Category Archives: Gene therapy

This years Nobel Prize for Chemistry has been awarded to Professor Emmanuelle Charpentier and Dr Jennifer Doudna for their discovery of natures sharpest genetic scissors CRISPR the tool that makes gene therapy possible. Gene therapy is an emerging key treatment for many conditions with an underlying genetic cause, including epilepsy.

The Nobel Prizes were first awarded in 1901 and are given annually for outstanding work in physics, chemistry, physiology or medicine, literature, economics, and the promotion of peace. The Chair of Nobel Committee for Chemistry, Professor Claes Gustafsson, said of this years award, There is enormous power in this genetic tool, which affects us all. This technology is a method that will lead to groundbreaking new medical treatments. This also marks the very first time in history that a Nobel Prize has been shared between two women.

The discovery of this gene editing tool known as CRISPR/Cas9 has revolutionised the life sciences and medicine. The tool is faster, cheaper and more accurate than previous techniques of editing DNA and has huge potential for a wide range of applications. Discovered in bacteria, CRISPR/Cas9 is a mechanism by which bacteria chop up the DNA of invading viruses hence the tool adopting the title of the sharpest genetic scissors. Bacteria then integrate these chopped up virus DNA into sections of their genome (the complete set of genes or genetic material present in a cell or organism). This process is known as CRISPR Clustered Regularly Interspaced Short Palindromic Repeats. These repeats are then used to detect and destroy similar viruses by Cas9 in the future.

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2020 Nobel Prize in Chemistry Awarded for Genetic Scissors that could revolutionise epilepsy treatment - Epilepsy Research UK

CRANBURY, N.J., Oct. 12, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive interim results from its CLN6 Batten disease gene therapy program, AT-GTX-501. The results are featured in a virtual poster presentation at the Joint 16th International Child Neurology Congress and 49th Annual Child Neurology Society Meeting being held October 12-23, 2020. The presentation is also available in the Events and Presentations section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events-and-presentations.

The Abigail Wexner Research Institute (AWRI) at Nationwide Childrens Hospital is conducting the ongoing Phase 1/2 clinical study of a single one-time intrathecal administration of AT-GTX-501 gene therapy for variant late-infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease. With no approved treatments, CLN6 Batten disease is a fatal neurologic disease that rapidly robs children of their ability to walk, speak, think, and see.

Clinical Data Highlights:Interim safety data are available for 13 children with CLN6 Batten disease. Interim efficacy data are available for the first 12 children reaching the 12-month timepoint and for eight children up to 24 months, post-administration of the AAV-CLN6 gene therapy.

Jeff Castelli, Ph.D., Chief Development Officer ofAmicus Therapeutics, stated, We are pleased to share these positive interim clinical data for our intrathecal AAV gene therapy with the CLN6 community. The data continues to suggest that our gene therapy has the potential to be a treatment option for children living with CLN6 Batten disease, an ultra-rare, debilitating condition that leads to progressive declines in cognitive and motor function, and often results in death early in life.

Emily de los Reyes, M.D., Ph.D., Principal Investigator of the CLN6 clinical trial at AWRI at Nationwide Childrens andProfessor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine, stated, I remain pleased with the progress of this trial as well as our collection of natural history data to further inform the results for the AAV-CLN6 gene therapy. The interim results show that this investigational gene therapy has the potential to slow the neurological disease progression in children with CLN6 Batten disease.

Regulatory interactions for AT-GTX-501 are ongoing and the Company expects to provide feedback on the path forward in 2021.

Amicus has exclusive rights to the CLN6 gene therapy program developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About AT-GTX-501AT-GTX-501 is a novel gene therapy in Phase 1/2 development for CLN6 Batten disease, a rare, fatal, inherited lysosomal disorder with no approved treatment that primarily affects the nervous system. AT-GTX-501 is dosed in a one-time intrathecal infusion to deliver a functional copy of the CLN6 gene to cells of the central nervous system. The therapy is designed to address the underlying enzyme deficiency that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., AT-GTX-501 was granted Rare Pediatric Disease and Orphan Drug designations by the United States Food and Drug Administration. In the EU, the Company holds PRIME and orphan medicinal product designations.

About Batten DiseaseBatten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these disorders, a defect in a specific gene triggers a cascade of problems that interferes with a cells ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden and die prematurely.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials and the prospects and timing of the potential regulatory approval of our product candidates. In particular, this press release relates to interim data from an ongoing Phase 1/2 study to investigate intrathecal administration of AAV-CLN6 gene therapy. The inclusion of forward-looking statements arising from this interim data, ongoing study and natural history preliminary data should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. The interim data and Phase 1/2 study discussed herein is inherently preliminary and early in the study, derived from a limited patient set, and later trial results with this patient set or others may not be consistent with these preliminary results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Amicus TherapeuticsAndrew FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Amicus TherapeuticsDiana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

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Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual Meeting of the Child...

LONDON--(BUSINESS WIRE)--Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GT005 for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). GT005 is an investigational one-time AAV-based gene therapy that is delivered under the retina and is intended to slow the progression of GA that can lead to blindness.

Fast Track designation was granted to GT005 for the treatment of people with GA who have specific mutations in their Complement Factor I (CFI) gene and low levels of the CFI protein in their blood. Enrolment in the Phase II EXPLORE study [NCT04437368] to evaluate GT005 in this group of people is underway.

Dry AMD is a life-altering diagnosis and there are currently no FDA-approved medicines available. Research suggests people with dry AMD who have certain CFI mutations that correlate with low CFI levels in the blood have a higher risk of developing AMD,1 said Nadia Waheed, M.D., MPH, Chief Medical Officer. We are pleased to receive Fast Track designation for our investigational gene therapy for this high-risk group. We look forward to working with the FDA as we advance our clinical programme evaluating the safety and effectiveness of GT005.

In addition to EXPLORE, Gyroscope also plans to initiate a second Phase II trial in 2020 that will evaluate GT005 in a broader group of people with GA.

The FDAs Fast Track programme streamlines the review of drugs for serious conditions without FDA-approved treatment options available. Fast Track designation gives applicants access to more frequent communication with the FDA throughout the review process, and the potential to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete.

About Age-Related Macular Degeneration (AMD) and Geographic Atrophy (GA)

AMD is a leading cause of blindness affecting an estimated 196 million people globally.2 AMD typically affects people aged 50 and older, and causes a gradual and permanent loss of central vision that worsens over time.3 There are no approved treatments for the dry form of AMD, which is the most common, impacting approximately 90% of people with the disease.4 As dry AMD advances it leads to GA, an irreversible degeneration of retinal cells. This vision loss can be devastating, severely impacting a persons daily life as they lose the ability to drive, read, and even see the faces of loved ones.

Gyroscope estimates that nearly 3.5 million people in the United States and EU5 European countries have GA, and that more than 100,000 people with GA have certain CFI mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD.5, 6, 7

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

References

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1 Kavanagh D, Yu Y, Schramm EC, et al. Rare genetic variants in the CFI gene are associated with advanced age-related macular degeneration and commonly result in reduced serum factor I levels. Hum Mol Genet. 2015;24(13):3861-3870.2 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106116.3 National Eye Institute. Age-Related Macular Degeneration. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration.4 Centers for Disease Control and Prevention. Age-Related Macular Degeneration. https://www.cdc.gov/visionhealth/basics/ced/.5 Friedman DS, O'Colmain BJ, Muoz B, et al. Prevalence of age-related macular degeneration in the United States [published correction appears in Arch Ophthalmol. 2011 Sep;129(9):1188]. Arch Ophthalmol. 2004;122(4):564-572.6 Rudnicka AR, Kapetanakis VV et al. Incidence of late-stage age-related macular degeneration in American whites: systematic review and meta-analysis. Am J Ophthalmol 2015;160:85-93.7 Data on File.

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Gyroscope Therapeutics Granted FDA Fast Track Designation for GT005, an Investigational Gene Therapy for Dry Age-Related Macular Degeneration -...

SOMERSET, N.J. (PRWEB) September 22, 2020

Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that George Buchman, Ph.D., Vice President, Preclinical & Process Development, Catalent Cell & Gene Therapy, will present at the World Vaccine Congress, which will take place virtually on Sept. 28 October 1, 2020.

On Sept. 30 at 4:05 p.m. EST, Dr. Buchman will present Next-Generation Vaccines and Oncolytic Viruses: Current Challenges and Future Promise, giving an overview of the benefits and limitations of oncolytic viruses as immunotherapies. His presentation will discuss the manufacturing and analytical considerations, as well as a collaborative study that evaluates a new technology to assess transfection efficiency.

Dr. Buchman joined Catalent through its acquisition of Paragon Gene Therapy in 2019. He has more than 30 years of experience in the biotech industry, and has held roles at companies including Life Technologies (now Thermo Fisher), Celera Genomics and Gene Logic. Dr. Buchman obtained a bachelors degree in biochemistry from Albright College, Reading, Pennsylvania, and a doctorate in biochemistry from University of Maryland.

For more information, please visit https://biologics.catalent.com/events/world-vaccine-congress/.

About Catalent Cell & Gene TherapyWith deep experience in viral vector scale-up and production, Catalent Cell & Gene Therapy is a full-service partner for adeno-associated virus (AAV) and lentiviral vectors, and CAR-T immunotherapies. When it acquired MaSTherCell, Catalent added expertise in autologous and allogeneic cell therapy development and manufacturing to position it as a premier technology, development and manufacturing partner for innovators across the entire field of advanced biotherapeutics. Catalent has a global cell and gene therapy network of dedicated, large-scale clinical and commercial manufacturing facilities, and fill-finish and packaging capabilities located in both the U.S. and Europe. An experienced partner, Catalent Cell & Gene Therapy has worked with industry leaders across 70+ clinical and commercial programs. For more information, visit biologics.catalent.com/cell-gene-therapy/

About CatalentCatalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs approximately 14,000 people, including around 2,400 scientists and technicians, at more than 45 facilities, and in fiscal year 2020 generated over $3 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com

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Advantages of Oncolytic Viruses as Immunotherapies to be Discussed by Catalent Gene Therapy Expert at World Vaccine Congress - PR Web

LONDONandNEW YORK, Sept. 22, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month results from the ongoing Phase 1/2 clinical trial(NCT03252847) of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), will be presented in an oral session at the EURETINA 2020 Virtual Meeting taking place October 2-4, 2020.

Details of the presentation are listed below. Data is embargoed until the date and time of presentation.

Title: Phase 1/2 Clinical Trial of AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month ResultsPresenter: Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACSDate and Time: Saturday, October 3, 10:45am EDT (4:45pm CEST)Session: EURETINA Session 11: Late Breaking & Reviews

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx announced six-month data from the ongoing Phase 1/2 MGT009 clinical trial, which demonstrated AAV-RPGR was generally well tolerated and produced significant improvement in vision in the dose escalation phase of the trial.

About AAV-RPGRAAV-RPGR is an investigational gene therapy for the treatment of patients with X-Linked Retinitis Pigmentosa (XLRP) caused by mutations in the eye specific form of theRPGRgene (RPGRORF15). AAV-RPGR is designed to deliver functional copies of theRPGRgene to the subretinal space in order to improve and preserve visual function.MeiraGTxand development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with mutations inRPGRORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by theU.S. Food and Drug Administration(FDA) and PRIME, ATMP and Orphan designations by theEuropean Medicines Agency (EMA).

About the Phase 1/2 MGT009 Clinical TrialMGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in theRPGRgene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patients other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in theRPGRgene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

AboutMeiraGTxMeiraGTx(Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs.MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team,MeiraGTxhas taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland,MeiraGTxintends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visitwww.meiragtx.com.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words expect, intend, plan, believe, project, forecast, estimate, may, should, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter endedMarch 31, 2020, as such factors may be updated from time to time in our other filings with theSEC, which are accessible on the SECs website atwww.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:MeiraGTxElizabeth (Broder) Anderson (646) 860-7983elizabeth@meiragtx.com

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Media:W2O pureChristiana Pascale(212) 257-6722cpascale@purecommunications.com

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MeiraGTx Announces Data from Ongoing Clinical Trial of AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa to be Presented at EURETINA 2020...

Market Study Report, LLC, adds a thorough analysis of the ' Hemophilia Gene Therapy market', offering a comprehensive report emphasizing every vital aspect of the business vertical. The study has collectively presented refined data characterized by market valuation, SWOT analysis, market participants, regional segmentation, and revenue forecasts, enabling stakeholders to make logical business decisions.

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Hemophilia Gene Therapy Market Comprehensive Analysis, Growth Forecast from 2020 to 2025 - AlgosOnline

When James Landon received a mantle cell lymphoma (MCL) diagnosis in 2017, he was told his disease was indolent, meaning it was slow growing and didnt need to be treated right away, especially since he was feeling fine.

But that changed in 2019. Landon, 50, an attorney in Tucson, Arizona, started to feel tired all the time. His physicians discovered that his white blood cell count was sky-high and his spleen was enlarged.

The first-line treatment for MCL has long been high doses of chemotherapy, often followed by a stem cell transplant. But when Landon traveled to The University of Texas MD Anderson CancerCenter in Houston to explore his options, he was offered an alternative: an experimental regimen combining the drugs Imbruvica (ibrutinib) and Rituxan (rituximab). Imbruvica blocks a protein called Bruton tyrosine kinase (BTK), which is a driver of MCL, and Rituxan is an antibody that targets overactive B cells of the immune system that have been implicated in the disease.

Five months after starting the combination four Imbruvica pills every morning and once-weekly infusions of Rituxan Landons positron emission tomography (PET) scan showed no evidence of disease. If he stays clear, hell undergo a short course of chemotherapy and then move to a maintenance therapy of one year of Ibrutinib and two of Rituxan.

Having this option has been fantastic, in my opinion, because the drugs so far have worked well for me, with no toxicity, says Landon, who adds that he has plenty of energy to work full time and play with his 10-year-old son.

BTK inhibitors and immunotherapy are among the newer therapeutic options for patients with MCL that are greatly improving the outlook for long-term survival. In addition to these targeted drugs, Tecartus (brexucabtagene autoleucel) the first cell-based gene therapy for MCL in patients who havent responded to or who have relapsed following other kinds of treatment was approved by the Food and Drug Administration (FDA) in July and is a one-time personalized treatment made from patients own immune cells.

We now have several good nonchemotherapy options for treating MCL, says Dr. Anthony Nguyen, a professor at the University of Nevada, Las Vegas School of Medicine and a medical oncologist at Comprehensive Cancer Centers of Nevada. We may be able to tell patients we can treat them without toxic chemotherapy, which can be reassuring, particularly for older patients.

MCL is a subtype of non-Hodgkin lymphoma (NHL) thats characterized by the overproduction of a protein called cyclin D1. In about 85% of patients, that overproduction is caused by a genetic abnormality called reciprocal chromosomal translocation, which can be detected with diagnostic testing of tumor samples. MCL accounts for about 6% of all NHL diagnoses and is more common in men than in women, according to the Leukemia & Lymphoma Society.

The standard first-line treatment for MCL is high-dose chemotherapy, often with a four-medicine regimen called hyper-CVAD, followed by a stem cell transplant with a patients own cells or with those from a donor. The regimen often puts patients into long-term remissions, but the side effects including nausea, mouth ulcers and kidney damage can be difficult or even dangerous, particularly for patients with other illnesses.

The newer medicines and cell therapy were approved by the FDA to treat patients with MCL who dont respond to chemotherapy and transplants or who relapse. But as physicians gain more experience with these therapies, theres a growing interest in using them earlier in the treatment process to not only improve the chances of long-term remissions, but also to improve the quality of life for patients by sparing them from harsh side effects.

The FDA approved the first BTK inhibitor to treat MCL, Imbruvica, in 2013, based on a study showing an overall response rate (meaning the disease responded to treatment) of 68% and a complete response rate (the disappearance of all signs of cancer) of 21%. The average period that patients lived without their disease progressing was more than a year, and side effects were mild stomach upset and fatigue.

The more recently approved BTK inhibitors Calquence (acalabrutinib) and Brukinsa (zanubrutinib) have improved on those response rates. Patients receiving Brukinsa in a late-stage trial, for example, had an overall response rate of 89% and a complete response rate of 59%.

Another targeted treatment, Venclexta (venetoclax) is also being studied in MCL. Venclexta targets the protein BCL2, which promotes cell survival and is abnormally elevated in MCL helping to drive progression of the disease. In a small trial of Venclexta, 75% of patients with relapsed MCL responded to the drug, 21% of whom had complete responses. Theres even more interest in studying BCL2 inhibition in combination with BTK blockers. In a recent study of Venclexta combined with Imbruvica, the median progression-free survival time was 29 months.

This is an extremely promising combination, says Dr. Abhijeet Kumar, assistant professor in the division of hematology and oncology at the University of Arizona College of Medicine. Kumar is an investigator in an ongoing trial of Venclexta and Imbruvica in MCL.

There is, however, a risk of increased side effects when targeted treatments are combined. Imbruvica can cause bleeding, for example, and both drugs can lower neutrophil (a type of white blood cell) counts. Venclexta is also known to cause tumor lysis syndrome, a rapid release of tumor cells into the bloodstream that can endanger the kidneys and other organs. Still, so far, the combination seems to be well-tolerated, Kumar says.

Another two-drug treatment for MCL that has generated enthusiasm among oncologists treating the disease is dubbed R-squared because it combines Rituxan with Revlimid (lenalidomide), a drug that works by boosting the immune systems T cells and natural killer cells, which work together to attack cancer.

In a study of R-squared in 38 patients with newly diagnosed MCL, the progression-free survival rate after three years was 80% and overall survival reached 90%. The response is durable, says Dr. Bijal Shah, an associate member in the department of malignant hematology at Moffitt Cancer Center and one of the study investigators. During the R-squared trial, patients typically stayed on the combination for three years and then took Revlimid alone as long as the disease remained stable.

Similar benefits have been seen with a combination of Velcade (bortezomib), Revlimid and chemotherapy, a regimen called VR-CAP. Velcade is a targeted drug that works by disrupting the growth of MCL cells and prompting them to die.

In a trial of patients with untreated MCL, adding Velcade to Revlimid and chemotherapy extended progression-free survival by 37%. The addition of Velcade more than doubled the median duration of response to 41 months.

Both R-squared and VR-CAP have moved into the frontline treatment setting, Shah says. With that, were able to see really pronounced clinical benefits. Weve seen very long remissions, he says.

Several other combination strategies also are being investigated for MCL, including some that incorporate the drug Treanda (bendamustine), which works by causing DNA damage to cancer cells. In one study, combining Treanda with Rituxan improved progression-free survival rates over chemotherapy in patients with MCL or indolent NHL. More than 15 studies are now underway combining Treanda with Rituxan and other MCL treatments.

Even though targeted and combination treatments have extended survival times in MCL, most patients eventually relapse. Now theres a new option for those patients: Tecartus, a personalized therapy made from a patients own immune cells. The one-time treatment was approved by the FDA to treat patients who have not responded or have relapsed following other kinds of treatment.

Tecartus is a chimeric antigen receptor (CAR)-T cell therapy similar to Yescarta (axicabtagene ciloleucel), a CAR-T cell therapy approved by the FDA in 2017 to treat some types of large B-cell lymphomas. Like Yescarta, Tecartus targets CD19, a protein thats prevalent in cancerous B cells. Tecartus is made by extracting T cells from the blood of the patient with MCL and genetically modifying those cells to recognize and attack the cancer. In addition, the cells are put through an enrichment process designed to prevent them from wearing down before they are infused back into the patient.

In the clinical trial that led up to the approval, 87% of patients responded to Tecartus and 62% went into remission. Side effects, which included the immune overreaction known as cytokine release syndrome and neurological events, were manageable during the clinical trial, says Dr. Michael Wang, a professor in the department of lymphoma and myeloma at The University of Texas MD Anderson Cancer Center and one of the clinical trial investigators.

Experience with previously approved CAR-T cell treatments led to the widespread use of anti-inflammatory medications such as interleuken-6 inhibitors and steroids to treat cytokine release syndrome, Wang says. We have a variety of supportive measures to manage the side effects, he says.

With the approval of Tecartus, Wang says oncologists can envision a flattening of the survival curve in MCL. Its an option for people who become resistant to targeted therapies and chemotherapy, he says. Its very possible we will be able to put some people into long- term remissions.

Bob Brixner, a 20-year survivor of MCL, has been watching all the new developments with interest. When he received an MCL diagnosis in 2000, he had no choice but to endure chemotherapy followed by a stem cell transplant with his own cells. When he relapsed in 2004, he was prescribed a more intense chemotherapy regimen, followed by a stem cell transplant from an unrelated donor.

Hes grateful the second treatment put him in a long-term remission, but he still remembers the brutal side effects, which included extreme fatigue and a bout with pneumonia. And with the transplants, my immune system didnt come back 100%, says Brixner, 70, a retired Chicago public schoolteacher. Nowadays if I catch a cold, instead of lasting a week, it will last three.

Brixner advises all newly diagnosed patients to ask a lot of questions about their treatment choices and to get a second opinion. I think its really important to be an informed patient, he says, especially since there are so many new choices. Some patients may not have to go through what I did, he says. Im delighted.

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Advances in the Treatment of Mantle Cell Lymphoma are Greatly Improving the Long-Term Outlook for Patients - Curetoday.com

No one knows for sure what causes Alzheimers disease. But one fact about the condition has acquired an almost irrefutable status. Depending on what variants of the APOE gene you inherit, the risk of brain disease can be half the average or more than 12 times as high.Often referred to as the forgetting gene, APOE comes in three popular forms, 2, 3, and 4. Type 2 decreases the risk of an individual, 3 is normal, and 4 increases the threat dramatically. The risk is so high that doctors would stop checking patients for APOE because a bad outcome may be upsetting, and theres nothing to do about it. There is no cure, and you cant change your genes, either.

Well, you cant today. But doctors in New York City say that beginning in May, they will start testing a novel gene therapy in which people with the unluckiest APOE genes will be given a huge dose to their brain of the low-risk version.In people who already have Alzheimers, if that slows the brain-wasting illness, it could eventually lead to a way of preventing the disease. The clinical trial, conducted at Weill Cornell Medicine in Manhattan by Ronald Crystal, is a novel tactic against dementia as well as a new gene therapy twist.

Most gene replacement efforts, which rely on viruses to carry DNA instructions into a person s cells, aim to fix rare diseases such as haemophilia by replacing a single malfunctioning gene.But there are no singular causes of common diseases, so gene therapy has never seemed so promising. A trade group, the Alliance for Regenerative Medicine, says it knows of no gene therapies currently being tested for Alzheimers disease patients.

Going into human clinical trials seems like a long shot, but there is a desperate need for any treatment, says Kiran Musunuru, a professor at the medical school of the University of Pennsylvania. Musunuru, who studies genetic treatments for heart disease, says the New York experiment represents a new category of gene therapy in which the goal is not to cure, but to reduce healthy peoples risk of future illnesses.

Crystal says his plan also sidesteps the debate about the real cause of Alzheimers disease, a multi-billion dollar roulette wheel where drug companies and patients continue to lose. In January, Roche launched two major antibody studies aimed at clearing up the characteristic plaques of a protein called beta-amyloid, the latest blow to the theory that the fundamental cause of Alzheimers is these plaques around neurons.

Crystal says, There are those in the field who strongly believe that amyloid does it, while others believe its another protein called tau, tangles of which are found in dying neurons. The answer is probably that its very complex, he says. The approach we have taken is to ignore all that and, from a genetic point of view, think about it.

Crystal s team is relying on a 25-year-old discovery in doing so. Duke University researchers went fishing for any proteins they could find attached to amyloid plaques in the 1990s. They identified the protein encoded by the APOE gene, apolipoprotein-e. They determined that one version, APOE4, was inexplicably common in those suffering from the disease by sequencing the gene in 121 patients.

The function of the gene is still not fully understood (it plays a role in transporting fats and cholesterol), but its status as a risk factor remains frightening. Roughly 65 percent of people with Alzheimers have at least one copy of the risky gene, according to the Alzheimers Association. Dementia becomes close to a sure thing for individuals born with two high-risk copies, one from each parent, if they live long enough.

Some individuals, though, inherit one 4 and one 2, the gene s lowest-risk version. These people are closer to the average risk, suggesting that the genes protective version offsets the risky one.This is the effect that the doctors at Weill Cornell will attempt to copy. The centre is now looking for individuals with two high-risk gene copies who already have memory loss, or even an Alzheimers diagnosis. Crystal says that starting in about a month, the first volunteers will receive a billion viruses carrying the 2 gene infusion into their spinal cords.

Crystal expects the viruses to spread the lucky gene to cells throughout the brains of the patients, based on tests in monkeys. Mice treated in the same way, his centre found, in their brains, accumulated less amyloid.The approach, Crystal says, does not depend on knowing all about what really causes the disease. What attracts us to Alzheimers is that it is so obvious from genetic epidemiology, he says. So the strategy is, can the brain be bathed in E2? Weve got the infrastructure to do it, so we were thinking, why not? The problem of the mechanism of the disease gets around it.

The concept is rational, adds Crystal. Another thing is whether it works within a human being.The study in New York is preliminary. Crystal states that his team needs to determine whether the added gene even works at a detectable level. Doctors will draw the patients spinal fluid and see if it contains the expected protein mix-the expected type 4, but now mixed in with an equal or greater amount of 2.

By the time people begin to forget names and where the car keys are, its a result of brain changes that started a decade earlier. That means that patients who are participating in the trial cant expect much. For them, its probably too late.Even so, Crystal is being given $3 million by the Alzheimers Drug Discovery Foundation to pay for the study, its largest grant to date. We dont know whats going to happen yet, says Nick McKeehan, the foundations assistant director. Its a stepping stone, though. Perhaps well need people to be treated earlier. It opens the door to this kind of therapy.

The hope is that middle-aged individuals with risky genes may eventually undergo one-time genetic tune-ups. Over time, even a small decrease in the pace at which brain changes occur could make a real difference.Alzheimers is the worlds most feared disease, because its horrifying to lose your mind. Susan Hahn, a genetic counsellor who doesnt think people should get their APOE gene tested for good reasons, says people would prefer to have cancer or a heart attack. Because its permanent, you have to be prepared for what you are going to hear. You cant change your genes, although you can perhaps do it with this study.

Source: MIT Technology Review

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Gene Therapy for Alzheimer's Disease Prevention - Credence Turbine

Axovant Gene Therapies Ltd. (NASDAQ:AXGT) has announced the signing of a strategic partnership with leading Contract Development and Manufacturing Organisation, Viralgen.

Viralgen, which is leveraging AskBios tech platforms, has supported Axovants all manufacturing aspects of the AAV program, including large scale manufacturing, quality control, and fill-finish GMP-certified environment that has been tailored to bring therapies to the market quickly. As per the terms of the agreement, Axovant will get access to manufacturing resources of its AAV-based gene therapy programs with adequate capacity to support the development and eventual commercialization of the therapies. Axovant has is developing the AXO-AAV-GM1 for GM gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis.

Gavin Corcoran, the Chief R & D Officer of the company, stated that they are pleased to collaborate with Viralgen, a leading manufacturer of AAV-based gene therapies. He said that the partnership will enable Axovant to produce their novel gene therapies that can improve or stabilize the course of GM1 and GM2 gangliosidoses on a commercial scale. The GM1 and GM2 gangliosidosis are two problematic pediatric diseases that do not have any approved treatment alternatives.

Corcoran said that the partnership with Viralgen offers the company access to a huge facility and experienced team that is focused on delivering tech that will speed up the development of life-saving therapies. He explained that the approach is important as the company continues to advance the AAV programs with data from the current Phase1/2 AXO-AAV-GM1 study expected in Q4 2020. The company is also expecting to file an IND clearance for AXO-AAV-GM2 before the end of this year.

Viralgen CEO Javier Garcia stated that they were delighted to partner with Axovant, and they are looking forward to offering priority access and support to advance AAV gene therapies for the devastating podiatric diseases. He added that the companys scalable and flexible production platform will be vital in complementing the development efforts of Axovant through commercialization from clinical trials.

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Axovant Gene Therapies Ltd. (NASDAQ:AXGT) Partners With Viralgen To Scale Production Of Its AAV Gene Therapies - BP Journal

The global Genetic Modification Therapies market report provides geographic analysis covering regions, such as North America, Europe, Asia-Pacific, and Rest of the World. The Genetic Modification Therapies market for each region is further segmented for major countries including the U.S., Canada, Germany, the U.K., France, Italy, China, India, Japan, Brazil, South Africa, and others.

The global Genetic Modification Therapies market is expected to exceed more than US$ 3.5 Billion by 2024 at a CAGR of 34% in the given forecast period.

Genetic modification therapies, significantly gene therapy and RNA therapy, have existed for many years, with very little clinical success. However, recent enhancements in these therapies, together with higher delivery systems, additional economical and sturdy gene expression constructs, precise polymer editing tools, have brought this industry to the forefront, and its currently poised for explosive growth within the coming back years.

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Because of the potentially curative nature of those medicines theres monumental potential in several applications, starting from cancer to neurology to rare diseases. Genetic modification therapies represent consecutive wave of medicines with monumental potential for treating and curing draining and high diseases. As a result of its wide scope, genetic modification therapy can play a vital role within the future world medical economy.

Continuing advances in key technologies like DNA editing, viral design and production, and gene expression, further as a pressing medical want in several serious and enervating disorders, are driving the expansion of the marketplace for genetic modification therapies. Developments in these multidisciplinary fields promise to advance the genetic modification therapies trade and build distinctive market opportunities.

The overall market is anticipated to witness important growth in opportunities for a spread of stakeholders within the returning decade. its necessary to spotlight that many technology suppliers, reaching to develop and / or support the event of gene therapies, with improved effectiveness and safety, have designed and already introduced advanced platforms for the engineering of vectors. Innovation during this domain has additionally semiconductor diode to the invention of novel molecular targets and strong the analysis pipelines of corporations targeted during this house. the potential to focus on numerous therapeutic areas is taken into account to be amongst the foremost outstanding growth drivers of this market.

Market Insights

The global Genetic Modification Therapies market is segregated on the basis of Platform Technology as Gene editing, Gene Therapies, Genetically Modified Cell Therapies, and RNA Therapies. Based on Delivery Technologies the global Genetic Modification Therapies market is segmented in AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral, and Nonviral Based on End-User Industry the global Genetic Modification Therapies market is segmented in Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations, and Others.

Based on Disease, the global Genetic Modification Therapies market is segmented in Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases, Other Indications.

Competitive Rivalry

4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others are among the major players in the global Genetic Modification Therapies market. The companies are involved in several growth and expansion strategies to gain a competitive advantage. Industry participants also follow value chain integration with business operations in multiple stages of the value chain.

The Genetic Modification Therapies Market has been segmented as below:

The Genetic Modification Therapies Market is segmented on the lines of Genetic Modification Therapies Market, By Platform Technology, Genetic Modification Therapies Market, By Delivery Technologies, Genetic Modification Therapies Market, By End-User Industry, Genetic Modification Therapies Market, By Disease, Genetic Modification Therapies Market, By Region and Genetic Modification Therapies Market, By Company.

Genetic Modification Therapies Market, By Platform Technology this market is segmented on the basis of Gene editing, Gene Therapies, Genetically Modified Cell Therapies and RNA Therapies. Genetic Modification Therapies Market, By Delivery Technologies this market is segmented on the basis of AAV, Adenovirus, Lentivirus, Retrovirus, Other Viral and Nonviral. Genetic Modification Therapies Market, By End-User Industry this market is segmented on the basis of Hospitals, Diagnostics and Testing Laboratories, Academic and Research Organizations and Others. Genetic Modification Therapies Market, By Disease this market is segmented on the basis of Cardiology, Oncology, Ophthalmology, Hematology, Musculoskeletal, Neurology, Rare Diseases and Other Indications. Genetic Modification Therapies Market, By Region this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World. Genetic Modification Therapies Market, By Company this market is segmented on the basis of 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore and Jivana Biotechnology.

The report covers:

Report Scope:

The global Genetic Modification Therapies market report scope includes detailed study covering underlying factors influencing the industry trends.

The report covers analysis on regional and country level market dynamics. The scope also covers competitive overview providing company market shares along with company profiles for major revenue contributing companies.

The report scope includes detailed competitive outlook covering market shares and profiles key participants in the global Genetic Modification Therapies market share. Major industry players with significant revenue share include 4D Molecular Therapeutics, Abeona Therapeutics, Beam Therapeutics, Casebia Therapeutics, Editas Medicine, Fate Therapeutics, GE Healthcare, Hitachi Chemical Advanced Therapeutics, Immunocore, Jivana Biotechnology, and others.

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Genetic Modification Therapies Market Share, Growth, Trends And Forecast To 2026 - The Daily Chronicle