Category Archives: Gene therapy

BOSTON--(BUSINESS WIRE)--BDOs BioProcess Technology Group (BPTG), the world leader in providing strategic, technical, regulatory, and business consulting services to the biopharmaceutical industry, will be presenting at the upcoming 2022 BIO International Convention, June 13-16, 2022 in San Diego. The annual convention, hosted by Biotechnology Innovation Organization (BIO), is the worlds largest industry gathering and has returned live and in person for the first time since the start of the COVID-19 pandemic.

Patti Seymour, managing director in BDOs BioProcess Technology Group, will chair the panel about cell and gene therapy development, on Tuesday, June 14, from 12:00 PM to 12:45 PM at the Emerging Therapies Track within the BPI Theater. Her discussion will focus on factors impacting the decision to develop one therapy type over another.

Nick Vrolijk, Ph.D., managing director in BDOs BioProcess Technology Group, will chair a panel about business and manufacturing strategies for therapeutic cell and gene therapy modalities, on Wednesday, June 15, at the Emerging Therapies Track, within the BPI Theater from 12:00 PM to 12:45 PM. The roundtable discussion will cover new technologies to compress timelines, increase capacity and reduce costs for cell and gene therapy development.

BPTG has presented at this conference in 2019, and for several years prior, before being acquired by BDO. The four-day convention presents an opportunity for BPTG to demonstrate its expertise in bioprocess manufacturing to senior biotech executives, business development leaders and investors.

BDOs BioProcess Technology Group is proud to exhibit and speak alongside biotech and pharmaceutical organizations showcasing groundbreaking development at this years BIO Convention, said Patti Seymour, Managing Director of BDOs BioProcess Technology Group. These organizations are on the frontiers of medical breakthroughs, and were excited to show how we can partner to help them solve their greatest challenges including CMC product development, quality, regulatory and compliance, and manufacturing strategy and operations.

To learn more about BDOs Life Sciences practice and our services, connect with BPTG professionals at booth 1414 and Biodefense professionals at booth 5113.

Visit our website for a complete overview of BDOs presence at BIO 2022.

About BDOs BioProcess Technology Group

BPTG is the recognized worldwide leader in biologics CMC consulting, providing a full range of technical, regulatory, and strategic assistance to pharmaceutical and biotechnology companies in the development and commercialization of biopharmaceutical products. The company works with clients globally to find and apply solutions to the challenges of biopharmaceutical product development from clone to commercial. BPTG helps develop manufacturing processes and strategies that enhance the overall value of client companies, de-risk biopharmaceutical development, and accelerate the advancement of new products from clone to clinic. BPTG also helps investors, service providers, and companies make informed decisions by providing technical due diligence services and business evaluations of new products as well as technologies for product discovery, development, or commercialization. Learn more about BPTG and our services here. Follow us on Twitter.

About BDO USA

BDO is the brand name for BDO USA, LLP, a U.S. professional services firm providing assurance, tax, and advisory services to a wide range of publicly traded and privately held companies. For more than 100 years, BDO has provided quality service through the active involvement of skilled and committed professionals. The firm serves clients through more than 65 offices and over 740 independent alliance firm locations nationwide. As an independent Member Firm of BDO International Limited, BDO serves multi-national clients through a global network of more than 91,000 people working out of over 1,600 offices across 167 countries.

BDO USA, LLP, a Delaware limited liability partnership, is the U.S. member of BDO International Limited, a UK company limited by guarantee, and forms part of the international BDO network of independent member firms. BDO is the brand name for the BDO network and for each of the BDO Member Firms. For more information please visit: http://www.bdo.com and follow us on LinkedIn.

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BDO's BioProcess Technology Group to Present at the 2022 BIO International Convention - Business Wire

AURORA | A new institute to develop more advanced cell and gene therapies is coming to the University of Colorado Anschutz Medical Campus thanks to a historic financial commitment by the Gates Frontiers Fund, a Colorado-based foundation.

The Gates Institute will be a partnership between CU Anschutz and the fund. Investments from the two partners are expected to grow to $200 million in the next five years, according to CU Anschutz officials.

The institute will build on the work of the Gates Center for Regenerative Medicine and Gates Biomanufacturing Facility, which university officials say have conducted groundbreaking stem cell research for cancer and rare diseases, pioneering new therapies in recent years.

The institute will give CU Anschutz scientists the opportunity to develop more effective cell and gene therapeutics, which have shown potential in fighting cancer. The institute will be led Dr. Terry Fry, a professor of pediatric oncology at the CU School of Medicine who has developed immunotherapy treatments for pediatric leukemia patients.

The Gates Institute will make it possible for our faculty to achieve the vast potential of cell and gene therapies, CU School of Medicine Dean John J. Reilly, Jr. said in a statement. With previous support, we have recruited talented faculty and staff, and we have invested in state-of-the-art equipment. With this investment, we will build on that foundation so our scientists can develop a new generation of therapies that allow our clinicians to offer hope to those facing serious disease.

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CU Anschutz receives $200m for cell and gene therapy institute - Sentinel Colorado

RESEARCH TRIANGLE PARK Opus Genetics welcomed a new executive who will lead the gene therapy companys clinical development and regulatory affairs.

Jennifer Hunt was named the chief development officer for the company, which launched last year with a $19 million investment from the Retinal Degeneration Fund, earlier this week.

In the role, Hunt will be responsible for progressing and expanding the companys gene therapy pipeline, the company said in a statement.

The company recently formed a new partnership, and announced plans for a third clinical trial. It added two executives in October 2021, according to prior reporting from WRAL TechWire.

Hunt brings 25 yeas of experience to the role, having served in clinical and regulatory positions at several biopharmaceutical companies, including Genzyme, Voyager Therapeutics, and Editas Medicine.

Opus is in a strong position to advance multiple therapies for inherited retinal diseases, said Hunt in a statement.

Opus Genetics pipeline includes three preclinical candidates, known as OPGx-001, OPGx-002 and OPGx-003. The gene therapy candidates aim to address forms of Leber congenital amaurosis, according to the company.

Were pleased to welcome Jennifer at this exciting time for Opus, as we build out the team that will be foundational to realizing our mission to efficiently advance therapies for inherited retinal diseases, said Ben Yerxa, Ph.D., the CEO of the Foundation Fighting Blindness and the Retinal Degeneration Fund, who is also the acting CEO of Opus Genetics, in a statement. Jennifers deep clinical development background and experience overseeing trials in rare diseases and LCA will be instrumental as we progress our current programs toward the patients that need these therapies.

Startup spotlight: Opus Genetics, with $19M, works to advance cures for blinding retinal diseases

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RTP gene therapy startup Opus Genetics adds industry vet to executive team - WRAL TechWire

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market Research Report by Type (Antigen Gene Therapy, Cancer Gene Therapy, and Cytokine Gene Therapy), Vector Type, Application, Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2027 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Gene Therapy Market size was estimated at USD 2,828.62 million in 2021, USD 3,463.59 million in 2022, and is projected to grow at a Compound Annual Growth Rate (CAGR) of 22.62% to reach USD 9,617.32 million by 2027.

In this report, the years 2019 and 2020 are considered historical years, 2021 as the base year, 2022 as the estimated year, and years from 2023 to 2027 are considered the forecast period.

Cumulative Impact of COVID-19:

The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window:

The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects.

It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

Market Share Analysis:

The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others.

Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

The report answers questions such as:

Key Topics Covered:

1. Preface

1.1. Objectives of the Study

1.2. Market Segmentation & Coverage

1.3. Years Considered for the Study

1.4. Currency & Pricing

1.5. Language

1.6. Limitations

1.7. Assumptions

1.8. Stakeholders

2. Research Methodology

2.1. Define: Research Objective

2.2. Determine: Research Design

2.3. Prepare: Research Instrument

2.4. Collect: Data Source

2.5. Analyze: Data Interpretation

2.6. Formulate: Data Verification

2.7. Publish: Research Report

2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

5.1. Market Dynamics

5.1.1. Drivers

5.1.1.1. Significant way of treatment to eradicates the practice of surgery, drugs, or other procedures that lead to side effects on the health of individuals

5.1.1.2. Innovation in gene therapy for rare & cardiovascular diseases treatment coupled with the awareness regarding the ability of gene therapy

5.1.1.3. Application in the treatment of various diseases such as cancer, haemophilia, parkinson's disease, and HIV

5.1.1.4. Government support and ethical acceptance of gene therapy for cancer treatment

5.1.2. Restraints

5.1.2.1. High cost associated with the gene therapies

5.1.3. Opportunities

5.1.3.1. Upsurge in a number of gene therapy treatment centers in developed countries

5.1.3.2. Increasing adoption for gene therapy for the oncological disorder

5.1.3.3. Growing strategic alliances and ongoing research for orphan diseases

5.1.4. Challenges

5.1.4.1. Potential disorders associated with the immune response

5.2. Cumulative Impact of COVID-19

5.3. Cumulative Impact of 2022 Russia Ukraine Conflict

6. Gene Therapy Market, by Type

6.1. Introduction

6.2. Antigen Gene Therapy

6.3. Cancer Gene Therapy

6.4. Cytokine Gene Therapy

6.5. Suicide Gene Therapy

6.6. Tumor Suppressor Gene Therapy

7. Gene Therapy Market, by Vector Type

7.1. Introduction

7.2. Non-viral Vectors

7.3. Viral Vectors

8. Gene Therapy Market, by Application

8.1. Introduction

8.2. Cardiovascular Diseases

8.3. Genetic Diseases

8.4. Infectious Diseases

8.5. Neurological Diseases

8.6. Oncological Disorders

For more information about this report visit https://www.researchandmarkets.com/r/mfwu95

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Gene Therapy Market Research Report 2022: Antigen Gene Therapy, Cancer Gene Therapy, and Cytokine Gene Therapy - Global Forecast to 2027 - Cumulative...

--ARU-1801 reducedvaso-occlusive events and days in the hospital for patients with severe sickle cell disease--

MILLBURN, N.J. and BASEL, Switzerland, May 12, 2022 /PRNewswire/ -- Aruvant Sciences ("Aruvant"), a private company focused on developing gene therapies for rare diseases, announced that data demonstrating the clinical benefit of the company's lead product candidate ARU-1801 for severe sickle cell disease (SCD) will be presented today in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25th annual meeting.Punam Malik, M.D., Director of the Cincinnati Comprehensive Sickle Cell Center and Program Leader of the Hematology and Gene Therapy Program at the Cincinnati Children's Hospital Medical Center, will deliver the oral presentation this afternoon at 1:30 PM EDT.

"The data demonstrate that the ARU-1801 gene therapy may not only be able to reduce severe vaso-occlusive events (VOEs) but also reduce days in the hospital for SCD patients which could provide a clinically meaningful benefit for patients and help reduce health care costs," said Dr. Malik. "ARU-1801 is the only gene therapy in development that has been shown to achieve durable responses in patients with severe SCD using only reduced intensity conditioninga key differentiator from other investigational gene therapy and gene editing regimens."

Dr. Malik will be presenting data from the ongoing MOMENTUM Phase 1/2 clinical trial that is examining ARU-1801, an autologous lentiviral cell therapy with a modified, highly potent gamma globin payload, in individuals with severe SCD. ARU-1801 is designed to address the limitations of current curative allogeneic transplant options, such as low donor availability, the risk of graft-versus-host disease and toxicity from myeloablative chemotherapy. Unlike investigational gene therapies that require fully myeloablative conditioning, ARU-1801 is given with reduced intensity conditioning (RIC), which is a lower dose chemotherapy associated with less chemotherapy side effects including shorter time in the hospital and less short- and long-term toxicity than myeloablative approaches.

The data being presented demonstrates:

The ASGCT annual meeting is taking place in Washington, D.C. and virtually starting today, May 16, and will go through to May 19, 2022. For more information about the conference, please visit https://annualmeeting.asgct.org/.

About Aruvant SciencesAruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talentedteamwith extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an activeresearchprogram with a lead product candidate, ARU-1801, in development for individuals suffering fromSCD. ARU-1801, an investigational lentiviral gene therapy, is being studied in aPhase 1/2 clinical trial,the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high morbidity and mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit https://sicklecellstudies.com/, and for more on the company, pleasevisitwww.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciences, Instagram @Aruvant_Sciencesor LinkedIn @AruvantSciences.

SOURCE Aruvant Sciences

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Aruvant Announces Oral Presentation at American Society of Gene and Cell Therapy (ASGCT) - PR Newswire

Facility in Alachua, Florida Is Expected to be Completed in the Fourth Quarter of 2022

GAINESVILLE, Fla. andCAMBRIDGE, Mass., May 10, 2022 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation(Nasdaq: AGTC), a clinical stage biotechnology company focused on the development of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, recently hosted a topping off ceremony for its state-of-the-art gene therapy manufacturing facility during the BioFlorida Celebration of Biotechnology, signaling the completion of the buildings exterior structure.

Leasing our own manufacturing facility is an essential part of our strategy to support the near term production of materials for upcoming clinical trials and eventually the potential production of commercial supply for any approved products, said President and CEO of AGTC, Sue Washer. We were thrilled to host this event as part of BioFloridas Celebration of Biotechnology and contribute to a growing biotech presence in the state. We look forward to the grand opening later this year and welcoming everyone to the facility.

The new manufacturing building is part of the Companys strategy to exert more control over its manufacturing needs and enable a more rapid filing of a Biologics Licensing Application and, if approved by the FDA, support the commercial launch of its XLRP candidate. The facility is also expected to support more rapid advancement of the Companys product pipeline, providing improved quality assurance and supply chain redundancy while reducing manufacturing risk.

The event, held on May 5, began with remarks from BioFloridas President & CEO, Nancy Bryan, Matt Cason, President of Concept Companies, the project developer, and Sue Washer, President and CEO of AGTC. The event was attended by many in the local biotech community as well as the vendors and sub-contractors supporting the project. To commemorate the event, key members of the project team signed the last beam to be put in place this week.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address unmet patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3). Its preclinical programs build on the companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Bionic Sight, an innovator in the emerging field of optogenetics, and retinal coding and Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. For more information, please visithttps://agtc.com/.

Forward-Looking StatementsThis release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs, including statements about the potential of the companys gene therapy platform, the strength of its XLRP clinical program, whether clinical trial results will support future regulatory filings, the expected timing for completion of the manufacturing facility and the potential for the new manufacturing facility to support AGTCs pipeline programs. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due toa number ofimportant factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; risks and uncertainties related to funding sources for our development programs; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Companys business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the companys most recent annual report on Form 10-K, as it may be supplemented by subsequent periodic reports filed with theSEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR Contact:

Kerry Sinclair

Spectrum Science Communications

ksinclair@spectrumscience.com

Corporate Contact:

Jonathan Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

jlieber@agtc.com

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AGTC Hosts Topping Off Ceremony for Gene Therapy Manufacturing Facility to Celebrate the Building's Structural Completion - GlobeNewswire

TheUniversity of Alabama at Birmingham Callahan Eye Hospital has been named one of the newest sites for Spark Therapeutics Luxturna, the first gene therapy treatment approved by the Food and Drug Administration for a genetic disease.

It is a prescription gene therapy for patients with inherited retinal disease due to mutations in both copies of the RPE65 gene, which can be confirmed only through genetic testing. Patients must have viable retinal cells as determined by the treating physicians.

Leber congenital amaurosis (LCA) is one retinal degenerative condition and a leading cause of genetic blindness in children. Patients with LCA start to lose their vision in the first five years of life, and it gets progressively worse as they age. Most patients are considered legally blind due to the profound vision loss it causes.

One subtype is caused by inherited mutations in both copies of the RPE65 gene. When patients have mutations in both copies of their RPE65 gene, the normal visual cycle cannot take place and retinal cells die over time.

Luxturna uses a non-disease-causing virus to deliver a normal copy of the RPE65 gene to retinal cells, enabling them to make proteins that have the potential to make the visual cycle work properly again.

Dr. Jason Crosson and Dr. Richard Feist Jr. of Retina Consultants of Alabama will treat patients with RPE65 LCA, also known as LCA 2, at UAB Callahan Eye Hospital.

We are excited to offer patients with this debilitating condition the opportunity to see more clearly in low-light environments for the first time in their lives, said Dr. Dawn DeCarlo, director of the UAB Center for Low Vision Rehabilitationin theMarnix E. Heersink School of Medicine. Patients in our area who were previously identified as good candidates for Luxturna have had to travel to other states to receive treatment. It is exciting that we will now not only be able to offer patients from Alabama treatment right here at UAB Callahan Eye Hospital, but we will also be a destination treatment center for patients throughout the Southeast.

UAB is now one of 14 treatment locations in the nation, and one of the few sites in the Southeast.

Our location, in Birmingham, is an asset because of our reputation as a top national eye center and the accessibility of our city for those living in the Southeast, said Brian Samuels, interim chair for the UAB Department of Ophthalmology and Visual Sciences. I am extremely proud of Drs. Paul Gamlin, Douglas Witherspoon, Dawn DeCarlo, Jason Crosson and Richard Feist Jr., who were instrumental in UABs becoming a designated treatment center.

We have already been notified there are patients from Alabama and the Southeast who are interested in receiving treatment here, Crosson said. We look forward to meeting our new patients soon and scheduling them for treatment.

This story originally appeared on theUAB News website.

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UAB Callahan Eye Hospital offering new gene therapy treatment for an inherited retinal disease - Alabama NewsCenter

ALBANY, N.Y., May 13, 2022 /PRNewswire/ --The value of the global particle therapy market stood at US$ 560.3 Mn in 2021. The global market is likely to rise at a CAGR of 8.6% during the forecast period, from 2022 to 2031. The global particle therapy market is anticipated to cross the value of US$ 1.2 Bn by 2031. Minimal risk of treatment-induced illnesses,risein cancer incidences, and increase in number of particle therapy centers throughout the world are projected to be the growth factors for the particle therapy market. Proton therapy's main objective is to enhance radiation therapy by employing proton beams in the treatmentof targeted tumors and radiosurgery.

A surge in the number of trials has demonstrated the positive effects of particle therapy in certain clinical settings, and breakthroughs in image guidance,beam delivery,as well astreatment planning are predicted to facilitatepositive particle therapy market projections during the forecast period. In addition, favorable clinical findings have resulted in a growth in the number of treatment centers globally, with some in the construction orplanningstages.

Companies in the global particle therapy marketare turning difficult cooperation initiatives and ideas into solutions fosteringsocial innovation to improve patient outcomes. Top research and care institutions are collaborating to promote radiotherapy advancements. Technological advancements in cyclotron devices are driving the market.

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Modernization of healthcare in terms of both infrastructure and services have pushed the healthcare industry to new heights, Stay Updated withLatest Healthcare Industry Research Reportsby Transparency Market Research:

Cancer Gene Therapy Market: Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market.

Gene Therapy Market: Increase in competition among the leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs to gain the first mover advantage is expected to boost the growth of the gene therapy market during the forecast period.

CAR T-cell Therapy Market: The CAR T-cell therapy is emerging as a breakthrough innovation in cancer treatment. Clinical trials conducted globally are showing promising results in end stage patients and especially, for patients suffering from Acute Lymphocytic Leukemia. Such findings are translating into revenue opportunities for companies in the CAR T-cell therapy market.

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Particle Therapy Market to Surpass Value of US$ 1.2 Bn by 2031, Observes TMR Study - PR Newswire

Genes can be delivered into a group of cells in a patient's body in two ways. The first, calledin vivo (in VEE-voh), is to inject the vector directly into the patient, aiming to target the affected cells.

The second, calledex vivo (ex VEE-voh), is to deliver the gene to cells that have been removed from the body and are growing in culture. After the gene is delivered, integration and activation are confirmed, and the cells are put back into the patient.

Ex vivo approaches are less likely to trigger an immune response, because no viruses are put into patients. They also allow researchers to make sure the cells are functioning properly before they're put in the patient. Several gene therapy successes useex vivo gene delivery as an alternative to bone marrow transplants.

Bone marrow containsstem cells that give rise to many types of blood cells. Bone marrow transplants are used to treat many genetic disorders, especially those that involve malfunctioning blood cells. Ideally, a "matched" donor, often a relative, donates bone marrow to the patient. The match decreases the chances that the patient's immune system will reject the donor cells. However, it's not always possible to find a match. In these cases, the patient's own bone marrow cells can be removed and the faulty gene corrected with gene therapy. The corrected cells can then be returned to the patient.

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Gene Delivery: Tools of the Trade - University of Utah

References

1. National Institutes of Health. Genetics Home Reference. Help me understand genetics. Accessed May 3, 2021. https://medlineplus.gov/download/genetics/understanding/primer.pdf2. FDA Commissioner. What is gene therapy? How does it work? US Food and Drug Administration. Accessed July 1, 2021. https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work3. Biology Online Dictionary. Monogenic disease. Accessed April 19, 2021. https://www.biologyonline.com/dictionary/monogenic-disease4. Griffiths AJF, Miller JH, Suzuki DT, et al. An Introduction to Genetic Analysis, 7th edition. W.H. Freeman; 2000.5. STAT Reports. The STAT guide to viral vectors, the linchpin of gene therapy. STAT News; 2019.6. Collins M, Thrasher A. Gene therapy: progress and predictions. Proc Biol Sci. 2015;282(1821):770-788.7. Guha TK, Wai A, Hausner G. Programmable genome editing tools and their regulation for efficient genome engineering. Comput Struct Biotechnol J. 2017;15:146-160.38. Koshravi MA, Abbasalipour M, Concordet J-P, et al. Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: a promising approach for gene therapy of beta thalassemia disease. Eur J Pharmacol. 2019;854:398-405.9. Ceasar SA, Rajan V, Prykhozhij SV, Berman J, Ignacimuthu S. Insert, remove or replace: A highly advanced genome editing system using CRISPR/Cas9. Biochimica et Biophysica Acta. 201610. Choi J. Huntingtons Outreach Project for Education, at Stanford: Gene Silencing. Accessed July 1, 2021. https://hopes.stanford.edu/gene-silencing/11. Balasubramanian S, Habegger L, Frankish A, MacArthur DG, et al. Gene inactivation and its implications for annotation in the era of personal genomics. Genes Dev. 2011;25(1):1-10.12. Grant SG, Campbell CE, Duff C, et al. Gene inactivation as a mechanism for the expression of recessive phenotypes. Am J Hum Genet. 1989;45:619-634.13. Ellebrecht CT, Bhoj VG, Nace A, et al. Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science. 2016;353(6295):179-184.14. Chandler RJ, Venditti CP. Gene therapy for metabolic diseases. Transl Sci Rare Dis. 016;1(1):73-89.15. Keeler AM, ElMallah MK, Flotte TR. Gene therapy 2017: progress and future directions. Clin Transl Sci. 2017:10(4):242-248.16. Rees H, Liu D. Base editing: prescision chemistry on the genome and transcriptome of living cells. Nat Rev Genet. 2018;19(12):770-788.17. American Society of Gene & Cell therapy (ASGCT). Gene and cell therapy FAQs. Accessed July 1, 2021. https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs18. NIH National Cancer Institute. NCI dictionary of cancer terms. CAR T-cell therapy. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/car-t-cell-therapy19. Almasbak H, Aarvak T, Vemuri MC. CAR T cell therapy: a game changer in cancer treatment. J Immunol Res. 2016;2016:5474602.20. National Cancer Institute. NCI dictionary of cancer terms: CRISPR-Cas9. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/crispr-cas921. Encyclopedia Britannica. Gene editing. Accessed July 1, 2021. https://www.britanica.com/science/gene-editing

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How does Gene Therapy Work | Types of Gene Therapy