Category Archives: Stem Cell Therapy

HOUSTON--(BUSINESS WIRE)--InGeneron, Inc., a clinical stage biotechnology company, announced the publication of a succinct scientific review of regenerative cell therapy, commonly called stem cell therapy, to treat orthopedic indications. This newly released paper, titled Why and how to use the bodys own stem cells for regeneration in musculoskeletal disorders: a primer, was published in the Journal of Orthopaedic Surgery and Research (J Orthop Surg Res 17, 36 (2022): https://doi.org/10.1186/s13018-022-02918-8). The publication provides an approachable overview of stem cell biology and clarifies common misconceptions about adipose-derived regenerative cells (ADRCs) including vascular-associated pluripotent stem cells (vaPS cells). The authors emphasize the ability of therapies using ADRCs to readily fit into modern orthopedic treatment concepts and reference InGenerons proprietary cell therapy platform, currently under evaluation in ongoing FDA-approved trials.

Summarizing 20 years of both basic and clinical research, the review aims to provide a straightforward look at the current state of orthopedic regenerative cell therapies and clarifies the role of different regenerative cells, such as vaPS cells, in tissue regeneration. The publication highlights the advantages of InGenerons therapeutic approach utilizing ADRCs to develop point-of-care therapies compared to other types of stem cell therapy, including techniques requiring cells to be cultured in a lab. Dr. Eckhard Alt, Director of Stem Cell Research at Tulane University (New Orleans, LA, USA), Executive Chairman of InGeneron and co-author of the paper explains: Using unmodified, uncultured, autologous cells allows for true point-of-care treatment, which can be performed within a short time on the same day in an outpatient facility. Culturing and modifying cells before treatment increases the complexity and cost for patients and physicians and also increases the possibility for contamination of the cells and other health concerns, such as autoimmune rejection, that are not an issue when using ADRCs.

Pointing out another advantage of InGenerons therapeutic approach, Dr. Christoph Schmitz, Head of the Department of Anatomy II at Ludwig-Maximilians University of Munich (Munich, Germany), Advisory Medical Director of InGeneron and co-author of the paper adds, We realized early on that stem cells were important but that they benefitted from other cells contained in ADRCs such as progenitor cells, pericytes, endothelial cells and fibroblasts, which we collect from patients adipose tissue along with their stem cells. All of these cell types play an essential role in tissue regeneration and work synergistically, each affecting the other to promote healing in specific ways that we are still working to fully understand. Therapies that isolate stem cells for culturing in the lab lack these other cell types.

The publication concludes that utilizing ADRCs offers the most attractive therapeutic approach for providing safe and effective treatments, which can be integrated into the modern orthopedic clinical paradigm.

Building on the insights obtained from years of research studying regenerative cells, InGeneron is currently conducting three actively enrolling FDA-approved clinical trials to evaluate its cell therapy platform for the treatment of musculoskeletal indications such as partial-thickness rotator cuff tear, wrist osteoarthritis, and facet joint syndrome.

Publication Details

DOI: https://doi.org/10.1186/s13018-022-02918-8

Citation: Furia, J.P., Lundeen, M.A., Hurd, J.L. et al. Why and how to use the body's own stem cells for regeneration in musculoskeletal disorders: a primer. J Orthop Surg Res 17, 36 (2022).

About the Transpose RT System and Current Clinical Trials

InGenerons Transpose RT cell therapy platform consists of a processing unit, a set of disposables, and Matrase, a proprietary enzyme mixture. The platform allows the isolation of regenerative cells from the patients' own adipose tissue at point-of-care in less than 90 minutes for same-day treatment. The cells are re-administered into the patients damaged tissue by injection under ultrasound or fluoroscopic guidance.

The Transpose RT System is being investigated in several FDA-approved clinical trials and is currently available in the U.S. for research use only. More information on InGenerons actively enrolling clinical trials can be found at http://www.clinicaltrials.gov under the identifiers NCT03752827, NCT03513731, and NCT03503305.

About InGeneron

InGeneron is a clinical-stage biotechnology company developing novel, safe, and evidence-based cell therapies. We are setting new therapeutic standards by enabling minimally invasive treatments that unlock the healing potential of each patients own regenerative cells processed at the point of care for same-day treatment. We currently focus on helping patients impacted by orthopedic conditions and are conducting several clinical trials to validate our technology as a disease-modifying treatment. Based on more than 20 years of research, InGeneron is dedicated to developing therapies supported by clinical evidence and approved by the FDA.

http://www.ingeneron.com

Read more:
InGeneron Publishes Overview on Current State and Potential of Regenerative Cell Therapy in Orthopedics - Business Wire

Global Cell Therapy Manufacturing Market, By Therapy (T-Cell Therapies, Dendritic Cell Therapies, Tumor Cell Therapies, Stem Cell Therapies), By Source of Cell (Autologous v/s Allogenic), By Scale of Operation (Preclinical, Clinical, Commercial), By Source (In-House v/s Contract Manufacturing), By Application (Oncology, Cardiovascular Diseases, Orthopedic Diseases, Others), By End User (Pharmaceutical & Biotechnology Companies, Academic & Research Institutes, Others), By Region, Competition Forecast and Opportunities, 2026

New York, Jan. 26, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Cell Therapy Manufacturing Market, By Therapy, By Source of Cell, By Scale of Operation, By Source, By Application, By End User, By Region, Competition Forecast and Opportunities, 2026" - https://www.reportlinker.com/p06131077/?utm_source=GNW

Global cell therapy manufacturing market was valued at USD3123.44 million in the year 2020 and is anticipated to grow with a CAGR value of 12.0% in the forecast period, 2022-2026 to reach market value of USD6015.09 million by 2026F. The market growth can be attributed to advancing biopharmaceutical industries across the globe. Moreover, increasing dependency on cell therapy and thus therapeutics & pharmaceutical products for the efficient treatment of the patients suffering from critical diseases and conditions is also supporting the growth of the global cell therapy manufacturing market in the next five years. Additionally, growing prevalence of chronic diseases like cardiovascular diseases, cancer disease, along with lifestyle diseases like obesity, diabetes that further deteriorates immunity of the humans further contributes to the growth of the global cell therapy manufacturing market in the future five years. Recent instance of COVID-19 outbreak that caused millions of deaths worldwide has created a demand for advanced cell therapy manufacturing to devise pharmaceuticals, vaccines, etc. Significant development and advancement in cell-based therapies for skin rejuvenation and tendon regeneration are further driving the growth of the global cell therapy manufacturing market in the forecast period.The global cell therapy manufacturing market is segmented by therapy, source of cell, scale of operation, source, application, end user, company, and regional distribution.Based on application, the market is further divided into oncology, cardiovascular diseases, orthopedic diseases, and others.

Oncology sub-segment is anticipated to hold more than 50% of the revenue shares of the market and dominate the application based market segmentation in the upcoming five years.The growth can be attributed to increasing instances of various types of cancer in the global population.

Moreover, increased technological advancement and consistent cell therapy based research for the treatment of cancer is further anticipated to support the market growth.Novartis AG, F. Hoffmann-La Roche AG, Gilead Sciences, Inc., Thermo Fischer Scientific, Inc., Catalent, Inc., JSR Life Sciences LLC (KBI Biopharma Inc), Waisman Center (Waisman Biomanufacturing), Cell and Gene Therapy Catapult, Merck KGaA, Lonza Group, Oxford Biomedica Plc, WuXi AppTec, Charles River Laboratoires International Inc., Institut Merieux (ABL Inc.), BioCentriq, Centre for Commercialization of Regenerative Medicine (CCRM), Fujifilm Holdings Corporation (Fujifilm Cellular Dynamics), Amgen Inc., Bluebird Bio Inc., Takeda Pharmaceutical Company Limited are among the major market players in the global platform that lead the market growth of the global cell therapy manufacturing market.

Years considered for this report:

Historical Years: 2016-2019Base Year: 2020Estimated Year: 2021Forecast Period: 20222026

Objective of the Study:

To analyze and estimate the market size of global cell therapy manufacturing market from 2016 to 2020. To estimate and forecast the market size of global cell therapy manufacturing market from 2021 to 2026 and growth rate until 2026. To classify and forecast global cell therapy manufacturing market based on therapy, source of cell, scale of operation, source, application, end user, company, and regional distribution. To identify dominant region or segment in the global cell therapy manufacturing market. To identify drivers and challenges for global cell therapy manufacturing market. To examine competitive developments such as expansions, mergers & acquisitions, etc., in global cell therapy manufacturing market. To identify and analyze the profile of leading players operating in global cell therapy manufacturing market. To identify key sustainable strategies adopted by market players in global cell therapy manufacturing market.The analyst performed both primary as well as exhaustive secondary research for this study.Initially, the analyst sourced a list of companies the globe.

Subsequently, the analyst conducted primary research surveys with the identified companies.While interviewing, the respondents were also enquired about their competitors.

Through this technique, the analyst could include the companies which could not be identified due to the limitations of secondary research. The analyst examined the companies, distribution channels and presence of all major players across the globe.The analyst calculated the market size of global cell therapy manufacturing market using a bottom-up approach, wherein data for various end-user segments was recorded and forecast for the future years. The analyst sourced these values from the industry experts and company representatives and externally validated through analyzing historical data of these applications for getting an appropriate, overall market size.

Various secondary sources such as company websites, news articles, press releases, company annual reports, investor presentations and financial reports were also studied by the analyst.

Key Target Audience:

Companies, and other stakeholders Government bodies such as regulating authorities and policy makers Organizations, forums and alliances related to cell therapy manufacturing Market research and consulting firmsThe study is useful in providing answers to several critical questions that are important for the industry stakeholders such as manufacturers, suppliers, partners, end users, etc., besides allowing them in strategizing investments and capitalizing on market opportunities.

Report Scope:

In this report, global cell therapy manufacturing market has been segmented into following categories, in addition to the industry trends which have also been detailed below: Global Cell Therapy Manufacturing Market, By Therapy:o T-Cell Therapieso Dendritic Cell Therapieso Tumor Cell Therapieso Stem Cell Therapies Global Cell Therapy Manufacturing Market, By Source of Cell:o Autologouso Allogenic Global Cell Therapy Manufacturing Market, By Scale of Operation:o Preclinicalo Clinicalo Commercial Global Cell Therapy Manufacturing Market, By Source:o In-Houseo Contract Manufacturing Global Cell Therapy Manufacturing Market, By Application:o Oncologyo Cardiovascular Diseaseso Orthopedic Diseaseso Others Global Cell Therapy Manufacturing Market, By End User:o Pharmaceutical & Biotechnology Companieso Academic & Research Instituteso Others Global Cell Therapy Manufacturing Market, By Region:o North AmericaUnited StatesCanadaMexicoo EuropeGermanyFranceUnited KingdomSpainItalyo Asia-PacificChinaJapanSouth KoreaAustraliaIndiao South AmericaBrazilArgentinaColombiao Middle East & AfricaIsraelSouth AfricaSaudi ArabiaUAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in global cell therapy manufacturing market.

Available Customizations:

With the given market data, we offers customizations according to a companys specific needs. The following customization options are available for the report:

Company Information

Detailed analysis and profiling of additional market players (up to five).Read the full report: https://www.reportlinker.com/p06131077/?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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Global Cell Therapy Manufacturing Market, By Therapy, By Source of Cell, By Scale of Operation, By Source, By Application, By End User, By Region,...

Avra, Inc.

Ann Shippy, MD

Dr. Charles A. Pereyra

ATLANTA, Jan. 27, 2022 (GLOBE NEWSWIRE) -- via NewMediaWire -- Avra, Inc. (OTC PINK: AVRN), operating as Springs Rejuvenation, (Spring), a Chamblee, Georgia based anti-aging and stem cell center focusing on stem cell therapy, today announced a partnership with Austin TX clinic to expand its operations.

Springs Rejuvenation is excited to announce its new partnership with Ann Shippy MD. A former IBM engineer, Ann Shippy, MD transitioned to the world of medicine in part, in search of better solutions to her own health ailments, which she hadnt found in traditional medicine. Her practice, which is based in Austin, Texas, takes a functional approach to a wide range of health concerns where she has spent more than a decade successfully treating conditions ranging from near-fatal toxicity, neurological disorders, to autoimmunity, infertility and beyond.

Dr. Pereyra, founder of Springs Rejuvenation, stated, She is one of the most intelligent and compassionate humans Ive ever known. When we first met I knew we had a special connection and were both excited about exploring the unknown and pushing to new horizons. I couldnt have made a better friend or partner. We will begin offering treatments immediately.

Dr. Shippy is board-certified in internal medicine and certified in functional medicine, authoring two health manuals, appearing as a guest speaker on numerous platforms, and has founded two health-based foundations. Gaining notoriety for her unique blend of measured, precise data with a heartfelt and sympathetic attitude towards her patients. Whether at a live event, webinar, mastermind presentation or private engagement, Dr. Shippy has always been excited to share with her community the most cutting edge guidance in epigenetic, detoxification, Functional Medicine, and optimal healing.

About Springs Rejuvenation Inc.

At Springs Rejuvenation we strive to be at the cutting edge of regenerative medicine and anti-aging research. Our mission is to provide patients with individualized, state-of-the-art treatment, returning each patient to their natural mobility as quickly as possible.

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With the goal of addressing the root cause of your pain, rather than just masking it. We want to help you achieve whole body health and rejuvenation. This which will help you feel stronger, improve mood, look younger, heal faster and perform at the optimal level. Our patients have found both freedom from pain and improved quality of life.

Our professional staff is uniquely composed of physicians/researchers in the Stem Cell, Anti-Aging research and regenerative medicine, also including sports-related injuries and chronic joint pain.

Our Medical Director & Founder Dr. Charles A. Pereyra is the founder of Springs Rejuvenation Stem Cell Therapy in Atlanta, GA (since 2018). He is our current lead clinical Physician and an expert in Stem Cell, Anti-Aging research and Regenerative medicine. Dr. Juan Pablo Nieto specializes in sports-related injuries as well as chronic joint pain. He has expertise experience with diagnostic ultrasound exams as well as joint and tendon injections with PRP, & prolotherapy. His highlights include caring/treating professional athletes: NBA Wizards & D1 level athletes from different multiple sports and presented research at national conferences.

FORWARD-LOOKING STATEMENTS:

This press release contains forward-looking statements as defined within Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements relate to future events, including our ability to raise capital, or to our future financial performance, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. You should not place undue reliance on forward-looking statements since they involve known and unknown risks, uncertainties and other factors which are, in some cases, beyond our control and which could, and likely will, materially affect actual results, levels of activity, performance or achievements. Any forward-looking statement reflects our current views with respect to future events and is subject to these and other risks, uncertainties and assumptions relating to our operations, results of operations, growth strategy and liquidity. We assume no obligation to publicly update or revise these forward-looking statements for any reason, or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

For a discussion of these risks and uncertainties, please see our filings with the OTC Markets Group Inc. Our public filings with the OTC Markets Group Inc are available from commercial document retrieval services and at the website maintained by the OTC Markets at https://www.otcmarkets.com/stock/AVRN/disclosure

Company website(s): http://www.avrabiz.com ; https://springsrejuvenation.com

Company Twitter: @SpringsRejuven1

Email: avrabiz21@gmail.com

Phone: 678-387-3515

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Springs Rejuvenation Partners with Ann Shippy, MD to Offer Treatments in Austin, TX - Yahoo Finance

Adult T-cell leukemia/lymphoma (ATL) is a refractory peripheral T-cell lymphoma (PTCL). Treatment with chemotherapy alone is associated with an extremely poor prognosis. Allogeneic stem cell transplantation (SCT) is the only modality shown to achieve long-term survival, but treatment-related mortality is a critical problem in the relatively elderly individuals most likely to develop with ATL [1]. Autologous stem cell transplantation (ASCT) is rarely performed in ATL, and the few studies of ASCT in ATL reported dismal outcomes [2,3,4].

To assess the efficacy of ASCT as a means of reducing tumor burden in the era of autologous immune activation therapies, such as immune checkpoint inhibitors [5], this study was performed to characterize the status of autografting in ATL using national registry data in Japan, one of the most endemic areas for ATL.

The clinical data of hematopoietic SCT recipients were collected by the Japan Society for Hematopoietic Cell Transplantation (JSHCT) and the Japanese Data Center for Hematopoietic Cell Transplantation using the Transplant Registry Unified Management Program (TRUMP) [6]. Data were maintained by the JSHCT (TRUMP database, last update, December 2018).

A total of 2216 patients with ATL who underwent SCT between 1991 and 2017 were registered in the TRUMP database. Twenty-seven patients underwent ASCT, one received syngeneic SCT, and 2188 were treated with allogeneic SCT. The 27 ASCT patients were retrospectively analyzed. Demographic characteristics, ATL subtypes, stem cell sources, preconditioning regimens, disease status at ASCT, ECOG performance status at ASCT, date at diagnosis, date at ASCT, date at neutrophil engraftment, date at last observation, and causes of death were collected. Overall survival (OS) was calculated from the date of ASCT and diagnosis until the date of death or final follow-up.

This study was approved by the data management committees of JSHCT and the institutional ethical committee of Imamura General Hospital.

OS was analyzed using the KaplanMeier method. In four patients who underwent SCT twice, the date of the second transplant was not censored. Univariate comparisons of OS were made using the log rank test.Pvalues<0.05 were considered statistically significant. Statistical analyses were performed using EZR software [7].

Patient characteristics are shown in Supplementary Table1. Patients consisted of 14 males and 13 females, with a median age at ASCT of 52 years (range, 3166 years). Twenty-four of 27 patients (88.9%) received ASCT in or before 2009. ASCT was performed in 11 patients (40.7%) with complete remission (CR), three (11.1%) with relapse, and 13 (48.1%) with primary induction failure. Eleven patients had aggressive-type ATL, including five with acute type and six with lymphoma type, whereas 16 patients had an unknown subtype. The Eastern Cooperative Oncology Group performance status was 0 in four patients, 1 in seven, and unknown in 16. Peripheral blood stem cells were used as the stem cell source in 24 patients (88.5%), while three patients received autologous bone marrow transplantation. The median duration from diagnosis to ASCT was 242.5 days (range, 641128 days). Four patients received SCT twice; of these, one underwent ASCT twice and the other three underwent ASCT followed by allogeneic SCT (Supplementary Table2).

Data on preconditioning regimens were available for only eight patients. Cyclophosphamide was used in five patients, etoposide in six, ranimustine in five, carboplatin in two, cytosine arabinoside in five, melphalan in three, ifosfamide in one, and dexamethasone in two. Only one patient underwent total body irradiation as preconditioning therapy.

Data on neutrophil engraftment were obtained for 25 patients (92.6%). Two patients died before engraftment. The 1-year OS rate after ASCT was 18.5%, and the median OS time (MST) was 5.6 months (Fig.1a). The 1-year OS rate and the MST were 27.3% and 7.7 months, respectively, in patients with CR at ASCT, and 13.3% and 3.6 months, respectively, in patients without CR (Fig.1b). The 3-year OS rate and the MST after diagnosis were 20.8% and 15.5 months, respectively. The MST was 15.1 and 16.2 months in patients with CR and without CR, respectively (Fig.1c, d). All 27 patients who underwent ASCT died during the observation period. The causes of death were ATL in 15 patients, infections in six, and unknown in six.

Fig. 1: Overall survival from ASCT and from diagnosis.

aOverall survival from ASCT in patients with ATL who underwent ASCT, (b) Overall survival according to CR or nonCR, (c) Overall survival from diagnosis, (d) Overall survival according to remission state. OS overall survival, ASCT autologous stem cell transplantation, CR complete remission, Diag diagnosis.

Full size image

All four patients who underwent SCT twice died, with OS ranging from 3 to 11 months. The causes of death were pneumonia in three patients and ATL in one (Supplementary Table2).

This study is the first descriptive case series to summarize the results of ASCT in patients with ATL. Patients with ATL who underwent ASCT had poor outcomes, with a 1-year OS rate of 18.5% and a MST of 5.6 months. Four patients survived for longer than 30 months after ASCT, and it should be noted that this duration of survival after ASCT has not been reported previously. Although the overall response to treatment remains poor, this study provides more accurate data on long-term outcomes since it used information from a registry that requires registration of almost all patients receiving SCT. The study also revealed that autologous transplants have rarely been performed in Japan since 2010.

Among patients with a known cause of death, 71.4% died of ATL relapse, and the remaining 28.6% died of infection. In a previous report by Tsukasaki et al., the causes of death of eight patients were recurrence of primary ATL in four, pneumonia in one, and unknown in three [2]. The percentage of deaths due to infection in this study was 28.6%; this result differed markedly from that based on Japanese registry data, which showed that only 2% of patients with PTCL-not otherwise specified who underwent ASCT died of infection [8]. This study therefore identified infection as a crucial contributor to treatment-related mortality following ASCT for ATL. This should be kept in mind when developing new therapies involving ASCT.

Most patients in this study underwent autologous transplants before 2010. Therefore, new molecular targeting drugs such as mogamulizumab, lenalidomide, and brentuximab vedotin were not used. In some cases, mogamulizumab can promptly eradicate CCR4-positive peripheral ATL lesions, which may allow for remission induction therapy with only low-intensity chemotherapy to reduce the initial tumor volume, and mogamulizumab may also reduce the contamination of autologous grafts with ATL cells during the collection of peripheral blood hematopoietic stem cells [9]. Therefore, autologous transplantation after remission induction therapy with mogamulizumab may reduce the risk of ATL relapse and the rate of complications due to infection. A new immunotherapy, the Tax peptide-pulsed dendritic cell vaccine, demonstrated good outcomes in patients with aggressive ATL who were in stable condition [10]. This approach may be another viable option to extend the applicability of ASCT for ATL patients who are ineligible for allogeneic SCT.

The limitations of this study include the small number of patients, the fact that most of the transplants were performed in the 1990s, and missing data, such as details of patient characteristics and preconditioning regimens.

Hematopoietic SCT, has been performed for ATL more often in Japan than in any other country worldwide [11,12]. We believe that it is important to report the outcomes and drawbacks of ASCT for ATL as basic data for development of new ATL therapies in the future.

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Long-term follow-up of patients with ATL after autologous stem cell transplantation - Newswise

The research report on the Global Cell Therapy Market from 2021 to 2027 published by MarketsandResearch.biz offers insights about market share analysis related to the revenue, volume, and noteworthy market trends, market dynamics, issues, current trends, and competition analysis. The Cell Therapy market report is expected to witness growth during the forecast year 2021 to 2027. The market report is analyzed with the use of growth prospects and fundamental data. The segments are analyzed using a data synthesis process involving a bottom-down and top-down approach. The precision of market reports is obtained by inculcating analytical tools such as SWOT, Porters five forces, and the PESTEL method.

The Cell Therapy research report encompasses an in-depth analysis of the essential elements that influence the markets growth, such as threats, drivers, entry barriers, obstacles, opportunities, competitive approach, and challenges. The report provides market attractiveness graphs, developments by leading players, R&D strengths, new product success rate, business strategies, and financial ratio. The report offers comprehensive information on the background of the market.The report creates a strong base for users wishing to enter a new market.

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Global Cell Therapy Market 2021 | Scope of Current and Future Industry 2027 The Oxford Spokesman - The Oxford Spokesman

TRUMBULL, Conn., January 27, 2022 /PRNewswire-PRWeb/ --The Rett Syndrome Research Trust (RSRT) is dedicating a further $3.1 million to drive forward the Cure 360 agenda aimed to cure Rett syndrome by continuing to fund the most promising research and investing in a new potentially curative strategy in 2022. RSRT's Cure 360 plan focuses on translating laboratory discoveries about the reversibility of Rett symptoms in mice into clinical successes by incentivizing and attracting biopharmaceutical companies to work on a cure. After rigorous evaluation by RSRT's board and scientific advisors, the most recent awards reflect the organization's commitment to rigorous science and promising business development.

Rett syndrome is a rare, devastating neurological disorder characterized by loss of language, hand skills, and motor function that primarily affects females. As the disorder progresses it brings breathing difficulties, seizures, anxiety, tremors, and gastrointestinal and muscular skeletal problems. Most people with Rett syndrome live into adulthood, requiring round-the-clock care. Rett syndrome results from a mutation in the MECP2 gene on the X chromosome.

RSRT will continue its investment in MECP2 reactivation efforts by Antonio Bedalov, MD, PhD, and Kyle Fink, PhD, of the Fred Hutchinson Cancer Research Institute and University of California, Davis, respectively, with an award of more than $1 million. MECP2 reactivation attempts to awaken the dormant, healthy MECP2 gene on the X chromosome in enough cells to reverse Rett symptoms. This funding deepens RSRT's investment in MECP2 reactivation, one of six genetic-based strategies for a cure, by adding Dr. Fink, who brings expertise from success reactivating a different gene on the X chromosome, to the work and knowledge that Dr. Bedalov has been generating with long-standing RSRT funding. RSRT will continue to look for ways to expand investment in MECP2 reactivation.

Victor Faundez, PhD, of Emory University, has been awarded more than $500,000 for the second phase of his research to identify a Rett syndrome biomarker in spinal fluid. Previous RSRT funding identified differences in certain molecules in the spinal fluid of healthy individuals compared to spinal fluid from people with Rett, and in Rett mouse models compared to normal mice. In this next phase of study Dr. Faundez will determine if these potential biomarkers respond to gene replacement therapy. Through an RSRT-facilitated collaboration between Dr. Faundez and Stuart Cobb, PhD, of the University of Edinburgh, this second phase will also correlate the potential biomarkers with varying levels of MECP2 protein. Having biomarkers that correlate with protein levels and disease severity could be a game-changer for designing and interpreting Rett clinical trials and for attracting increased biopharmaceutical interest.

RSRT has awarded $444,000 to Ciitizen, a technology company that collects, digitizes, and summarizes patient medical records, to create a comprehensive digital natural history study of Rett syndrome. Existing medical records constitute a treasure trove of untapped longitudinal data, which provides a valuable resource for biopharmaceutical companies with Rett drug development programs. This award will gather and analyze medical records for an initial cohort of 120 Rett individuals, however RSRT intends to extend beyond this cohort and collect medical records for Rett families who join RSRT's Rett Syndrome Global Registry. The digitized medical records will be combined with parent-reported data solicited through the registry and together will create a powerful, integrated dataset that will fuel research and drug development programs.

RSRT is investing more than $186,000 in a new strategy to tackle Rett. Joseph Anderson, PhD, of the University of California Davis Medical Center, will remove hematopoietic stem cells from Rett mice, introduce a modified MECP2 gene into the cells with a lentivirus, and transplant the cells back into the mice. Some of these cells naturally migrate to the brain, where they will secrete the modified MECP2 protein to supply nearby cells. This approach has resulted in surprising and encouraging preclinical data for Angelman syndrome, another single-gene neurological disorder with similarities to Rett syndrome. A clinical trial using this type of cell therapy for Angelman syndrome is poised to begin shortly.

In 2022 RSRT will also launch the Rett Syndrome Global Registry, a virtual, caregiver-driven registry that will revolutionize what is known about Rett syndrome by bringing together previously disparate clinical data and tapping into the knowledge of Rett caregivers. The Global Registry will provide a comprehensive view of the disorder by aggregating data from caregivers, digitized medical records, patient cell lines, biosensors, and clinical trials. Access to the Global Registry will be available to the Rett community in early 2022, and caregivers will be able to centralize, track, and share data about their loved one in a clinical trial-quality database built in partnership with clinical trial database developer DSG.

"As the only organization dedicated solely to identifying and accelerating a cure for Rett syndrome, RSRT rigorously evaluates the science and funds comprehensive programs our leading scientific staff and advisors believe can work," said RSRT CEO Monica Coenraads. "These exciting research awards would not be possible without our loyal and generous donors, and the affected families that take action and fundraise. I'm profoundly grateful to every one of them."

RSRT bridges the divide between academic labs and biopharmaceutical companies through its prodigious scientific and business connections, and drug development experience. As a result of RSRT-led collaborations, and scientific and clinical resources funded by RSRT, today there are seven biopharmaceutical companies pursuing a cure for Rett.

LIST OF 2021 AWARDS FOR PROJECTS TO BE CONDUCTED IN 2022 AND BEYOND:

MECP2 REACTIVATION: $1,090,919

Antonio Bedalov / Kyle Fink

Fred Hutchinson Cancer Research Institute / University of California Davis

Reactivation of MECP2

CELL THERAPY: $186,254

Joseph Anderson, PhD

University of California Davis Medical Center

Feasibility of a stem cell approach for the treatment of Rett Syndrome

CLINICAL INITIATIVES: $1,394,914

Victor Faundez, PhD

Emory University

Biomarker Development - Genetic Analysis of the Rett Syndrome Cerebrospinal Fluid Proteome

$584,304

Stuart Cobb, PhD

University of Edinburgh

Biomarker Development - Genetic Analysis of the Rett Syndrome Cerebrospinal Fluid Proteome

$47,014

Ciitizen

Digital Natural History Study

$444,000

Joni N. Saby, PhD / Eric D. Marsh, MD, PhD

Children's Hospital of Philadelphia (CHOP)

Electrophysiological (EEG) Outcome Measures for Rett Syndrome Clinical Trials

$115,906

David Lieberman, MD, PhD

Boston Children's Hospital

Biosensor Development

$67,821

Sasha Djukic, MD, PhD

Albert Einstein College of Medicine

Support for continuing work at the Rett Syndrome Center

$25,000

Bryce Reeve, PhD

Duke University School of Medicine

Development of the Observer-Reported Communication Ability (ORCA) for Rett Syndrome

$15,294 (additional funding)

Coriell Institute

Rett Syndrome biorepository

$53,612 (additional funding)

Harvard Stem Cell Institute

Development of patient derived induced pluripotent stem cell lines

$36,343 (additional funding)

The Jackson Laboratory

Generation and phenotypic assessment of mouse models for Rett Syndrome

$5,620 (additional funding)

MECP2 DUPLICATION: $487,930

The Jackson Laboratory

Testing of siRNA compounds from Khvorova lab for MECP2 Duplication Syndrome

$362,930

Davut Pehlivan, MD

Texas Children's Hospital

Clinical studies in MECP2 Duplication Syndrome as foundation for antisense oligonucleotide drug trials

$125,000

2021 AWARDS TOTAL: $3,160,017

ABOUT THE RETT SYNDROME RESEARCH TRUST

The Rett Syndrome Research Trust is a nonprofit organization with a highly personal and urgent mission: achieving a cure for Rett syndrome and related disorders caused by defects in the MECP2 gene. Since its founding in 2008, RSRT has awarded $64 million, more than any other Rett organization in the world, to leading scientists pursuing targeted research on Rett. RSRT funds and spearheads global scientific and clinical activities advancing the most promising curative approaches. To date, every biopharmaceutical company pursuing a cure for Rett syndrome is doing so because they leveraged discoveries and resources incubated with RSRT funding. A highly efficient nonprofit, RSRT has spent an average of 95 percent of every dollar on its research program. To learn more, visit http://www.reverserett.org.

Media Contact

Emily Dulcan, Rett Syndrome Research Trust, 1 720-773-2196, emily@rsrt.org

SOURCE Rett Syndrome Research Trust

Originally posted here:
RSRT Invests an Additional $3.1 Million in the Plan for a Rett Syndrome Cure - Kilgore News Herald