StemCell Therapy

Management to Highlight Progress in Bone Marrow Transplant (BMT) and Chimeric Antigen Receptor T Cell (CAR T) Business Collaborations

Tel Aviv, Israel, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of innovative technology which enables the functional selection of cells facilitating safer and more efficacious cell and gene therapies, today announced that the leadership will be presenting at the Cell & Gene Meeting on the Mesa, which is being held from October 12th 16th. In addition to providing an overview of the Company, the management team will provide a progress update on the clinical and development programs and an overview of the Companys business strategy. Management will be virtually meeting cell and gene therapy companies to support collaborations.

This is one of the most prestigious meetings for our industry, and despite it being virtual this year due to the COVID-19 pandemic, it will not dampen our enthusiasm commented Dr. Yarkoni. We have successfully adjusted our operations, and we continue to make significant progress with our Israel and U.S.-based clinical trials. We are also taking meaningful steps to accelerate nearer-term revenue opportunities as we are collaborating with several partners that are looking to leverage our technology platform to help improve their products, especially in high-growth areas such as CAR T, NK (natural killers) and MSCs. We are looking forward to sharing the progress we have made and also look forward to meeting with current and potential partners developing cell and gene therapies.

To schedule a meeting with the Companys Chief Executive Officer, Dr. Shai Yarkoni or Chief Operating Officer, Amos Ofer, please request a meeting through the meeting portal and/or contact the Company direct at shai@cellect.co or amoso@cellect.co. The presentation will be available on the Companys website prior to the commencement of the meeting. The Cell & Gene Meeting on the Mesa is the sectors foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (APOP) has developed a breakthrough technology, for the functional selection of cells that aims to improve the robustness, safety and efficacy of a variety of cell and gene therapies. The Companys technology can be used by researchers, clinical community and pharma companies in a wide variety of applications including next generation Car T, NK, MSC and gene therapies.

The Company is also developing its own product that is an improved BMT which is in a current clinical trial for cancer treatment.

Forward Looking Statements

This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects expectations regarding timing of the commencement of its planned U.S. clinical trial and its plan to reduce operating costs. These forward-looking statements and their implications are based on the current expectations of the management of the Company only and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: the Companys history of losses and needs for additional capital to fund its operations and its inability to obtain additional capital on acceptable terms, or at all; the Companys ability to continue as a going concern; uncertainties of cash flows and inability to meet working capital needs; the Companys ability to obtain regulatory approvals; the Companys ability to obtain favorable pre-clinical and clinical trial results; the Companys technology may not be validated and its methods may not be accepted by the scientific community; difficulties enrolling patients in the Companys clinical trials; the ability to timely source adequate supply of FasL; risks resulting from unforeseen side effects; the Companys ability to establish and maintain strategic partnerships and other corporate collaborations; the scope of protection the Company is able to establish and maintain for intellectual property rights and its ability to operate its business without infringing the intellectual property rights of others; competitive companies, technologies and the Companys industry; unforeseen scientific difficulties may develop with the Companys technology; the Companys ability to retain or attract key employees whose knowledge is essential to the development of its products; and the Companys ability to pursue any strategic transaction or that any transaction, if pursued, will be completed. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.s Annual Report on Form 20-F for the fiscal year ended December 31, 2019 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SECs website, http://www.sec.gov, and in the Companys periodic filings with the SEC.

ContactCellect Biotechnology Ltd.Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

Or

EVC Group LLCMichael Polyviou(732) 933-2754mpolyviou@evcgroup.com

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Cellect Biotechnology to Present at the 2020 Cell & Gene Meeting on the Mesa - GlobeNewswire

Cell and Gene Therapy Market Research Report is a Proficient and In-Depth Study on the Existing State of Cell and Gene Therapy Industry. This Report Focuses on the Major Drivers, Restraints, Opportunities and Threats for Key Players. It also Provides Granular Analysis of Market Share, Segmentation, Revenue Forecasts and Regional Analysis till 2025.

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Cell and Gene Therapy Market Report 2020 Industry Capacity, Manufactur - News by aeresearch

T-knife GmbH, a next-gen adoptive T-cell company, and Catalent, have signed an agreement to provide technology transfer and CGMP clinical manufacturing of T-knifes T1367 T-cell receptor (TCR) program.

T1367 is an autologous T-cell receptor-based cell therapy derived from T-knifes proprietary humanized T-cell receptor (HuTCR) mouse platform and specifically targets MAGE-A1 positive tumors in cancer patients. The therapy is expected to be manufactured for clinical trials in both the EU and U.S.

Catalent will undertake transfer of T-knifes platform process for T-cell receptor-based cell therapy at its site in Gosselies, Belgium, with the goal of manufacturing clinical batches for European trials in 2021. T-knife will also prepare for the transfer of the TCR manufacturing platform to Catalents Houston, TX, facility for clinical trials in North America in the future.

The product candidates based on our proprietary HuTCR platform require sophisticated, state-of-the-art manufacturing capabilities and deep cell and gene therapy know-how, said Michael Buchholz, Director Manufacturing of T-knife. We are convinced that Catalent is the right partner for T-knife to ensure premier manufacturing of our pipeline programs, covering all stages from clinical trials to market.

Catalent is well-suited to support T-knife with focused technology transfer and process industrialization in both Gosselies and Houston, commented Manja Boerman, Ph.D., President, Cell & Gene Therapy, Catalent. Emerging and innovative treatments like T1367 are moving rapidly to the clinic. Catalent is committed to continual investment and expansion to support our clients as they continue on the journey to commercialization.

Catalents 25,830 sq.-ft. facility in Gosselies, Belgium, provides clinical through commercial-scale cell therapy manufacturing, for both autologous and allogeneic cell therapy treatments. An additional large-scale commercial manufacturing plant is currently under construction at the site and expected to be fully commissioned in 2021. The companys clinical manufacturing site in Houston is under qualification and expected to be fully commissioned in 2020.

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T-knife, Catalent Sign Tech Transfer And Mfg. Pact For Cell Therapy - Contract Pharma

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced it has named Thomas C. Gallagher, Esq. as the Companys Senior Vice President of Intellectual Property and Licensing. Mr. Gallagher has extensive experience in the area of biotechnology intellectual property (IP) law, business development, and licensing transactions with industry and academic institutions.

Mr. Gallagher will play a critical role in advancing our expanding intellectual property estate by spearheading IP strategy, which is an important element of the Companys overall success and value creation. A seasoned biotech executive, he will provide significant support as we execute on broadening our research and development programs, explore opportunities for partnerships on our existing programs, and opportunistically acquire new technologies to further expand our pipeline, said Rodney Varner, President and Chief Executive Officer of Genprex.

Mr. Gallagher has more than 20 years of experience as an intellectual property attorney. Prior to joining Genprex, he served as Principal at the Fenagh Group, an IP and licensing consultancy providing clients in the healthcare sector guidance on all aspects of patent and trademark portfolio management, intellectual property due diligence, freedom-to-operate analysis and related transactional work. He has also served as Senior Vice President of Intellectual Property and Licensing at Kadmon Corporation, LLC, a biopharmaceutical company based in Manhattan. Prior to joining Kadmon, he served as in-house IP counsel at Neostem Inc. (now Caladrius Biosciences, Inc.), a company focused on stem cell biology. Previously, he held several positions at ImClone Systems Incorporated, most recently as Vice President of Intellectual Property and Licensing. While at ImClone, he was responsible for all aspects of intellectual property and led the IP function in multiple due diligence undertakings by major pharmaceutical companies, which resulted in a $2 billion strategic investment, the highest-valued biotech deal ever at the time, and the eventual sale of the company to Eli Lilly and Company for $6.5 billion.

Mr. Gallagher is experienced in both patent prosecution and litigation, as well as intellectual property issues relating to business development and licensing matters. His patent litigation experience includes European litigation and opposition proceedings. In addition to a law degree, Mr. Gallagher holds a Masters degree in molecular biology. Before becoming an attorney, Mr. Gallagher worked as a molecular biologist in France, Spain and the United States.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, GPX-001 (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). GPX-001 has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. GPX-001 has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for GPX-001 for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of GPX-001, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of GPX-001 and our other potential product candidates including whether we receive fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex Names Seasoned IP Executive, Thomas C. Gallagher, as Senior Vice President of Intellectual Property and Licensing - Business Wire

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy - Business Wire

PARIS--(BUSINESS WIRE)--Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported that statistical analysis of pooled data from LUMEVOQ trials and natural history studies found a statistically significant and clinically meaningful difference between the visual outcomes in LUMEVOQ-treated patients and untreated patients.

Treated eyes showed progressive and sustained improvement from Month 12 to Month 52, in contrast to the absence of recovery over the same period for untreated eyes. At Month 18, the difference became statistically significant (p=0.01). By Month 48, the difference between the mean visual acuity in treated patients and that in untreated patients was both statistically significant (p<0.01) and clinically meaningful (-0.33 LogMAR, or +16.5 ETDRS letters equivalent, in favor of treated eyes).

Note: All patients had a confirmed G11778A mutation in the ND4 mitochondrial gene and were at least 15 years old. The diagram shows the Locally Estimated Scatterplot Smoothing (LOESS) curves for visual acuity in LUMEVOQ-treated patients and untreated patients. The shaded areas represent the 95% confidence interval for the mean BCVA. Treated eyes refer to all eyes (LUMEVOQ and sham) from the RESCUE, REVERSE and CLIN06 trials (N=76 patients / 152 eyes). Untreated eyes refer to patient-level data from the REALITY study and a matched data set from two prospective and eight retrospective natural history studies1 (N=208 patients / 408 eyes). LOESS curves were estimated using a non-parametric, local regression model that treated each eye as independent of the other. LOESS curves are shown from Month 12 to depict post-treatment progression among treated patients (93% of LUMEVOQ patients had already been treated within 12 months from onset). *Statistically significant difference between mean visual acuity of treated and untreated eyes at M18, M24, M36 and M48, as illustrated by the non-overlapping confidence intervals.

The analysis compared data from the completed Phase III trials RESCUE and REVERSE studies and interim results from the long-term follow-up CLIN06 study to a matched sample created from the REALITY registry study and 10 other natural history studies1. The natural history studies were identified from an extensive review of the scientific literature and selected based on specific inclusion criteria for their patient-level data. In all, the visual outcomes in 76 treated patients could be compared to the visual outcomes of 208 untreated patients.

The extra granularity of this analysis down to individual patient data confirms that the natural history of visual outcomes in ND4-LHON patients is poor and provides the best comparison we have for assessing the therapeutic efficacy of gene therapy, commented Dr. Nancy J. Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at the Emory University School of Medicine in Atlanta, GA, USA, and a global authority on LHON who recently completed a meta-analysis of the natural history of ND4-LHON.2

Separate analyses of patients enrolled in RESCUE and REVERSE demonstrated similarly favorable results compared to untreated patients. Full findings from the indirect comparison were included in the European Marketing Authorisation Application (MAA) for LUMEVOQ and are being prepared for publication in a peer-reviewed journal.

This indirect comparison represents a significant contribution to our understanding of LUMEVOQs therapeutic effect," said Dr. Jos-Alain Sahel, MD, Director of the Institut de la Vision (Sorbonne-Universit/Inserm/CNRS), Paris, France; Chairman of the Department of Ophthalmology at Centre Hospitalier National dOphtalmologie des XV-XX, Paris, France; Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center), USA; and Co-Founder of GenSight Biologics. The use of a large external control group, including analyses of patient-level data, provides more evidence for a significant treatment-related visual improvement.

The findings are a gratifying outcome of our push to overcome a key challenge for assessing LUMEVOQs efficacy, namely the inability of sham eyes to act as a control group, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. We are excited to take this evidence that LUMEVOQ modifies the disease outcome forward into our conversations with national and regional authorities.

The LUMEVOQ MAA was filed in September, and the decision is expected in H2 2021. The Company is also working towards submitting LUMEVOQs Biologics License Application (BLA) to the FDA in H2 2021.

GenSight will host a conference call today, September 21, 2020, at 10am CEST in French, and at 2.00pm CEST (8.00am EST) in English, to discuss these results.

Webcast & Conference call in French (10am CEST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0)1 7037 7166United Kingdom: +44 (0)20 3003 2666Password: GenSight FR

Webcast link: https://bit.ly/3mAS0Vy

Webcast & Conference call in English (2.00pm CEST / 8.00am EST)

Dial-in numbers:

United States: +1 212 999 6659France: +33 (0) 1 7037 7166United Kingdom: +44 (0) 20 3003 2666Password: GenSight ENG

Webcast link: https://bit.ly/35RCfnl

A replay of the calls and webcasts will be available by using the above links.

Rationale for the Indirect Comparison

In both the REVERSE and RESCUE studies, as well as in the long-term follow-up study CLIN06, unilaterally injected patients experienced an unexpected visual improvement in their contralateral eye, which mirrored the sustained and clinically relevant gain in eyes treated with LUMEVOQ. This bilateral improvement eliminated the control group that was to consist of sham eyes in the original trial design. An indirect comparison methodology, based on formal statistical methods applied to an external control group, was needed to assess the magnitude of LUMEVOQ efficacy.

Methodology Highlights

The sample of LUMEVOQ-treated patients included all data from the two pivotal studies REVERSE and RESCUE and Year 3 data from the ongoing extension study CLIN06. This approach yielded a treated patient pool consisting of 76 patients (152 eyes), with LUMEVOQ-injected eyes and sham-treated eyes considered equivalent, based on the contralateral effect demonstrated in the studies.

The external control group included data from the REALITY Natural History registry and patient-level data from 10 published articles1 on ND4-LHON, which were identified from a systematic review of the literature. ND4-LHON studies were included in the indirect comparison only if they had individual patient data that would allow indirect comparison with LUMEVOQ-treated patients: confirmed ND4 genotype, at least 15 years of age, at least one BCVA measurement with associated time of vision loss. The final external control group included 208 patients (408 eyes).

The visual acuity curves of treated and untreated patients were defined using a Locally Estimated Scatterplot Smoothing (LOESS), non-parametric, local regression model. They were aligned in terms of time from vision loss for the statistical tests performed at M12, M18, M24, M36 and M48 from vision loss and at the last available observation. The starting time point for the statistical tests (12 months post-vision loss) was defined to enable assessment of LUMEVOQ efficacy, as 93% of LUMEVOQ patients were treated by that time.

1The 10 studies that passed the inclusion criteria were: Hotta 1995, Lam 2014, Nakamura 1993, Newman 1991, Qu 2007, Qu 2009, Romero 2014, Sadun 2004, Yang 2016, and Zhou 2010.

2Newman NJ, Carelli V, Taiel M and Yu-Wai Man P. Visual outcomes in Leber hereditary optic neuropathy patients with the m.11778G>A (MTDN4) mitochondrial DNA mutation. J Neuro-Ophthalmol. In Press. 2020.

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQ (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About LUMEVOQ (GS010)

LUMEVOQ (GS010) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. LUMEVOQ was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint measures the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis was evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials were conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:REVERSE: NCT02652780RESCUE: NCT02652767

About CLIN06 (RESCUE and REVERSE Long-term Follow-up)

CLIN06 is the long-term follow-up study of ND4 LHON subjects treated with LUMEVOQ (GS010) gene therapy in the RESCUE or REVERSE Phase III Clinical Trials. The total study period for an individual subject is 3 years, i.e., 5 years post-gene therapy administration. No study treatment is administered during CLIN06.

The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment. The secondary objective is to assess the long-term treatment efficacy of the therapy and the quality of life (QoL) in subjects up to 5 years post-treatment. The first subject was enrolled on January 9, 2018. 61 subjects have enrolled.

ClinicalTrials.gov Identifiers:CLIN06: NCT03406104

About REALITY

REALITY is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on subjects medical charts and the administration of surveys on Health-Related Quality of Life (HRQoL) and direct and indirect costs associated with the disease.

The study aimed to recruit at least 50 subjects (both adult and pediatric) chiefly in the following countries: Spain, Italy, France, United Kingdom and the United States.

The primary objectives for the REALITY study were: to describe the evolution of visual functional and structural changes and other associated symptoms in patients with LHON; understand the impact of LHON-related vision loss on the HRQoL; and understand the economic burden for patients and their families arising from direct and indirect costs associated with the disease. The secondary objective is to describe the relationship between genetic, lifestyle and/or environmental factors and the expression of the LHON phenotype.

The first subject was enrolled on 3 January 2018. Enrollment was completed in early Q2 2020.

ClinicalTrials.gov Identifiers:REALITY LHON Registry: NCT03295071

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GenSight Biologics Reports New Analysis Demonstrating Statistically Significant and Clinically Meaningful Difference Between Visual Outcomes in...

The Global Urology Implants And Devices Market is projected to grow considerably on account of increasing incidence of urology disorders. Key insights into this development have been provided by Fortune Business Insights in its report, titled Urology Implants and Devices Market Size, Share and Global Trend By Product Type (Endoscopes, Lithotripsy Devices, Urodynamic Systems, Urostomy Bag, Endo Vision Systems & Peripheral Instruments, Urology Implants), By Disease (Kidney Disorders, Urology Cancers, Urinary Tract Disorders, Pelvic Organ Prolapse), By End User (Hospitals, Pharmacies, Ambulatory Surgery Centers (ASC)) and Geography Forecast till 2026.

The report covers:

Request a Sample Copy of the Research Report: https://www.fortunebusinessinsights.com/enquiry/request-sample-pdf/urology-implants-and-devices-market-100434

Leading Players operating in the Urology Implants and Devices Market are:

Key players are involved in mergers and acquisition to strengthen their market position. Owing to increasing competition frequent innovations are taking place in the market. Some of the companies operating the industry are:

Segmentation

By Products

By Disease

By End-Users

By Region

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Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

Gene Therapy Market

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Urology Implants and Devices Market Size 2020 Top Manufacturers, Share, Opportunities and Forecast to 2026 - The Daily Chronicle

Viral Vector and Vaccine Market report is an excellent report that makes it possible to the Viral Vector and Vaccine industry can be highly benefited with this market research report which brings market and competitive landscape clearly into the focus and help make better decisions. Market segmentation has also been performed in detail based on various parameters that include applications, verticals, deployment model, end user, and geography. Expert solutions combined with potential capabilities prepare this winning Viral Vector and Vaccine Market document to be outperforming for the Viral Vector and Vaccine

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Viral vector & vaccine market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing with the CAGR of 14.50% in the above mentioned forecast period. Rising prevalence of contagious and chronic diseases such astuberculosis, HIV, and cancer has given a boost to market for finding effective solution for these problems whereas upcoming new disease like with outbreak of COVID-19 has raised demand for minimal invasive technique for treatment for these rising medical problems.

The Leading Market Players Covered in this Report are :

Regional Analysis Includes:

Asia-Pacific-China, Japan, Korea, India, and Southeast Asia

Europe-Germany, France, UK, Russia, and Italy etc.

North America-The United States, Mexico, and Canada

South America-Brazil, Argentina, Columbia, etc.

The Middle East and Africa-Saudi Arabia, UAE, Egypt, Nigeria, and South Africa

Deep analysis about market status, enterprise competition pattern, advantages and disadvantages of enterprise products, industry development trends, regional industrial layout characteristics and macroeconomic policies, industrial policy has also be included.

Viral vector & vaccine market is segmented on the basis of type, workflow, application and end use. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Viral vector & vaccine market is segmented on the basis of type, workflow, application and end use. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

From raw materials to downstream buyers of this industry will be analyzed scientifically, the feature of product circulation and sales channel will be presented as well. In a word, this report will help you to establish a panorama of industrial development and characteristics of the Viral Vector and Vaccine Market.

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Recent Developments

o Market Overview and growth analysiso Import and Export Overviewo Volume Analysiso Current Market Trends and Future Outlooko Market Opportunistic and Attractive Investment Segment

Key Questions Answered by Viral Vector and Vaccine Market Report

1. What was the Viral Vector and Vaccine Market size in 2018 and 2019?; what are the estimated growth trends and market forecast?2. What will be the CAGR of Viral Vector and Vaccine Market during the forecast period (2020 2027)?3. Which segments (product type/applications/end-user) were most attractive for investments in 2018?How these segments are expected to grow during the forecast period (2020 2027).4. Which manufacturer/vendor/players in the Viral Vector and Vaccine Market was the market leader in 2018?5. Overview on the existing product portfolio, products in the pipeline, and strategic initiatives taken by key vendors in the market.

There are 13 Chapters to thoroughly display the Viral Vector and Vaccine Market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

Chapter 1: Viral Vector and Vaccine Overview, Product Overview, Market Segmentation, Market Overview of Regions, Market Dynamics, Limitations, Opportunities and Industry News and Policies.

Chapter 2: Viral Vector and Vaccine Industry Chain Analysis, Upstream Raw Material Suppliers, Major Players, Production Process Analysis, Cost Analysis, Market Channels and Major Downstream Buyers.

Chapter 3: Value Analysis, Production, Growth Rate and Price Analysis by Type of Viral Vector and Vaccine.

Chapter 4: Downstream Characteristics, Consumption and Market Share by Application of Viral Vector and Vaccine.

Chapter 5: Production Volume, Price, Gross Margin, and Revenue ($) of Viral Vector and Vaccine by Regions (2020 2027).

Chapter 6: Viral Vector and Vaccine Production, Consumption, Export and Import by Regions (2020 2027).

Chapter 7: Viral Vector and Vaccine Market Status and SWOT Analysis by Regions.

Chapter 8: Competitive Landscape, Product Introduction, Company Profiles, Market Distribution Status by Players of Viral Vector and Vaccine.

Chapter 9: Viral Vector and Vaccine Market Analysis and Forecast by Type and Application (2020 2027).

Chapter 10: Market Analysis and Forecast by Regions (2020 2027).

Chapter 11:Industry Characteristics, Key Factors, New Entrants SWOT Analysis, Investment Feasibility Analysis.

Chapter 12:Market Conclusion of the Whole Report.

Chapter 13:Appendix Such as Methodology and Data Resources of This Research.

Find More Competitor in TOC with Profile Overview Share Growth Analysis @https://www.databridgemarketresearch.com/toc/?dbmr=global-viral-vector-and-vaccine-market

Customization of the Report:

Data Bridge Market Research also provides customization options to tailor the reports as per client requirements. This report can be personalized to cater to your research needs. Feel free to get in touch with our sales team, who will ensure that you get a report as per your needs.

Thanks for reading this article; you can also get individual chapter wise section or region wise report versions like North America, Europe, or Asia Etc.

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GLOBAL CONGRESS ON ELDERLY CARE, GERONTOLOGY AND GERIATRICS

The world is ageing rapidly. People aged 60 and older make up 12.3 per cent of the global population, and by 2050, that number will rise to almost 22 per cent. With this surge many issues are underlying related to blood pressure, diabetes, heart failure issues, arthritis, cancer malignancy, joint pains, tuberculosis and majorly the lack of awareness regarding the changing behavioural patterns in elderly people at home leading to abuse of them by their kin.

The goal of this Conference is uniting all well-known gerontologists, geriatrics scholars, policy decision-makers, professional activists, related companies, and researchers to discuss and share knowledge in the emerging field of Aging and Gerontology and its related areas of research and how we can move towards more Healthy Living, Healthy Aging to Promote an Ageless Era and hence widening professional contact and create new opportunities, including establishing new collaborations to all the representatives.Registration Link: https://bit.ly/34ueelz

This content has been distributed via CDN Newswire press release distribution service. For press release enquires please mail us at contact@cdnnewswire.com.

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Viral Vector and Vaccine Market To 2027 Reporting And Evaluation Of Recent Industry Developments | - PharmiWeb.com

Gene Therapy Market - Snapshot

Introduction of new production, availability of reimbursement together with high occurrences of cancer are estimated to propel growth of the global gene therapy market in the years to come. Gene therapy refers to an experimental technique, which utilizes genes for the prevention and treatment of various diseases. It is expected that in the near future, this technology could assist doctors to place a gene into the cells of a patient for the purpose of his treatment. This therapy could be used as an alternative to surgery or drugs. Scientists are examining various approaches to this therapy, which could comprise

Gene therapy has emerged as a promising treatment option for a large number of diseases such as certain viral infections, certain cancers, and inherited disorders. This factor is likely to work in favor of the global gene therapy market in the years to come.

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Progress in Gene-editing and Genomics Tools to Stoke Demand of Gene Therapy

CAR T-cell use has recently garnered considerable attention from the sponsors following the exhibition of its immense promise in the treatment of several diseases. The promising future of CAR T-cell is estimated to amplify the growth opportunities of the global gene therapy market. Sponsors hail CAR T-cell use as a brand new business model of the future.

In the pipeline of pharmaceutical industry, gene therapy account for a considerable share and this trend is likely to continue in the years to come. In addition, significant advancement has been made in the fields of cellular and molecular biology is likely to fuel growth of the global gene therapy market in the years to come. rapid technological progress made in the gene-editing and genomics tools are further estimated to drive the demand for gene therapy.

Global Gene Therapy Market Snapshot

Expanding at a stellar, double-digits CAGR (Compound Annual Growth Rate) of 40% over the forecast period of 2018 to 2026, the global gene therapy market is a dizzying trajectory, marking out a rosy landscape for players operating in the playfield. As per a Transparency Market Research report, based on extensive primary and secondary research, states that over the period states, the market would accrue a worth of USD 5164.03 million a steep and impressive increase from the USD 17 million worth noted in 2017.

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Fixing defective genes via introduction of new and healthy ones to fix severe genetic and chronic disorders is seeing an upward curve in demand for reasons of improved medical outcomes, major leaps in terms of technological advancement and minimal die-effects as compared to alternatives. Some of the most significant alternatives include surgery, and drug use.

Some of the most significant factors that the analysts of the report note include focused efforts towards marketing and commercialization, and a slew of approvals of new products hitting the global gene therapy market. Additionally, there are factors such as growing demand experienced for this treatment by a large pool of patients.

It is pertinent to note here that the global gene therapy market is consolidated and is dependent on clinincal research and development of the highest standards in order to chart growth. And, some of the players that operate the market landscape, and are into significant research projects include Gilead Sciences, Inc., Novartis AG, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., CELGENE CORPORATION, and Orchard Therapeutics Limited.

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Yescarta to Dominate Global Gene Therapy Market over the Forecast Period

The global gene therapy market report by TMR is segmented based on type, application, and region. The former includes the only five products that have been approved so far for commercial use. These include Gendicine, Yescarta, Strimvelis, Kymriah, and Luxturna. Riding the first mover benefits, Yescarta helf the dominant position in the market in 2017, and the trend will continue, adding more worth to the sub-segment. This is the product that brought out the initial CAR T therapy in the market for large B-cell lymphoma that relapse.

It is worth noting here that as per the global gene therapy market report, the high incidence of DLBCL and massive commercialization efforts directed towards Yescarta, particularly in Europe, will contribute positively and significantly to the overall growth of the global gene therapy market. The other sub-segment to make a mark over the global gene therapy market landscape will be Luxturna, owing to rising awareness levels and massive efforts towards comercialization.

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Europe to be Ahead of the Global Gene Therapy Market Growth Curve over the Forecast Period

The global gene therapy markets regional segmentation includes incisive growth insights into some of the most significant areas that will shape up the overall growth in the market. These include North America, Europe, and Rest of the World. Researchers involved with the preparation of report claim that a massive chunk of about 40% would be accounted for by Europe over the forecast period. Some of the factors backing-up the market dominance of the region include high incidence of non Hodgkin lymphoma and increase in number of treatment centers into gene therapy.

This growth would be followed by North America region, owing to huge contributions from the United States of America which witnesses about 7500 cases of refractory DLBCL each year. These are ones that qualify for the CAR T therapy.

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Gene Therapy Market Progress in Gene-editing and Genomics Tools to Stoke Demand of Gene Therapy - BioSpace

It turns out a regulatory delay isnt the only bump on Sareptas road to gene therapy glory.

The biotech is being sued by Regenxbio, which is alleging that its slate of gene therapy programs, including two for Duchenne muscular dystrophy and limb-girdle muscular dystrophy infringes on a patent originally owned by the University of Pennsylvania.

The lawsuit, which was first reported by Bloomberg Law, adds another wrinkle as Sarepta strives to stay in the lead in a race to deliver the first genetic fix for Duchenne muscular dystrophy. After making its name with two antisense drugs neither of which has been proven to have an effect against the crippling disease the Cambridge, MA-based biotech has been viewed as the frontrunner versus Pfizer and trouble-prone Solid Bio. Roche was impressed enough to pay $1.15 billion to acquire ex-US rights to the program.

But Regenxbio wants Sarepta to stop stepping on their patents and pay up. The biotech, which is now based in Rockville, MD, is seeking damages for past, present and/or future infringement equaling no less than a reasonable royalty.

At the center of the dispute is US Patent No. 10,526,617. Jim Wilson, the gene therapy luminary who co-founded Regenxbio, was cited as an inventor on that patent, granted this January.

As it covers a cultured host cell containing a recombinant nucleic acid molecule encoding the capsid protein, the technology can be used to create adeno-associated vectors both in animal studies and for delivering a transgene into humans.

The vectors made using the claimed subject matter of the 617 Patent have unique properties, e.g., an ability to target certain types of cells in the body, the lawsuit states.

Specifically, Regenxbio alleges, Sareptas Duchenne program SRP-9001 is manufactured by a process that includes making and using a cultured host cell a recombinant nucleic acid molecule encoding an AAVrh74 vp1 capsid protein. The same capsid protein is also integral to SRP-9003, the limb girdle candidate, and a host of other follow-on gene therapies.

Regenxbio is suing as Sarepta is sorting out a new request from regulators that can push back its pivotal study for Duchenne. While CEO Doug Ingram assured investors that quality control issues such as this are not uncommon and pinned the delay on an overburdened FDA, he stopped short of promising concrete timelines.

Focused on retinal, metabolic and neurodegenerative diseases for its internal pipeline, Regenxbio is perhaps more accomplished as a gene therapy tech provider. Novartis and Abeona have both licensed its NAV tech platform.

It also doesnt shy away from legal actions. In its most recent quarterly report, Regenxbio disclosed that Abeona failed to make a $8 million payment due in April, effectively terminating their licensing agreement. In response to an arbitration claim Abeona filed in May alleging we breached certain responsibilities to communicate with Abeona regarding our prosecution of licensed patents, Regenxbio filed a counterclaim to ask for $28 million including $20 million that would have been owed under the pact.

Last November Regenxbio challenged the FDAs arbitrary and capricious decisions to issue a full clinical hold on its diabetic retinopathy trial and a partial hold on wet age-related macular degeneration. The company had withdrawn the IND for diabetic retinopathy, and the FDA lifted the partial hold for wet AMD two months later.

More:
Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent - Endpoints News

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Pfizer has spent $800 million to build the leading production platform for gene therapies, the company said on Tuesday in its second day of talks to show how it plans to become a fast-growing innovator. Within three years, the drug giant hopes to launch three potential cures for hemophilia and muscular dystrophyworth as much as $4 billion in annual revenue.

We are in an unrivaled position to go to market, said the head of Pfizers rare-disease business, Suneet Varma, in his Tuesday morning presentation. In another Tuesday talk, the companys immunology leaders highlighted coming breakthroughs for the tens of millions who suffer miserable autoimmune skin disorders.

Pfizer (ticker: PFE) is eager to recast itself as a high-multiple growth stock. By year-end, its slower-selling products will be spun off into a business run by Mylan (MYL). From that point, Pfizer thinks it can boost revenue by 6% a year, despite the patent expirations of nearly $20 billion worth of its products in the latter part of this decade. The expected growth will be fired up by 25 product launches, which Pfizer is explaining in two days of online talks.

After rising Monday, Pfizer stock was down 0.3%, at $36.91, in recent trading. The S&P 500 was up 0.4%.

A rare disease may affect only a few hundred thousand people, but there are a lot of these diseases. Cumulatively, they add up to 400 million people worldwide, said Varma, with fewer than 5% having approved treatments. Pfizer believes the rare-disease market will grow at a double-digit annual rate.

Clinical trials are under way for treatments for the bleeding disorders known as hemophilia A and hemophilia B. After treatment, none of the enrolled patients have had bleeding problems, including some in which more than a year has passed since dosing.

Pfizer hopes for approval and launch of its hemophilia B treatment in 2022. Peak annual sales could hit $500 million to $1 billion, says the company. The hemophilia A launch could happen in 2023 and eventually exceed $1 billion in annual sales.

The gene-therapy treatments can require a short hospital stay. But the possibility of a cure will inspire 30% to 40% of hemophilia patients to seek treatment, Pfizer predicts.

A rival in hemophilia A gene therapy, BioMarin Pharmaceutical (BMRN), had a head start over Pfizer in clinical trials. But last month, the U.S. Food and Drug Administration rebuffed BioMarins application for approval and demanded additional follow-up of its patients for a period that will extend through the end of 2021.

The FDA hasnt asked Pfizer to change its design for the hemophilia A trial that the company will begin in a few weeks, said the chief medical officer of the rare-disease unit, Brenda Cooperstone,

There were some worrisome immune reactions among boys in the continuing clinical trial for Pfizers gene therapy for Duchenne muscular dystrophy. But after adjustments in the trials preparatory regime, Pfizer says there have been no more of those events. The treatment showed promising efficacy in early phase trials.

The pivotal Phase 3 muscular dystrophy trial will begin within weeks, with the first data expected in 2022. Pfizer hopes for a launch in 2023 of a muscular dystrophy therapy that would be used by 30,000 people a year in the U.S. and Europe, generating over $2 billion in annual revenue. to reach that goal, Pfizer is racing with Sarepta Therapeutics (SRPT).

In addition to its rare-disease treatments, Pfizer plans to launch four products for autoimmune disorders by 2025. Two of the products would treat atopic dermatitis, which causes painful itching in up to 30 million Americans. Only a portion of those patients get any treatment today.

Based on successful clinical trials, sufferers of the skin disease can look forward to breakthrough treatments, similar to those launched in recent years for psoriasis, says Pfizers inflammation and immunology business head, Richard Blackburn.

Corrections & Amplifications

Pfizers gene-therapy treatments can require short hospital stays. An earlier version of this article incorrectly said the treatments are like bone-marrow transplants, which require months of grueling hospital care.

Write to Bill Alpert at william.alpert@barrons.com

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How Pfizer Plans to Lead the Industry in Gene Therapies - Barron's

China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs

CPhI Annual Report has launched ahead of the firstCPhI Festival of Pharma(5-16 October 2020), the worlds largest digital pharma Expo that predicts dramatic growth of new mAb production in China, capacity shortages for cell and gene therapies in the USA, and the widespread global adoption of single-use technologies, but only limited continuous bioprocessing.

Three CPhI experts from BioPlan Associates Vicky Qing XIA, Leo Cai Yang and Eric Langer explore the rapidly changing global biologics markets, with special reference to the implications for contract outsourcing and Chinas continued emergence as a hub for both bio innovation and contract services.

Remarkably, China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs predicted to be launched per year in the country by 2025. In fact, the total market size will quadruple by 2025, reaching 120bn RMB, and rising further to 190bn RMB by 2030.

According to the CPhI report, bioprocessing outsourcing in China is currently highly stratified with four tiers and just one domestic company intierone WuXi Biologics and a number of international CDMOs including BI, Lonza and Merck. However, by 2025 it is anticipated that as many as five more domestic CDMOs may have reached tier one status, with FDA and EU facility approvals.

Single Use Systems (SUS) are now far and away the leader at both pre-clinical and clinical stages, with nearly 85% now involving a substantial SUS component. Yet whilst its usage continues to grow, continuous bioprocessing is not anticipated to be in mainstream usage by 2025.

The report also suggested that in the US and Europe there is likely to be a cell and gene therapy capacity crunch by 2025, with CDMOs investing in this area already expanding to try and meet the pipelines demand.

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2025 will see cell & gene therapy capacity shortages in US: CPhI report - BSA bureau

Gene Therapy for Age-related Macular Degeneration Market research report is the new statistical data source added by A2Z Market Research.

Gene Therapy for Age-related Macular Degeneration Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

Gene Therapy for Age-related Macular Degeneration Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

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Top Key Players Profiled in this report are:

RetroSense Therapeutics, REGENXBIO, AGTC

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Gene Therapy for Age-related Macular Degeneration market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Gene Therapy for Age-related Macular Degeneration markets trajectory between forecast periods.

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The cost analysis of the Global Gene Therapy for Age-related Macular Degeneration Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

The report provides insights on the following pointers:

Table of Contents

Global Gene Therapy for Age-related Macular Degeneration Market Research Report 2020 2026

Chapter 1 Gene Therapy for Age-related Macular Degeneration Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Gene Therapy for Age-related Macular Degeneration Market Forecast

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Massive Growth in Gene Therapy for Age-related Macular Degeneration Market to Witness Astonishing Growth by 2026 | RetroSense Therapeutics, REGENXBIO,...

Technological advancements have blurred the borders of natural biological processes by giving humans more control. One such area is that of gene editing that allows us to modify the childs genetic make up to not only prevent diseases, and lead to a healthier life, but also personality traits for a more fulfilling life. However, this raises question on ethics, consent of the child and rights of parents. RAGHAV AHOOJA,addresses this issue with the lens of state involvement in controlling the private realm.

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WE are at crossroads wherein we may be able to customize and design the futures of our future generations. Lawmakers, thus, need to solve the ethical dilemmas brought about by such giant scientific leaps.

Even though it may sound like science fiction, the future is here.

Gene editing for therapeutic purposes (namely gene therapy) is being conducted for the removal of diseases in humans and foetuses both. When changes are made to the somatic cells, the modifications arent hereditary. However, when the changes are made to the sperm, egg, or embryo, the modifications are transferred on to the next generation. It is done through a process called germline gene editing.

It seems like German philosopher Neitzsches post-human concept of bermensch (translated as beyond-man or superman) might become real. Using a technology known as CRISPR, one can not only treat diseases but also customize a baby in terms of intelligence, athleticism, and so on. This can permanently enhance the lineage of the family.

Believe it or not, the future is here.

Somatic gene editing has been approved in countries like the United States, although with restrictions. It is for the Courts and lawmakers to decide whether this right to enhance children for their own welfare falls within the right to privacy of the parents, or whether the state has a right to curtail such an action.

According to American jurisprudence, there is a private realm of family life which the state cannot enter. Yet time and again the state has entered this realm, claiming a legitimate interest in regulating the family, especially for the welfare of the child. Thus, whilst there exists a right to privacy under the Fourteenth Amendment, it is not absolute.

In the famous case of Roe v. Wade, the United States Supreme Court held that the right to abortion is embedded in the right to privacy of the parent bearing the child. While the judgment was pro-choice, gene editing doesnt quite fit the bifurcated and antagonistic pro-choice v. pro-life debate. It gives parents the choice to genetically edit their children, and the aim of such editing inter alia is to eradicate disease and enhance children for their own welfare.

(Credit: Edward Jenner, Source: Pexels)

Germany, as opposed to the US, recognises the unborn as an individual with a genetically determined identity, which is unique and inseparable. According to German courts, as the unborn baby grows, it does not only develop into a human being but develops as a human being and is worthy of human dignity. Such human dignity would also include the right to live a dignified life, which is free of disease. Therefore, a blanket ban on gene editing would be violative of human dignity.

It is quite clear that therapeutic gene editing is not violative of human dignity, insofar its aim is to eradicate disease. However, such human dignity also includes the right to free development of personality.

It is quite clear that therapeutic gene editing is not violative of human dignity, insofar its aim is to eradicate disease. However, such human dignity also includes the right to free development of personality.

A plain reading of the German constitutional text would suggest that non-therapeutic editing for personality factors is violative of human dignity. But to the contrary, heritable gene editing for purposes such as personality building will be permissible for strengthening the autonomy of the child. Furthermore, it must be for the welfare of the child and must not restrict the free development of their personality. A thumb rule could be whether the child would subsequently consent to such a modification.

Thus, there is a tripartite relationship of the right of the parents to choose the genetic makeup of their children, the right of the state to regulate such an act, and the right of the unborn baby to consent to such editing.

In India, guidelines permitting development of therapeutic gene editing products were introduced. Currently, heritable gene editing can only be done for purposes of experimentation and the embryo cannot have a life beyond 14 days. Thus, heritable gene editing is yet not fully permitted in India. However, there is scope as the Indian Supreme Court in K.S. Puttaswamy (2017) recognised that the right to privacy encompasses family affairs and childrearing. This would possibly entail the parents right to enhance their child as an extension of their right to choose.

It is an established position in law that technology mustnt be prohibited due to a mere possibility of harm. Rather, a positivist approach must be adopted so as to do the greatest good to the greatest number. In fact, a recent report by a German government-appointed council of experts stated that heritable gene editing is not violative of human dignity.

Further, a joint statement by the councils of the United Kingdom, France, and Germany stated that heritable gene editing is permissible. However, there must be a risk assessment and the risk must be brought down to a minimum acceptable level.

And that at the heart of this liberty lies the right to define ones own concept of existence, of meaning, of the universe, and of the mystery of human life, without the interference of the state.

The question, when looked at more deeply, is whether the personality and well-being of future generations ought to be subjected to medical decisions or not.

Thus, there is a tripartite relationship of the right of the parents to choose the genetic makeup of their children, the right of the state to regulate such an act, and the right of the unborn baby to consent to such editing.

The American Supreme Court in Planned Parenthood v. Casey has reiterated that the matters involving the intimate choices of a person are central to dignity and autonomy protected under liberty enshrined in the American Constitution. And that at the heart of this liberty lies the right to define ones own concept of existence, of meaning, of the universe, and of the mystery of human life, without the interference of the state.

(Credit: Ian Panelo, Source: Pexels)

Ultra-modern technologies such as artificial wombs make one think whether a birth is really an event, or a process, and if so then where does the process begin?

Partial ectogenesis (the growth of a baby outside the womb) is already happening. But in time, we might be able to carry out full ectogenesis. As the reliability of such technologies increases, so does their capability. The potential to live outside the mothers womb as opposed to the usual 24 weeks would begin right from the stage of development of the foetus, which is at 8 weeks. Further, with the help of biotech, the ability of an unborn baby to live outside the mothers womb might begin right at fertilisation.

Do we fast forward using techno solutionism or do we let evolution do its thing?

The American Courts have held that an individual whether single or married has the right to privacy which the state cannot infringe. Thus, even a single parent, out of wedlock, can bear such a child and the right to genetically edit unborn babies would be extended to them. The mother and father would be on an equal footing while deciding whether to genetically edit the baby or not.

The aim of the process of childrearing is to produce children with favourable traits and personality and allow them to have a dignified life.

In the first such successfully germline edited babies, scientists predict that it might have actually led to having enhanced their ability to learn and form memories. In another case of successful gene therapy, young children were saved from potentially deadly diseases and lives of isolation.

Therefore, the moot question is: Do we fast forward using techno solutionism or do we let evolution do its thing? Where do we draw the line? Should one circumscribe the limits of technology, or let it take a well-designed course?

Believe it or not, the future is here.

Where does that leave us?

It is for us to decide.

(Raghav Ahooja is a final year student at Rajiv Gandhi National Law University, Punjab. Views are personal.)

Excerpt from:
Gene Editing: Do We Have The Right to Genetically Enhance Our Children? - The Leaflet

New York, Sept. 18, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p05953100/?utm_source=GNW

The Global Advanced Therapy Medicinal Products Market is expected to grow from USD 2,946.38 Million in 2019 to USD 6,524.94 Million by the end of 2025 at a Compound Annual Growth Rate (CAGR) of 14.16%.

Market Segmentation & Coverage:This research report categorizes the Advanced Therapy Medicinal Products to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Therapy Type , the Advanced Therapy Medicinal Products Market studied across CAR-T Therapy, Cell Therapy, Gene Therapy, and Tissue Engineered Product. The Cell Therapy further studied across Non-stem Cell Therapy and Stem Cell Therapy.

Based on Geography, the Advanced Therapy Medicinal Products Market studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas region surveyed across Argentina, Brazil, Canada, Mexico, and United States. The Asia-Pacific region surveyed across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, South Korea, and Thailand. The Europe, Middle East & Africa region surveyed across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Company Usability Profiles:The report deeply explores the recent significant developments by the leading vendors and innovation profiles in the Global Advanced Therapy Medicinal Products Market including AveXis, Inc., Bluebird Bio, Inc., Celgene Corporation, Gilead Lifesciences, Inc., JCR Pharmaceuticals Co., Ltd., Kolon TissueGene, Inc., MEDIPOST, Novartis AG, Organogenesis Inc., PHARMICELL Co., Ltd, Spark Therapeutics, Inc., UniQure N.V., and Vericel Corporation.

FPNV Positioning Matrix:The FPNV Positioning Matrix evaluates and categorizes the vendors in the Advanced Therapy Medicinal Products Market on the basis of Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Competitive Strategic Window:The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies. The Competitive Strategic Window helps the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. During a forecast period, it defines 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.

Cumulative Impact of COVID-19:COVID-19 is an incomparable global public health emergency that has affected almost every industry, so for and, the long-term effects projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlaying COVID-19 issues and potential paths forward. The report is delivering 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 forecast, considering the COVID-19 impact on the market.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyzes the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and new product developments

The report answers questions such as:1. What is the market size and forecast of the Global Advanced Therapy Medicinal Products Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Advanced Therapy Medicinal Products Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Advanced Therapy Medicinal Products Market?4. What is the competitive strategic window for opportunities in the Global Advanced Therapy Medicinal Products Market?5. What are the technology trends and regulatory frameworks in the Global Advanced Therapy Medicinal Products Market?6. What are the modes and strategic moves considered suitable for entering the Global Advanced Therapy Medicinal Products Market?Read the full report: https://www.reportlinker.com/p05953100/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Advanced Therapy Medicinal Products Market Research Report by Therapy Type - Global Forecast to 2025 - Cumulative Impact of COVID-19 - GlobeNewswire

The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments. The sector can certainly point to the significant progress that has taken place during the past few years, with new medicines reaching the market and several novel therapeutics with new mechanisms of action advancing in the pipeline.

Ted Love, president and CEO of Global Blood Therapeutics Inc. (GBT), said 2019 was a landmark year with the FDA approval of two new novel therapies to treat sickle cell disease. He was speaking at the virtual annual Sickle Cell Disease (SCD) Therapeutics Conference this week. His company, together with the Sickle Cell Disease Association of America, was hosting the one-day event featuring discussions on the latest advances and future trends.

Approvals

The key manifestation of the inherited blood disorder is that red blood cells (RBCs) are abnormally shaped (crescent), which restricts their flow in blood vessels and limits oxygen delivery to the bodys tissues, leading to severe pain and organ damage. The condition is also characterized by severe chronic inflammation that results in vaso-occlusive crisis (VOC) where patients experience episodes of extreme pain and organ damage.

Late November, GBT gained accelerated approval for its Oxbryta (voxelotor) tablets for the treatment SCD in adults and pediatric patients 12 and older. The agencys green light came less than two weeks after it gave the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of VOCs in adult and pediatric patients ages 16 and older with SCD.

According to Love, Oxbryta is a new class of therapy binding to hemoglobin and stabilizing RBCs in an oxygenated state and inhibiting deoxygenated sickle hemoglobin polymerization, making cells less likely to bind together and form the distinctive sickle shape.

The launch of the drug has gone well since it was approved, he said in the companys second-quarter financial report and business update, despite the impact of COVID-19. Net sales in the period reached $31.5 million, well ahead of the Streets expectations. Going forward, the company is planning to expand the potential use of Oxbryta for the treatment of SCD in children as young as 4 years old and also seek marketing authorization in Europe for Oxbryta to treat hemolytic anemia in SCD patients ages 12 and older with a marketing authorization application being submitted to the EMA by the middle of next year.

Pipeline progress

Cambridge, Mass.-based Agios Pharmaceuticals Inc. is working on mitapivat (AG-348), an investigational, oral, small-molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes, in patients with SCD. The compound has been shown to decrease 2,3-diphosphoglycerate (2,3-DPG) and increase adenosine triphosphate (ATP), and through that mechanism, it may reduce hemoglobin (Hb) S polymerization and red blood cell sickling. In June, the company reported that clinical proof of concept had been established based on a preliminary analysis in a phase I trial being conducted in collaboration with the U.S. NIH as part of a cooperative research and development agreement.

The ongoing study had enrolled nine patients, with eight completing all planned dose levels of mitapivat. Seven of eight patients who completed all dose levels experienced a Hb increase, with five of eight patients (63%) achieving a hemoglobin increase of 1 g/dL from baseline (range 1-2.7 g/dL). All five patients who achieved a hemoglobin increase of 1 g/dL did so at doses of 50 mg BID or lower. Decreases in 2,3-DPG and increases in ATP levels were observed, consistent with the proposed mechanism of action and comparable to that observed in healthy volunteer studies with mitapivat.

The company said it expects to report data from ACTIVATE and ACTIVATE-T, its two global pivotal trials for mitapivat in adults with pyruvate kinase deficiency, between the end of 2020 and mid-2021.

Watertown, Mass.-based Forma Therapeutics Holdings Inc., which made its public debut this year, also has a selective RBC pyruvate kinase-R activator in its pipeline for treating SCD. FT-4202 is being evaluated in a phase I trial in SCD patients ages 12 and older and has been granted fast track, rare pediatric and orphan drug designations. The compound is a potent activator of pyruvate kinase-R designed to improve RBC metabolism, function and survival by decreasing 2,3 DPG and increasing ATP, potentially resulting in both increased hemoglobin levels and reduced VOCs.

Olinciguat, an oral guanylate cyclase (sGC) stimulator, being developed by Cyclerion Therapeutics Inc., has completed the treatment period in its STRONG-SCD study with a total of 70 patients randomized. The placebo-controlled, dose-ranging study is designed to evaluate safety, tolerability and pharmacokinetics, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period. Top-line results are expected this year. Olinciguat is a compound that aims to stimulate sGC production, leading to the production of a signaling molecule called cyclic guanosine monophosphate (cGMP). High levels of cGMP help reduce inflammation in blood vessels, decrease adhesion between RBCs, and allow for improved blood flow by increasing the availability of nitric oxide.

Boston-based Imara Inc. is developing IMR-687, a small-molecule inhibitor of PDE9 that degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology. The company said that lower levels of cGMP are often found in people with SCD and beta-thalassemia and are associated with impaired blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide-mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which are associated with reactivation of fetal hemoglobin.

In August, the company dosed the first patient in its Ardent phase IIb trial of IMR-687 for adult patients with SCD. The planned primary efficacy objective is to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an increase of 3% in HbF from baseline to week 24, compared to placebo.

Gene therapy/gene editing

Since SCD is a monogenic disease condition, researchers believe that it would be a good candidate for gene therapy therapeutic approaches. For example, significant progress is being made by Cambridge, Mass.-based Bluebird Bio Inc. with lentiglobin, its lentiviral-based gene therapy that inserts an anti-sickling beta-globin variant into CD34-positive cells, progenitors of red blood cells.

At the virtual European Hematology Association (EHA) meeting in June, it reported new data from its ongoing phase I/II study involving adult and adolescent patients with SCD that showed a near-complete reduction of serious VOCs and acute chest syndrome. The company expects to submit a BLA to the FDA for the gene therapy next year.

Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. are progressing CTX-001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. At EHA, the companies reported that in the phase I/II Climb-121 study, at nine months after CTX-001 infusion, the first treated patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%.

Last month, Cambridge, Mass.-based Editas Medicine Inc., a genome editing company, reported that the FDA had granted rare pediatric disease designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for SCD. The company plans to file an investigational new drug application for EDIT-301 by the end of this year. EDIT-301 comprises sickle patient CD34+ cells genetically modified using a hCRISPR/Cas12a (also known as Cpf1) ribonucleoprotein to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production.

In its second-quarter financial report and business update, Beam Therapeutics Inc. announced the nomination of its first two adenine base editing development candidates, BEAM-101, targeting patients with hereditary persistence of fetal hemoglobin, and BEAM-102 (Makassar variant), both aimed at correcting SCD.

New Initiative

The NIH, which reports it spends approximately $100 million on sickle cell disease research, announced that is has launched The Cure Sickle Cell Initiative designed to speed the development of cures for the disease. It will take advantage of the latest genetic discoveries and technological advances to progress the most promising genetic-based curative therapies safely into clinical trials within five to 10 years.

Aided by research partners, the initiative will establish a national data warehouse of genetic therapies for sickle cell disease and conduct comparative analyses of therapeutic approaches to assess both clinical and cost effectiveness. National networks will also be created to make it easier for patients and providers to interact with the research, clinical trials, and other activities.

Read this article:
New medicines in the pipeline to treat sickle cell disease - BioWorld Online

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

Follow this link:
Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

NEW YORK--(BUSINESS WIRE)--As part of a two-day virtual Investor Day, Pfizer Inc. (NYSE: PFE) provided an extensive overview of pipeline advances and shared updates on the Companys efforts to battle the COVID-19 pandemic on multiple fronts, including new data on the BNT162b2 vaccine candidate being developed in collaboration with BioNTech SE. The pipeline updates contribute to the Companys expectation of at least a 6% revenue CAGR over the next five years, as well as delivery of longer-term topline growth beyond that period.

Pfizers goal of delivering up to 25 breakthroughs to patients by the year 2025 has 38 such opportunities to draw from as of today, including the companys 20-valent pneumococcal conjugate vaccine candidate (20vPnC). On a non-risk adjusted basis, these opportunities collectively represent more than $15 billion (excluding 20vPnC) in potential incremental revenue for Pfizer from 2020 to 2025, as well as aggregate peak annual sales potential of $35 billion to $40 billion (including 20vPnC). If successful, the Companys COVID-19 programs would be incremental to these estimates.

Pfizers purpose Breakthroughs that change patients lives has never been more relevant, and our R&D pipeline has never been more dynamic, said Dr. Albert Bourla, Pfizer Chairman and CEO. I am proud of the truly transformational science that our research and clinical teams are bringing to the fight against disease, as well as the unprecedented speed with which we are advancing our clinical programs in the battle against COVID-19. In the coming months and years, I look forward to the new Pfizer continuing to demonstrate the agility and innovative spirit of a biotech combined with the scale of Big Pharma. With the depth and breadth of our current portfolio, the tremendous potential of our pipeline and scientific engine, and the power of our culture of innovation, we are poised to continue delivering meaningful value to patients by addressing some of the worlds most difficult health challenges.

UPDATES ON COVID-19 DEVELOPMENT PROGRAMS

Pfizer announced several key advances in its efforts to protect humankind from the COVID-19 pandemic and prepare the pharmaceutical industry to better respond to future global health crises.

BNT162 mRNA-based Vaccine Program

Pfizer and BioNTech shared several updates from their BNT162 mRNA-based vaccine program against SARS-CoV-2, the virus that causes COVID-19 disease, including:

Protease Inhibitor Program

The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814. Of note,

THERAPEUTIC AREAS OF FOCUS

Pfizer shared significant research advances across its various therapeutic areas including candidates with blockbuster potential expected to launch by 2025.

Vaccines

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Updates on these late-stage clinical development programs include:

Rare Disease

Pfizers Rare Disease late-stage pipeline currently includes three gene therapy programs that, if successful, are expected to gain regulatory approval by the end of 2023, with an additional pipeline of 10 preclinical initiatives that are at various stages of maturity. Key updates include:

Oncology

Pfizers Oncology pipeline has the potential to deliver up to 14 approvals expected by the end of 2025 and the potential for 24 new molecular entities in the clinic by the end of 2021. Key updates included, for the first time, early-stage opportunities obtained from the 2019 acquisition of Array BioPharma:

Inflammation and Immunology

The Inflammation & Immunology pipeline is focused on patients with autoimmune and chronic inflammatory diseases across rheumatology, gastroenterology and dermatology, with five distinct immuno-kinases, in oral and topical formulations, studied for potential treatment of 10 diseases, and three additional novel biologics in Phase 2 studies. Key updates included:

Internal Medicine

The Internal Medicine pipeline addresses the increasing global burden of cardiometabolic disease, with nine investigational medicines in active clinical studies and additional therapies in the pre-clinical pipeline. Key updates included:

To access a replay of the webcast, including audio, video and presentation slides, visit our web site at http://www.pfizer.com/investors.

About Pfizer: Breakthroughs That Change Patients Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice: The information contained in this release is as of September 15, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release or the webcast as the result of new information or future events or developments.

This release and the webcast contain forward-looking information about Pfizers anticipated operating and financial performance, business plans and prospects, Pfizers pipeline portfolio (including anticipated regulatory submissions, data read-outs, study starts, approvals, revenue contributions and market opportunities), and our efforts to respond to COVID-19, including our investigational vaccine candidate against SARS-CoV-2 and our investigational protease inhibitor, including their potential benefits, among other things, that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizers product candidates; whether and when any such applications that may be filed for any of Pfizers product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizers product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; uncertainties regarding the ability to obtain recommendations from vaccine technical committees and other public health authorities and uncertainties regarding the commercial impact of any such recommendations; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

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Pfizer Investor Day Features Significant Number of Pipeline Advances for COVID-19 Programs and Across Numerous Therapeutic Areas - Business Wire

Here are the top biotech companies you will find in Oxford, a city with an old scientific tradition and an enormous output of biotech applications.

Oxford is well known for its university, one of the oldest in Europe and considered to be one of the best in the world. Recently, the University of Oxford has been the center of attention thanks to an experimental Covid-19 vaccine that the university is developing in partnership with big pharmaceutical companies. If successful, the vaccine, already in phase III testing, could be one of the first to get approval for this new disease.

The University of Oxford has hosted thousands of bright minds over the years. Thanks to its emphasis on technology transfer, the university has also helped a large number of them turn their ideas into successful spinout companies. The environment created around the university has also attracted many talents and businesses to the city, making it the ideal melting pot for new and promising ideas.

Biotech is one of the fortes of the innovation seen in the city of Oxford. So we consulted with local experts to put together a list of the most remarkable companies in the city, be it for their size, innovation, or influence in the sector.

Founded in 2008, Immunocore is one of just a few private biotech companies in Europe that are estimated to be worth over 1B. The company is tackling multiple forms of cancer as well as infectious and autoimmune diseases using T-cell receptor (TCR) technology. TCRs are proteins on the surface of immune T cells that are responsible for identifying a threat that must be destroyed, such as cancerous or infected cells. Immunocore aims to patients with engineered TCRs to circumvent the mechanisms by which these threats evade the immune system, restoring its ability to fight disease.

The company is collaborating on several projects with Genentech, AstraZeneca, Eli Lilly, and GSK. Its most advanced program is a treatment for uveal melanoma that is currently in phase III trials. Other programs target solid tumors, hepatitis, HIV, and type 1 diabetes.

Adaptimmune Therapeutics was founded at the same time as Immunocore with the goal of exploiting TCR technology in the form of T-cell therapy. The company engineers the TCRs naturally present on the patients own immune T cells to improve their ability to identify cancerous cells.

Adaptimmune is now getting ready to start late-stage clinical trials in multiple cancer types. Thanks to a deal with Astellas Pharma, the company is also gearing up to start clinical testing of a version of its T-cell therapy that doesnt require engineering each dose individually for each patient, using donor cells instead. Adaptimmune also has several partnerships with companies including GSK, Noile-Immune Biotech, and Alpine Immune Sciences.

Oxford Biodynamics was spun out of Oxford University in 2007 with the goal of developing liquid biopsy tests that can perform a diagnosis from just a drop of blood. The company specializes in epigenetics, that is changes to the structure of our DNA that determines which genes are switched on or off.

Oxford Biodynamics works in a wide range of indications, including cancer, diabetes, Alzheimers, multiple sclerosis, and rheumatoid arthritis among many others. The tests are not only designed to diagnose a disease; they can also be made to determine which patients are going to benefit the most from a specific drug, and how likely the disease is to progress faster or relapse.

With these tests, the company is supporting the development of personalized medicine approaches and helping drug developers increase their chances of succeeding in clinical trials. Partners include big pharma such as Pfizer and EMD, as well as universities and research institutes.

Chronos Therapeutics started out in 2009 as a spinout of the University of Oxford with the goal of developing drugs for age-related conditions. The companys lead program targets amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, for which there are no treatments able to stop its progression.

Over time, the company has expanded its portfolio into other indications, particularly those that affect the brain, by acquiring assets from third parties. They include programs targeting fatigue caused by multiple sclerosis, addictive behaviors such as binge eating and alcohol use disorder, and post-traumatic stress disorder.

Evox Therapeutics is developing a drug delivery technology based on exosomes nanoparticles that our bodies naturally use to transport molecules. The company engineers exosomes to carry different types of drugs, such as proteins, RNA, or small drugs. The key advantage is that these natural carriers are able to reach targets that conventional drug delivery systems cant, such as the brain.

Founded in 2016, the company has signed big deals with Takeda and Eli Lilly. Its programs are all in preclinical testing and mostly target rare diseases. One of them targets the rare liver disorder argininosuccinic aciduria and is scheduled to enter clinical trials in 2021.

Exscientia is a pioneer in the application of artificial intelligence to drug discovery. The company uses AI to identify potential drug candidates and optimize their structure to maximize their chances of success in clinical trials. The goal is to speed up and reduce the costs of the drug discovery process.

Earlier this year, the companys drug candidate for obsessive-compulsive disorder became one of the first AI-designed drugs to enter clinical trials. While a drug typically takes five years from identification until clinical trials, this one did so in just a year.

Exscientia is partnered with Bayer, BMS, Sanofi, and GSk among others. In the wake of the Covid-19 pandemic, the company set out to go through a database of 15,000 approved and investigational drugs that had already passed safety testing to find candidates that can then be fast-tracked to clinical testing in Covid-19 patients.

Oxford Biomedica was set up in 1995 as a developer and provider of lentiviral vectors for gene and cell therapy. These vectors allow the permanent introduction of a desired DNA sequence into a target cell, be it in the test tube or directly in the patients body.

The technology of Oxford Biomedica is regularly used by companies such as Novartis, Sanofi, Boehringer Ingelheim, Imperial Innovations, and Orchard Therapeutics. Notably, the vectors developed by the company are used in Novartis Kymriah, the first CAR T-cell therapy approved in Europe and the US as a cancer treatment.

Oxford Biomedica also has a preclinical pipeline of proprietary programs in a wide range of applications, including cancer, eye disease, ALS, and liver disease. Last year, the company struck a deal with Microsoft to reduce the complexity and costs of gene and cell therapy manufacturing using artificial intelligence.

Founded in 2016, Arctoris aims to bring the benefits of automation to cancer research. Through the companys services, a researcher could just order an experiment online and spend their valuable time designing experiments and analyzing results rather than performing the repetitive tasks needed to complete them.

Arctoris aims to contribute to reducing the costs of drug discovery, which are increasing every year as treatments become personalized and results more difficult to replicate. In the context of the Covid-19 pandemic, Arctoris has established assays that allow scientists the possibility of running Covid-19 experiments remotely.

Founded in 2015, Orbit Discovery is a drug discovery company focusing on the identification of peptide drugs. The company has developed a technology that significantly improves on conventional methods of drug screening such as phage display or mRNA display.

The technology consists of fusing peptides to the DNA sequence encoding them and presenting them to live cells. This method allows the screening of peptide targets that were previously missed by other technologies, and to study their effect on live cells to better predict their function.

The company has already identified several candidates in the areas of cardiology, immunology, and cancer, and is working with partners such as Zealand Pharma in their preclinical development.

OxStem is a drug discovery company with an unusual approach to stem cell treatments. Instead of using stem cells as a therapy, the firm focuses on developing small molecule drugs that can reprogram the patients own stem cells to treat a wide range of diseases related to aging, including cancer and diabetes as well as neurological, cardiovascular, and ocular conditions.

Founded in 2013, the strategy of OxStem is to spin out companies that specialize in each disease area to focus on the development of the drugs found by the parent company, with five subsidiaries set up so far.

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The Top 10 Biotech Companies Brewing at... - Labiotech.eu

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the currently available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

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Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene-editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts as a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up our genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for the immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the number of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibodies against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually, the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology, provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

This article is republished from The Conversation by Samira Kiani, Associate Professor of Pathology, University of Pittsburghunder a Creative Commons license. Read the original article.

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How CRISPR is tackling the troubling immune response thats plagued gene therapy until now - TNW