneering field of medicine and that this new centre will build on our reputation as an international centre of excellence for gene therapeutics.

This is fantastic news for the City Region and the North of England and we look forward to working collaboratively to share technical skills and resources across the coordinated network.

Alongside the national network funding from LifeArc, the MRC and BBSRC, the GTIMC was made possible thanks to a 3 million donation from The Law Family Charitable Foundation, established by Andrew Law and his wife Zo. This funding was part of a record 5 million donation from the University of Sheffield alumnus, which will also see the launch of a new student support programme.

Andrew Law, who is Chairman and CEO of Caxton Associates, said: The University of Sheffield is rapidly developing a global reputation in gene therapy. The new Gene Therapy Innovation and Manufacturing Centre will drive innovation and world-class research, while presenting a real opportunity to catalyse the creation of new start-up companies to facilitate commercialisation in the North.

This investment will enhance vital partnerships with biotechnology and pharmaceutical companies to help accelerate gene therapy programmes and clinical trials, at the same time as supporting regional economic growth and job creation.

The GTIMC is planned for a site on the University of Sheffields Innovation District close to existing translational research facilities and will contribute to an ongoing programme of regional investment and regeneration.

The centre will include a cutting-edge GMP (good manufacturing practice) facility that will support gene therapy projects emerging from universities across the UK.

The facility will utilise highly efficient processes to manufacture clinical grade adeno-associated viruses (AAV) and provide all the necessary quality assurance, regulatory certification and governance for human trials at Advanced Therapies Treatment Centres and NHS trusts within the GTIMC and the national network.

The three national hubs, located at the University of Sheffield, Kings College London, and NHS Blood and Transplant in Bristol will operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research.

Dr Melanie Lee, CEO of LifeArc, said: Recent innovations in gene therapies hold enormous potential for treating conditions such as rare diseases, but often promising ideas particularly in academia are not making it through to patients. Through our collaboration, we aim to meet the need for researchers to have access to the essential facilities and translational advice to progress promising research.

The GTIMC will manufacture commonly used vectors including both lentivirus and adeno-associated virus that are needed for genetic therapy trials, while positioning the UK for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years. The hub network will also design and share commercially ready platforms, using common cell-lines, plasmids and reagents to reduce costs, facilitate simplified licensing agreements and streamline regulatory reviews. A key aim is to smooth the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond.

Professor Fiona Watt, MRC's Executive Chair, said: The new network of Innovation Hubs for gene therapies will build on the UKs great strengths in this area, providing targeted investment in vital infrastructure to accelerate academic research programmes down the path to patient benefit, supporting the delivery of a new wave of genetic medicines.

Notes for editors

Sheffield GTIMC Lead Institution

The University of Sheffield

Sheffield GTIMC Co-Lead Institutions

Cell & Gene Therapy Catapult

Centre of Process Innovation (CPI)

University of Birmingham

University of Liverpool

The Midlands-Wales Advanced Therapies Treatment Centre [MW-ATTC]

Sheffield GTIMC Partners

University of Leeds

University of Leicester

University of York

Cardiff University

University of Bradford

Northern Health Science Alliance (NHSA)

NHS Hospital Trusts of Sheffield, Leeds, Birmingham Womens and Children Hospital

Leeds Teaching Hospitals NHS Foundation Trust

Genomic Laboratory Hubs

Lonza AG

Cobra Biologics

Advanced Manufacturing Research Centre (AMRC)

National Horizons Centre (NHC)

National Institute for Biological Standards

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New gene therapy innovation centre to advance scientific discoveries into life-changing treatments - University of Sheffield News

LEXINGTON, Mass., March 15, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a clinical- stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today reported financial results for the year ended December 31, 2020 and provided a business update.

"The fourth quarter of 2020 marked a continuation of strong momentum for LogicBio which has also extended into 2021," said Frederic Chereau, President and CEO. "Most notably, we continue to anticipate enrolling the first patient in our SUNRISE Trial of LB-001 for the treatment of MMA in the very near future. We expect this event will be followed by additional enrollments soon after and allow us to begin to generate data regarding the safety, and clinical impact of treatment with LB-001 for these children. In addition, we also recently announced that we have extended our collaboration with the Children's Medical Research Institute of Australia to continue to develop sAAVy, our next generation capsids platform, which has already yielded novel liver-tropic capsids that we believe are superior to ones that are currently used in the clinic. We believe these capsids will allow LogicBio to continue to expand its gene editing platform, GeneRide, and potentially initiate gene therapy programs, either alone or in collaboration with strategic partners. We look forward to providing further updates on these programs throughout 2021." Mr. Chereau concluded by saying, "I am also very excited by the new members we have added to our team. Both Mariana Nacht, our new CSO, and Cecilia Jones, our new CFO, bring a wealth of biotech industry experience, and I am delighted they have chosen to join LogicBio as we move into this exciting next phase of progress."

Anticipated LogicBio Milestones for 2021:

LB-001 for MMA

Pipeline

Full Year 2020 Financial Results

Twelve Months Ended December 31, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVy capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit http://www.logicbio.com.

Forward Looking Statements

This press release contains "forward-looking" statements within the meaning of the federal securities laws, including those related to the Company's plans to enroll patients in, advance and complete its planned Phase 1/2 SUNRISE clinical trial of LB-001 in MMA and the potential benefits to patients of LB-001; the timing, progress and results of the Company's research and development activities, including those related to the GeneRide technology platform and sAAVy, our next generation capsids program; and the sufficiency of our cash on hand to fund our operating expenses and capital expenditures. These are not statements of historical facts and are based on management's beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Company's plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Company's current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Company's ability to progress with its research, development, manufacturing and regulatory efforts, including the Company's plans to enroll patients in, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Company's common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Company's filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Company's Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Company's Quarterly Report on Form 10-Q filed on May 11, 2020, and the Company's subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

LogicBio Therapeutics, Inc.

Condensed Consolidated Statements of Operations

(In thousands, except share and per share data)

Year Ended December 31,

2020

2019

REVENUE

Service revenue

$ 3,454

$

Total revenue

3,454

OPERATING EXPENSES

Research and development

22,753

30,656

General and administrative

12,212

10,385

Total operating expenses

34,965

41,041

LOSS FROM OPERATIONS

(31,511)

(41,041)

OTHER (EXPENSE) INCOME, NET:

Interest income

181

1,500

Interest expense

(1,098)

(546)

Other expense, net

(5)

(19)

Total other (expense) income, net

(922)

935

Loss before income taxes

(32,433)

(40,106)

Income tax provision

(188)

(22)

Net loss

$ (32,621)

$ (40,128)

Net loss per sharebasic and diluted

$ (1.29)

$ (1.78)

Weighted-average common stock outstandingbasic and diluted

25,364,453

22,602,954

LogicBio Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(In thousands)

As of

December 31, 2020

December 31, 2019

Cash, cash equivalents and investments

$ 70,075

$ 50,647

Other assets

10,565

5,013

TOTAL ASSETS

$ 80,640

$ 55,660

Accounts payable, accrued expenses and other liabilities

$ 19,213

$ 13,373

Stockholders' equity

61,427

42,287

TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY

$ 80,640

$ 55,660

SOURCE LogicBio Therapeutics, Inc.

http://www.logicbio.com

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LogicBio Therapeutics Reports Full Year 2020 Financial Results and Provides Business Update - PRNewswire

AUniversity of Virginia School of Medicinescientist is developing an innovative gene therapy she hopes will slow disease progression and improve movement, coordination and communication in children with Rett syndrome. The approach also may be useful for battling other genetic disorders involving the X chromosome.

UVA researcher Sanchita Bhatnagar discovered that tiny bits of RNA, called microRNAs, play an important role in Rett, a rare genetic disorder that can impair childrens ability to speak, move and even breathe. Based on that finding, she is seeking to sop up those RNA bits, called microRNA, using absorbent particles called microRNA sponges.

Early work in lab models has produced promising results, and she hopes the approach could lead to a better quality of life for children with Rett.

We are seeing that lab animals treated with this gene therapy are more mobile. Theyre moving faster, theyre smarter, Bhatnagar said. And if that translates into even modest improvements for children, it could make a big difference, she said: If we can help a child to move more independently, or improve their ability to communicate, I think for a parent, thats a big win.

Rett syndrome affects approximately 1 in 10,000 children, almost exclusively girls. Thats because its caused by a mutation in theMECP2gene found on the X chromosome. Girls have two X chromosomes, while boys have an X and a Y.

Bhatnagars discovery of the role of microRNA in Rett came as a surprise, because it was the first time microRNA had been linked to X chromosome biology. That finding dovetailed perfectly with her labs expertise in creating microRNA sponges that can target microRNA, absorbing it and then breaking it down.

We are not altering the genomic DNA, Bhatnagar emphasized. We are just using them [the microRNA sponges] as inhibitors that are delivered through [adeno-associated viral] vectors. These vectors do not go and integrate into the genome, so we hope for minimal secondary effects.

Bhatnagar hopes her new approach could be useful both for treating Rett and other genetic disorders that involve the X chromosome, such as Fragile X syndrome, a condition that causes learning disabilities, developmental delays and behavioral problems. To advance her research toward patient impact, she works with the UVA Licensing & Ventures Group to navigate patent protection and industry partnerships. After disclosing her discovery to the Licensing & Ventures Group in 2019, Bhatnagar presented her research and attracted a top expert in developing new gene therapies, Kathrin Meyer of the Abigail Wexner Research Institute at Nationwide Childrens Hospital, as a research collaborator. Massachusetts-based Alcyone Therapeutics Inc. is sponsoring the research at both UVA and the Wexner Research Institute to support its transition to clinic.

The research collaborators hope to begin clinical trials in the near future. If the initial trials prove safe and successful, that would lead to larger trials to evaluate the effectiveness of the approach. The federal government would then consider the trial results to determine if the approach should be approved as a treatment.

We think what Dr. Bhatnagar has come up with is very elegant, and we are excited about its potential to improve the lives of many patients, PJ Anand, CEO of Alcyone Therapeutics, said. The beautiful part is that this gene therapy is what we call in the industry a pipeline in a product. The exact same gene therapy product can be used to potentially address diseases that are caused by other genes on the X chromosome. Once we have the initial proof of concept in the clinic for the first disease, we can use much of that same information to move toward clinical trials in other X-linked diseases as well, so it will really be an efficient path forward.

Bhatnagar, of UVAs Department of Biochemistry and Molecular Genetics, said she feels very emotional about the potential clinical implications of her work, especially knowing the dramatic impact Rett has on the affected girls and their families.

Rett syndromeleads to severe impairments, affecting nearly every aspect of a childs life.Its often a full-time job as a parent, because these girls need assistance with all their living skills, Bhatnagar said. I dont know if this therapy is going to fix all of that. But it may be able to help them, and any help will be a big step forward.

The early-stage studies for the project in Bhatnagar lab were supported by the Hartwell Foundation and the UVA Brain Center.

To keep up with the latest medical research news from UVA, subscribe to theMaking of Medicineblog.

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UVA Scientist Developing Gene Therapy to Help Girls With Rett Syndrome - University of Virginia

$262.3 million cash balance at the end of 2020 bolstered by additional net proceeds of $211.3 million from follow-on offering, funding key milestones into 2024

Ron Cooper appointed to Board of Directors

Company remains on track to begin IND-enabling studies for hemophilia A in 2021

CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) --Generation BioCo. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, reported recent business highlights and fourth quarter and full year 2020 financial results.

2020 was a foundational year for Generation Bio in which we strengthened the leadership team, advanced our platform science, and added substantially to our balance sheet to fund our lead programs to human data, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. We entered 2021 with preclinical data in mice and non-human primates that we believe demonstrate our platforms potential to go beyond what is currently possible with gene therapy for hemophilia A. We expect to announce additional data throughout the remainder of this year that will support the rapid expansion of our pipeline and showcase our differentiated approach to creating a new class of genetic medicine.

Recent Business Highlights

Selected Anticipated Company Milestones in 2021

Fourth Quarter and Full Year 2020 Financial Results

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is engineered to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about our strategic plans or objectives, our technology platforms, our research and clinical development plans, and other statements containing the words believes, anticipates, plans, expects, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; whether the Companys cash resources are sufficient to fund the Companys operating expenses and capital expenditure requirements for the period anticipated; the impact of the COVID-19 pandemic on the Companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of our most recent quarterly report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change.However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsMaren KillackeyGeneration Bio541-646-2420mkillackey@generationbio.com

MediaAlicia WebbGeneration Bio847-254-4275awebb@generationbio.com

Stephanie SimonTen Bridge Communications617-581-9333stephanie@tenbridgecommunications.com

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA (Unaudited)(In thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Fourth Quarter and Full Year 2020 Financial Results - BioSpace

This article is part of a Technology and Innovation Insights series paid for by Samsung.

In this episode of The Next Wave, Young Sohn sits down with Lisa Alderson, CEO of Genome Medical, an innovative healthcare firm that is working hard to ensure that genomic medicine is made accessible to everyone.

Alderson shares her views on a range of issues around genomics. She discusses the vast promise of the technology and her companys efforts to bring its game-changing potential to all patients by providing clinical support tools to non-genetics professionals. She also addresses the ethical questions raised by genomics, particularly its use in prenatal diagnostics. And she highlights the critical role genomics played in developing COVID vaccines in record time.

Genomics is swiftly moving to the forefront of medicine. This potentially limitless technology is transforming health care by making it more targeted, personalized, and proactive.

Genomics can not only help with preventive care but also with determining treatment options for chronic ailments such as heart disease. It is becoming essential in reproductive health counseling, as well as in screening for infertility and newborn genetic disorders. And it is proving to be instrumental in the development of advanced therapeutics for diseases such as cancer and even for treating global outbreaks like COVID-19.

But, to date, there has been one big problem with genomic medicine: access. Lisa Alderson, CEO of Genome Medical, explains that even though the technology is now advanced, the vast majority of patients are still not getting access to genomics, even when it can be medically beneficial. This is mostly due to insurance barriers, a broad lack of clinical understanding, and the sheer volume and complexity of new tests being brought to market.

Her company is on a mission to change the dynamic by bringing genome-enabled health care to everyone through Genome Medicals extensive network of genetic specialists and a technology platform delivering genomics as a service.

Alderson points out that virtually everybody can benefit from access to genetic insights. Not only does genetics offer the promise of getting the right therapy to the right patient at the right time, it can also uncover new information to better inform clinical care.

And as genomics advances, its ability to detect and treat diseases like cancer vastly improves. As a result, the technology is increasingly important not just for individuals with rare genetic conditions but for everyone from birth to old age.

Historically, for instance, only one or two genes were used to test for hereditary breast cancer or ovarian cancer. But because the science has evolved, we now know there are 11 genes that can help detect hereditary breast and ovarian cancer. In fact, says Alderson, there are over a hundred genes that can increase our risk of cancer. So as genetic testing becomes more precise and accurate, it has the potential to save exponentially more lives.

One way to help genomics reach its full potential is by providing clinical support tools to non-genetics professionals. This will allow them to better understand which patients would benefit from what tests, and how to interpret and use the resulting information to guide clinical care.

One quandary, of course, is that most local health care systems dont have full-time metabolic geneticists on staff. As a result, they end up referring their patients out to leading academic centers, which is where the bulk of genomic health care is carried out today. And that means local health systems could lose those patients for the full continuum of their care.

To address this, Genome Medicals genomics as a service SaaS platform provides rapid patient education and engagement and also supports providers with the necessary clinical tools and knowledge they need to utilize genomics, as appropriate. Alderson says she wants every hospital and health system to have access to genomics in its community setting.

Alderson is increasingly heartened by the fact that genetic testing is expanding beyond its origins in prenatal medicine and oncology to new areas such cardiology, endocrinology, nephrology, and urology.

In the past, for example, most urologists did not have strong use cases for ordering genetic testing. But now, genetic testing is recommended for nearly every metastatic prostate cancer patient. Ultimately, genomics will touch all medical professionals, whether they are pediatricians, oncologists, cardiologists, neurologists, urologists the list goes on and on.

Were sitting at the precipice of what is a huge inflection point in the use of genetics and genomics, says Alderson. Were going to see it having a profound impact on human health, not just for prevention but also to get a deeper understanding of what is causing the disease and thus a richer opportunity to improve how we treat disease. Were getting to the root cause rather than just observing symptoms and then trying to treat those symptoms.

It needs to be mentioned that genomics is not without controversy. With new genetic capabilities comes much debate about how they should be used. For instance, can genetic information be used by insurance companies to discriminate against those with risk factors for certain disorders?

Or what about the use of genetic testing to determine whether youre having a healthy child? And then there is the issue of gene editing and giving scientists the ability to alter the DNA of many organisms. These are areas fraught with significant moral and ethical questions.

Whats powerful about the technology is it can actually cure disease, says Alderson. What is scary about the technology is, do we understand the full downstream effects in editing the genome? We dont know everything about the genome and its role and implications in human health. So there is a lot of complexity here.

There is also much hope. After all, genomics recently played a starring role in the development of COVID-19 vaccines in record time. The vaccines wouldnt be here without genomics and its power to sequence the virus rapidly and correctly.

Alderson says that genomics helped scientists gain a deep understanding of the virus and gave them the ability to create the vaccine in less than a year a previously unheard-of accomplishment. She believes the use of genomics in immunology will accelerate the advancement of treatment options in other disease areas and help address any new viruses that could emerge.

Genomics is obviously one of the most exciting advancements in our lifetime in health care, Alderson concludes. I couldnt be more excited about how far weve come over the last couple of decades but also for what lies ahead.

VB Lab Insights content is created in collaboration with a company that is either paying for the post or has a business relationship with VentureBeat, and theyre always clearly marked. Content produced by our editorial team is never influenced by advertisers or sponsors in any way. For more information, contact sales@venturebeat.com.

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Genomics has the power to transform medicine but it must be made accessible - VentureBeat

Most genetic tests focus on your odds of developing certain diseases or health conditions. But some known as pharmacogenomic (or pharmacogenetic) tests can reveal how your body may respond and react to different medications. To date, researchers have identified more than 400 genetic variations known to affect the metabolism of numerous drugs, including some that help lower cholesterol or prevent blood clots (see "Pharmacogenomics of common heart drugs").

In theory, knowing how people metabolize specific drugs could help doctors choose the safest, most effective treatment for their patients. But in practice, it's not that straightforward, says Dr. Jason Vassy, assistant professor of medicine at Harvard Medical School and a primary care physician at the VA Boston Healthcare System.

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Genetic testing to tailor heart drug prescriptions? - Harvard Health - Harvard Health

Increasing Demand for Gene Mutation and Cancer Research to Drive Market

Over the past decade, organoids are being increasingly used in a range of applications, including disease modeling, pathogenesis, drug screening, and regenerative medicine, and the trend is set to continue during the forecast period. Organoid technologies have witnessed considerable developments in recent years, and continue to pave the way for the development of in vitro physiologic systems that model the origin tissues with high degree of accuracy compared to conventional approaches. The increasing focus on research and development activities is expected to play a key role in the development of the global organoids market during the assessment period.

The capability of 3D-organoid cultures to mimic organ functionality to some degree is one of the key factors likely to establish organoids as an ideal model for applications such as stem cell research to precision medicine. Furthermore, the increasing adoption of the organoid technology for use in a range of applications, including genetic mutation, gene assessment, and tumor modelling is expected to accelerate the overall development of the global organoids market during the assessment period. The assessment of different diseases by leveraging the organoid technology is anticipated to remain one of the most prominent applications of organoids in the upcoming years.

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At the back of these factors coupled with the consistent rise in the investment for research and development, the global orgnoids market is expected to cross the market value of US$ 12.8 Bn by the end of 2030.

Increasing Demand for Tumor Modelling and Biobanking to Propel Market Growth

The consistent progress in the organoid technology has gradually paved the way for tumouroids and patient-centric 3D cultures of cells that are isolated from tumor biopsies. Over the past few decades, tumouroid lines have been established from a plethora of cancer types such as liver, breast, prostate, brain, bladder, and more from metastasis as well as primary tumors. Due to consistent advancements, in the current scenario, tumouroid lines are increasingly being passed in vitro, which opens up new avenues for a range of downstream applications.

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Moreover, 3D-based cultures offer additional advantages over the conventional 2D cancer-derived cell lines. Moreover, research and development activities have indicated that tumouroid culturing is highly efficient in enabling the collection of various cancer subtypes from a large pool of patients. As organoids can be easily derived and expanded from single cancer cells, the adoption of organoids to mimic intratumour diversification in culture is on the rise a factor that is projected to propel the global organoids market during the assessment period.

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New Discoveries Driven by Research & Development to Boost Prospects

Research and development activities are set to play a key role in the overall development of the global organoids market during the assessment period. In addition, increasing collaborations between researchers and medical experts are anticipated to accelerate the development of complex organoids. For instance, two recent discoveries are expected to boost the overall prospects of next-generation organoid development. The new research collaboration between a team of scientists from the Cincinnati Childrens and Japan was announced in August 2020.

As per the research team, discoveries from the new research collaboration are likely to be one of the most critical aspects for the development of a new wave of complex organoids. Although the organoid technology has progressed at a consistent pace over the past few years, the application of the same continues to remain sluggish in the current scenario. However, increasing focus on the commercialization of organoids and consistent progress in genome editing techniques are expected to bolster the growth of the global organoids market during the assessment period.

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Demand for Human and Animal Organoids on Rise amid COVID-19 Pandemic

The advent of the novel COVID-19 pandemic is anticipated to open up new opportunities for the players involved in the current organoids market landscape. While researchers continue to assess the biology of the novel coronavirus, human and animal organoids are gradually anticipated to prove their value as an experimental virology platform. Furthermore, several researchers from various regions of the world have turned toward organoid technologies to assess the tissue tropism of the novel SARS-COV-2 virus.

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Organoids Market: Increasing Demand for Gene Mutation and Cancer Research to Drive Market - BioSpace

Monkgomotsi J Maseng,1,2 Leabaneng Tawe,1 3 Prisca K Thami,2,4 Kaelo K Seatla,1,2 Sikhulile Moyo,2,5 Axel Martinelli,6 Ishmael Kasvosve,1 Vladimir Novitsky,2,5 Max Essex,2,5 Gianluca Russo,7 Simani Gaseitsiwe,2,5 Giacomo M Paganotti3,8,9

1School of Allied Health Professions, Faculty of Health Sciences, University of Botswana, Gaborone, Botswana; 2Botswana-Harvard AIDS Institute Partnership, Gaborone, Botswana; 3Botswana-University of Pennsylvania Partnership, Gaborone, Botswana; 4Division of Human Genetics, Department of Pathology, Faculty of Health Sciences, University of Cape Town, Cape Town, South Africa; 5Department of Immunology and Infectious Diseases, Harvard School of Public Health, Boston, MA, USA; 6BigOmics Analytics, Bellinzona, Switzerland; 7Department of Public Health and Infectious Disease, Faculty of Medicine, Sapienza University of Rome, Rome, Italy; 8Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; 9Department of Biomedical Sciences, Faculty of Medicine, University of Botswana, Gaborone, Botswana

Correspondence: Giacomo M PaganottiBotswana-University of Pennsylvania Partnership, PO Box AC 147 ACH, Gaborone, BotswanaTel +267 76416198Email paganottig@bup.org.bw

Purpose: CYP2B6 liver enzyme metabolizes the two non-nucleoside reverse transcriptase inhibitors Efavirenz (EFV) and Nevirapine (NVP) used in the antiretroviral therapy (ART) regimens for HIV-infected individuals. Polymorphisms of the CYP2B6 gene influence drug levels in plasma and possibly virological outcomes. The aim of this study was to explore the potential impact of CYP2B6 genotype and haplotype variation on the risk of developing EFV/NVP drug resistance mutations (DRMs) in HIV-1 patients receiving EFV-/NVP-containing regimens in Botswana.Patients and Methods: Participants were a sub-sample of a larger study (Tshepo study) conducted in Gaborone, Botswana, among HIV-infected individuals taking EFV/NVP containing ART. Study samples were retrieved and assigned to cases (with DRMs) and controls (without DRMs). Four single-nucleotide polymorphisms (SNPs) in the CYP2B6 gene ( 82T>C; 516G>T; 785A>G; 983T>C) were genotyped, the haplotypes reconstructed, and the metabolic score assigned. The possible association between drug resistance and several independent factors (baseline characteristics and CYP2B6 genotypes) was assessed by Binary Logistic Regression (BLR) analysis. EFV/NVP resistance status and CYP2B6 haplotypes were also analyzed using Z-test, chi-square and Fishers exact test statistics.Results: Two hundred and twenty-seven samples were analysed (40 with DRMs, 187 without DRMs). BLR analysis showed an association between EFV/NVP resistance and CYP2B6 516G allele (OR: 2.26; 95% CI: 1.27 4.01; P=0.005). Moreover, haplotype analysis revealed that the proportion of EFV/NVP-resistant infections was higher among CYP2B6 fast than extensive/slow metabolizers (30.8% vs 16.8%; P= 0.035), with the 516G allele more represented in the haplotypes of fast than extensive/slow metabolizers (100.0% vs 53.8%; P< 0.001).Conclusion: We demonstrated that the CYP2B6 516G allele, and even more when combined in fast metabolic haplotypes, is associated with the presence of EFV/NVP resistance, strengthening the need to assess the CYP2B6 genetic profiles in HIV-infected patients in order to improve the virologic outcomes of NNRTI containing ART.

Keywords: ART, CYP2B6 gene, drug resistance selection, fast metabolizers, HIV

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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CYP2B6 genetic variation with efavirenz and nevirapine | PGPM - Dove Medical Press

America was sick before COVID-19 struck. The pandemic has made our national sickness more acute and illustrated the critical importance of wellness in preventing disease and optimizing health. We know this because COVID disproportionately affected people with chronic illness and unhealthful lifestyles.

As a scientist who has worked at the leading edge of medicine, engineering and genetics for decades, Im on a quest to give mind and body wellness the scientific rigor and urgency it deserves. No doctor, policy or breakthrough drug is as effective as wellness at minimizing disease and enhancing the length and quality of life.

From 1959 to 2014, America experienced a dramatic rise in life expectancy due to advances in medicine, nutrition, lifestyle, the environment, safety and economic well-being. Since then, life expectancy has declined four of the past five years, something we havent seen in a century.

Although COVID-19 was a major factor in 2020s life expectancy decline, it is only part of a bigger national health catastrophe. More than 45% of Americans suffer from at least one chronic condition, and 70% of all deaths in America are attributable to chronic disease.

Consistent with these grim statistics, the U.S. devotes 90% of its healthcare spending to treating patients with chronic physical and mental health conditions. Devastating chronic disease will be with us long after this pandemic recedes unless we make major changes in how we promote health and treat illness.

Our healthcare system is great at fighting disease retroactively, but its dismal at keeping people healthy proactively. Waiting to treat disease after it emerges is not the answer. Once heart disease sets in, cancer spreads or Alzheimers takes root, its too late. Shorter lifespans and diminished quality of life are the result.

In contrast, wellness is the absence of disease in the body, and the most powerful force in human health. While preventive medicine seeks earlier detection of already established disease, scientific wellness gives medical providers a new way of treating patients based on a data-informed understanding of their personal health.

Each persons genome, or genetic makeup, is unique. Other factors, including our epigenomes, blood analytes, microbiomes, environmental toxins, diet and lifestyle, contribute in complex but interrelated ways to disease susceptibility and overall health.

As an example, consider the gut microbiome, which consists of trillions of bacteria of multiple species that live in the gut. When functioning well, these bacteria regulate metabolism, bolster immune responsiveness and even promote cognitive health. Their diversity is essential to human health.

My research organization, the Institute for Systems Biology, did an experiment recently where we studied the gut microbiomes of 9,000 individuals across the entire adult human lifespan. We demonstrated that gut microbiomes in healthy people change markedly as they age. These healthy gut biomes individualize in unique ways, maintaining species diversity while deleting major bacterial species common in young people. These were surprising results.

From our four-year observational studies, we found that people in their 80s with less microbiome change were four times more likely to die than those with markedly changed microbiomes. Four times!

Why healthy microbiomes for the elderly differ from healthy microbiomes for the young is a fascinating research question. Answering it will increase our understanding of the aging process and lead to powerful new strategies for promoting lifelong health.

This is how we need big data and scientific wellness to work together. Starting in 2014, my organization has sequenced genomes and cataloged health measures of 5,000 patients over five years.

We were able to elevate individual wellness and extend its duration by data-driven individual analyses from the genome, blood and lifestyle reports, which led to actionable possibilities.

For example, 91% of a population we studied had very low vitamin D levels (low levels probably increase susceptibility to cancer, Alzheimers, COVID-19 and other diseases). We found that some individuals were brought back to normal by a daily dose of just 1,000 international units of vitamin D, but many others were not. Many of those who were unresponsive had one to several gene variants that blocked the uptake of vitamin D. They often required mega doses, up to 15,000 units per day, to return to normal.

This integration of two data types (genome variants and blood vitamin D level) was necessary for this actionable possibility. This is the essence of personalized medicine treating each individual according to that persons unique traits.

We have found striking blood signals long before disease symptoms can traditionally be diagnosed. That transformational research offers exciting opportunities to pursue interventions that delay or prevent the onset of disease.

Imagine the data-based insights gained by studying the genomes and health measures of a million people. That is our goal. With that depth and breadth of understanding, we can significantly extend the lifespan and enhance the quality of life of every person living today.

In a healthcare system focused on wellness, physicians would spend as much or more time helping patients achieve healthy lifestyles as they currently spend treating patients for preventable or manageable diseases. Using a scientific or quantitative approach to wellness, medical providers will be able to develop tailored interventions based on each patients unique genome, blood, gut microbe, diet and digital physiology.

By identifying transitionary states best described as pre-pre-disease, wellness-oriented physicians could prescribe treatments that reverse and heal adverse health conditions before they become pathologic. Through wellness-focused medicine, individual health spans could extend well into the 90s and beyond.

COVID-19 has wrought a year of sickness and death. We cannot change what has already happened, but we can commit our energy and resources to combating the ongoing pandemic of chronic illness and diminished health.

In a world in which predictive, preventive and personalized care is standard practice, people would not only live longer but also be able to lead more satisfying, productive and active lives. No breakthrough drug or treatment could compete with that. And thats a world within reach today.

Leroy Hood, a member of the National Academies of Sciences, Engineering and Medicine, is a professor and co-founder of the Institute for Systems Biology and senior vice president and chief science officer of the Providence St. Joseph Health system.

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Second Opinion: Getting past disease to the science of wellness - Los Angeles Times

SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting

Paris, March 19, 2021 SparingVision (the Company), a genomic medicine company developing vision saving treatments for ocular diseases, announces today that three abstracts highlighting the companys recent research into ocular diseases and its lead gene therapy treatment SPVN06 have been accepted for the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, to be held virtually from 1-7 May. The three abstracts will be given as poster presentations for which the details can be found below.

Title: SPVN06, a Novel Mutation-Independent AAV-based Gene Therapy, Protects Cone Degeneration in a Pig Model of Retinitis PigmentosaDate and Time: May 3, 2021 from 11:15 AM to 1:00 PM EDT

Presenter: Dr. Jennifer Noel, University of LouisvilleSession Title: Drug delivery and Gene Therapy

Title: Correlations between progression markers in rod-cone dystrophy due to mutations in RHO, PDE6A, or PDE6BDate and Time: May 3, 2021 from 4:30 PM to 6:15 PM EDT

Presenter: Dr. Daniel Chung, Chief Medical Officer, SparingVisionSession Title: Visual Impairment - Assessment and Measurement

Title: A 1-Month Toxicology and Biodistribution NHP Pilot Study Evaluating a Single Subretinal Bilateral Administration of SPVN06 - A Novel AAV-Based Gene Therapy for the Treatment of Rod-Cone Dystrophies Agnostic of the Causative Mutation Date and Time: May 5, 2021 from 2:45 PM to 4:30 PM EDT

Presenter: Dr. Melanie Marie, SparingVisionSession Title: AMD and retinal physiology

**ENDS**

Contacts:

NOTES TO EDITORS:

About SparingVision:SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised 60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit http://www.sparingvision.com.

About SPVN06:SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVisions primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2021.

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SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting - GlobeNewswire