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AACR: Resistance Mechanisms to CAR-T Cell Therapy ID’d in ALL – HealthDay News
Posted: April 19, 2022 at 2:28 am
TUESDAY, April 12, 2022 (HealthDay News) -- For patients with acute lymphoblastic leukemia with resistance to C19 CAR T-cell therapy, resistance mechanisms that can be detected prior to treatment have been identified, according to a study presented at the annual meeting of the American Association for Cancer Research, held from April 8 to 13 in New Orleans.
Katherine E. Masih, from the National Cancer Institute in Bethesda, Maryland, and colleagues examined differences between resistant and sensitive leukemias using pretreatment bone marrow aspirates from patients enrolled in a clinical trial. Samples were categorized according to patient response; nonresponse was defined as not achieving and maintaining minimal residual disease negativity at day +63. Seven resistant and seven sensitive leukemias were included in the study.
The researchers found that the nonresponders had a distinctive DNA methylation pattern, which was characterized by hypermethylation of PRC2 targets in embryonic and cancer stem cells. Increased accessibility of chromatin was found at regions associated with stem cell proliferation and cell cycling using gene set enrichment analysis of ATAC-seq data. A greater similarity was seen between accessibility patterns of nonresponders to hematopoietic progenitors, including hematopoietic stem cells and common myeloid progenitors. Increased frequency of cell subpopulations expressing a multi-lineage phenotype supported these findings. In addition, relative to responders, there was decreased expression of antigen presentation and processing pathways observed across all leukemic cells.
"Our next steps would be to recapitulate this clinical phenomenon in a mouse model, using different subpopulations of leukemia cells, and then examine if we can overcome this primary resistance with combinatorial or alternative therapies," Masih said in a statement.
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2022-04-18 | NDAQ:BRTX | Press Release | BioRestorative Therapies Inc – Stockhouse
Posted: April 19, 2022 at 2:28 am
MELVILLE, N.Y.,, April 18, 2022 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company” or BioRestorative”) (NASDAQ: BRTX), a clinical stage company focused on stem cell-based therapies, today announced that it has selected 10 of its 15 clinical sites for its Phase 2 clinical trial targeting chronic lumbar disc disease.
BioRestorative’s Phase 2 trial is a double-blind controlled, randomized study to evaluate the safety and preliminary efficacy of a single dose intradiscal injection of BRTX-100. A total of up to 99 eligible patients will be randomized at 15 centers in the United States to receive either the investigational drug (BRTX-100) or control in a 2:1 fashion.
The initial 10 clinical sites for the Phase 2 are located across the United States in the following cities:
We are pleased to have selected our initial clinical sites and look forward to initiating patient enrollment”, said Lance Alstodt, the Company’s CEO. The caliber of clinics that have been selected represents some of the best spine care facilities in the country as this selection process represents a major milestone for BioRestorative. We believe that an intradiscal injection of BRTX-100 has the potential to transform the treatment paradigm for patients suffering from chronic lumbar disc disease.”
About BioRestorative Therapies, Inc.
BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:
Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patient’s bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patient’s damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.
Metabolic Program (ThermoStem®): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT”). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT: Email: ir@biorestorative.com
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2022-04-18 | NDAQ:BRTX | Press Release | BioRestorative Therapies Inc - Stockhouse
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First Patient With Lymphoma Receives Novel Cell Therapy in Large-Scale Trial – Curetoday.com
Posted: April 6, 2022 at 1:56 am
The first patient with lymphoma received treatment with AB-205 in the phase 3 E-CELERATE trial assessing the novel cell therapy with high-dose chemotherapy and blood stem cell transplantation, according to a press release from Angiocrine Bioscience, the manufacturer of the therapy.
AB-205 is a genetically engineered cell therapy that is administered intravenously and consists of allogeneic engineered human endothelial cells. It is currently being assessed in the E-CELERATE trial, in which researchers will evaluate the efficacy and safety of this cell therapy for the treatment of damaged organ vascular stem cell niches from high-dose chemotherapy. Researchers will also see whether the cell therapy can prevent the progression of severe multiorgan complications, which can potentially prolong hospitalization and can be life threatening, according to the release.
According to the trials ClinicalTrials.gov listing, researchers estimate to enroll 148 patients aged 40 years and older with lymphoma. Patients who will be assigned treatment with AB-205 or placebo, and both groups will be treated with standard-of-care preventive and supportive therapies. All patients enrolled in the trial will be candidates for high-dose chemotherapy and autologous hematopoietic cell transplantation.
We are excited to initiate this pivotal phase 3 study, said Dr. Paul Finnegan, chief executive officer of Angiocrine Bioscience, in the release. We look forward to continuing to work with many of the leading cancer centers in the United States as we advance into the final clinical stages of this exciting program.
Patients with lymphoma are often treated with high-dose chemotherapy followed by blood stem cell transplantation with the intention to cure, according to the trials ClinicalTrials.gov listing. Despite this approach, high-dose chemotherapy may cause damage to health tissues resulting in complications related to the immune system, blood, gastrointestinal systems and other organs.
The estimated study completion date for the E-CELERATE trial is January 2023, according to ClinicalTrials.gov.
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Approval Sought for Ibrutinib Combination in Patients With Mantle Cell Lymphoma – Targeted Oncology
Posted: April 6, 2022 at 1:56 am
An application seeking the approval of a new indication for the combination of ibrutinib, bendamustine, and rituximab in adult patients with previously untreated mantle cell lymphoma has been submitted.
A Type II variation application seeking the approval of a new indication for the combination of ibrutinib (Imbruvica), bendamustine, and rituximab (Rituxan) in adult patients with previously untreated mantle cell lymphoma (MCL) who are not candidates for autologous stem cell transplant (ASCT) has been submitted to the European Medicines Agency (EMA).1
Findings from the phase 3 SHINE trial (NCT01776840), which examined the combination in patients with newly diagnosed MCL aged 65 years or older, is the basis of the application. Results showed that the combination demonstrated a significant improvement in progression-free survival (PFS) compared to rituximab alone.
MCL can be a difficult blood cancer to treat, and despite progress in this area of the last few years, an unmet need remains for new treatment approaches, Edmond Chan, MBChB, MD (Res), EMEA therapeutic area lead hematology, at Janssen-Cilag Limited, stated in a press release. This submission to the EMA is a testament to our commitment to deepening the impact ibrutinib can have for patients and represents an important step toward providing patients and health care professionals with the addition of targeted therapy to standard therapy.
The trial included 523 participants with MCL who had clinical stage II, III, or IV disease by Ann Arbor classification.2Eligibility for participation was open to patients with at least 1 measurable site of disease, and an ECOG performance status of 0 or 1. Additionally, patients could not have received prior therapies for their disease.
Patients were randomized 1:1 to receive either ibrutinib or placebo, both in combination with an open-label bendamustine plus rituximab background treatment for a maximum of 6 cycles. Of the participants who achieved a complete (CR) or partial response (PR), open-label background therapy with rituximab maintenance was continued every second cycle for up to 12 additional doses. Patients then received ibrutinib or placebo in addition to this background treatment.
In the experimental treatment arm, bendamustine was given intravenously (IV) at a dose of 90 mg/m2 on days 1 and 2 of cycles 1 through 6, and IV rituximab was delivered at a dose of 375 mg/m2on day 1 of cycles 1 through 6. If either CR or PR was achieved at this time, patients were administered rituximab at the same dose on day 1 of every second cycle for up to 12 additional doses. Additionally, ibrutinib was given orally, once a day at a dose of 560 mg, continually starting on day 1 of cycle 1. Either combination was given until progressive disease, intolerable toxicity, or study completion.
If stable disease following initial chemoimmunotherapy was achieved, patients continued to receive ibrutinib or placebo until progressive disease, intolerable disease, or study end. Patients who experienced disease progression needed to discontinue study treatment.
The primary end point of the trial was PFS, with secondary end points including overall survival, overall response rate, minimal residual disease negativity rate, duration of response, time to next treatment, number of participants affected by adverse events, toxicity, and other various pharmacokinetic measures.
The trial, which met its primary end point, will have its data shared at an upcoming medical conference, according to a press release issued by the Janssen Pharmaceutical Companies of Johnson & Johnson.
As the first approved BTK inhibitor, ibrutinib has now been used to treat more than 250,000 patients globally. It is also the first BTK inhibitor to be studied as a frontline treatment option for patients with MCL. We are committed to the continued development of ibrutinib in B-cell malignancies where unmet needs remain in our efforts to make meaningful differences and change outcomes for patients, Craig Tendler, MD, global head of late development, Diagnostic & Medical Affairs, Hematology & Oncology, at Janssen Research & Development, LLC, added in the press release.
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Bayer reloads Leaps with 1.3 billion to step up investments in biotech innovation – MedCity News
Posted: April 6, 2022 at 1:56 am
Over the past seven years, Bayers investment arm has infused 50-plus companies with more than 1.3 billion. Leaps by Bayer is accelerating its dealmaking pace and Bayer is committing another 1.3 billion, which the multinational corporation estimates will fuel its investment vehicle for two more years.
Bayer announced the capital commitment on Friday during the companys Breakthrough Innovation Forum, an event that covered the corporations plans in healthcare and agriculture. Those two fields were the core focus areas when Bayer formed Leaps in 2015, aiming to invest in companies developing breakthrough solutions to big challenges facing humanity, challenges that the corporation termed leaps. At the start, Bayer identified 10 leaps. The healthcare leaps span genetic diseases; organ and tissue replacement; cancer; neurological disorders; autoimmune diseases and inflammation; and the application of data to health.
Leaps has not tipped its hand on its plans for future investments. But if Bayers dealmaking in recent years is any indication, cell and gene therapies are likely bets. In 2019, Bayer fully acquired BlueRock Therapeutics, a cell therapy developer it had formed three years earlier as a joint venture with venture capital firm Versant Ventures. A BlueRock cell therapy in development for Parkinsons disease began clinical testing last year. BlueRock develops off-the-shelf therapies from induced pluripotent stem cells. The experimental cell therapy DA01 is comprised of neurons that produce dopamine that Parkinsons patients lack. These cells are surgically transplanted in the brain, where its hoped they will produce dopamine and potentially offer a superior alternative to the older dopamine substitutes that are part of the current standard of care.
Bayers gene therapy investment includes the commitment of up to $4 billion to acquire AskBio, a gene therapy biotech whose pipeline most advanced programs are for Parkinsons and the neuromuscular disorder Pompe disease. And earlier this year, Bayer struck up a partnership that enables it to use Mammoth Biosciences CRISPR to develop new in vivo gene editing therapies. The initial focus of the Mammoth alliance is liver diseases. Bayer hasnt abandoned traditional small molecule drugs, but its toolbox for discovering them. Last August, Bayer paid $1.5 billion up front to acquire Vividion Therapeutics, a biotech whose technology finds binding pockets on proteins that had been considered undruggable.
In an interview during the J.P Morgan Health Care Conference in January, Christian Rommel, the head of research and development of Bayers pharmaceuticals division, told MedCity News that Bayers investments are part of a broader strategic transformation oriented around innovative new medicines, some of them in new modalities. Rommel said Bayer will look for additional acquisitions or partnerships that fit this strategy.
Its now in our DNA, he said. We will continue to look for things that enhance our capabilities and success of our pipeline.
Cell therapy investments by Leaps include Indapta, a biotech that is researching therapies employing natural killer immune cells. The most recent Leaps cell therapy investment is Affini-T Therapeutics. In late March, Leaps co-led a $175 million investment the startup, which is based on cell therapy research from the Fred Hutchinson Cancer Research Center.
Leaps has also deployed its cash toward artificial intelligence. It was among the investors in AI-based biotech company Recursion, which went public last year. Other companies whose AI drug discovery work has financial backing from Leaps include Dewpoint Therapeutics, which employs AI to develop biomolecular condensate drugs. Leaps has also shown interest in new approaches to developing protein drugs, co-leading the investment rounds of GRObio and Gandeeva Therapeutics.
The interest of Bayers investment arm in AI extends to healthcare software. Last summer, Leaps led a $90 million investment in Ada, a German startup that is developing AI-based software for checking symptoms. Leaps is also an investor in Transcarent, a healthcare navigation formed started by Livongo Health founder Glen Tullman.
Photo by Flicker user Bex Walton via a Creative Commons license
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Support for blood cancer patients is thin. This needs to change – TimesLIVE
Posted: April 6, 2022 at 1:56 am
While South Africans recently observed Human Rights Day, the notion of basic human rights continues to be a global topic of contention, particularly as the World Health Organisation (WHO) asserts that health is a fundamental human right.
The organisations director-general, Dr Tedros Adhanom Ghebreyesus, expands on this notion well, explaining that enjoying the highest attainable standard of health is a fundamental right of every person.
In SA, the publics right to health is protected by Section 27a of the constitution, which declares that everyone has the right to access to healthcare services. While this is embedded in our rights as citizens, it is often our misinterpretations and the failures of providers that lead to the collapse of such rights.
The right to adequate healthcare is particularly relevant when addressing dread diseases such as cancer, where specialised medical interventions can prolong or save a life. Most notable is the need for interventions to address more pressing cancers, such as hematologic malignancies, or blood cancers. These are orphan diseases and are not nearly as common as the more predominant types, such as breast and prostate cancer.
All cancers require immediate intervention, but once blood cancer is detected in a patient, the clock starts ticking to seek appropriate treatment. While conventional intensive treatments such as chemotherapy have proven useful, they can only drive the disease into remission and patients have a high likelihood of relapse.
Treatments such as blood stem cell therapy have a high probability of eradicating the disease. However, there have been challenges to the procedure in SA.
The department of health promulgated the Medical Schemes Act 131 in 1998, which featured an annexure that defines what stem cell transplantation is and how patients would qualify. It also noted that the act would be reviewed and updated every two years. However, this has not happened.
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Addition of Lenalidomide to SOC Shows Feasibility, Safety in MDS, AML and CMML – Targeted Oncology
Posted: April 6, 2022 at 1:56 am
AZALENA trial shows encouraging response rates, duration of response and survival including patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, and acute myeloid leukemia with MDS-related changes.
Following allogenic stem cell transplant (allo-SCT), lenalidomide (Revlimid) at a dose of 5 mg a day can be added safely to the standard of care combination of azacitidine (Vidaza) and donor lymphocyte infusions (DLI) as salvage therapy for patients with relapse of myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) with MDS-related changes, results from the phase 2 AZALENA study (NCT02472691) show.1
The open-label, single-arm, multicenter study investigated the safety and feasibility of lenalidomide in addition to the standard therapy of azacitidine and DLI as first salvage therapy for this patient population.
We have seen encouraging response rates, duration of response and survival including patients with early and/or hematologic relapse in AZALENA. Azacitidine, lenalidomide and DLI favorable modulated the balance between GvHD and [graft-versus-leukemia; GvL] and is an efficient treatment option for patients with myeloid neoplasms relapsing after allogeneic stem cell transplantation which should be further investigated in randomized trials, stated Thomas Schroeder, MD, assistant professor, section head, Department of Internal Medicine, Division of Hematology/Oncology, medical director of Radiation Oncology, University of New Mexico, during an presentation of the data at the 48th Annual EBMT Meeting.
AZALENA enrolled 50 participants aged 18-99 with first relapse of de novo or therapy-related MDS, CMML or AML in various participating centers across Germany.2 Other requirements for inclusion in the study included possibility of DLI, no previous therapy for relapse after allo-SCT, an ECOG status of 0-2, and no uncontrolled infection at the time of enrolment.
Of the patients enrolled in the trial, the median age was 63 years. Forty-six percent of the patients with AML (n = 23), 48% with MDS (n = 24) and 6% with CMML (n = 3). A majority of patients (72%) were not in remission at the time of transplantation but received standard-dose conditioning (68%).
Patients included in the study received a median of 275 treatment cycles and were given azacitidine as a standard of care at 75 mg for 7 days every 28 days for up to 8 cycles. DLIs were then administered after cycle 4, 6 and 8 at a dose of 5-10x105CD3+/kg (1st DLI), 1-5x106CD3/kg (2nd DLI) and 5-15x106CD3/kg (3rd DLI).
The investigational drug, lenalidomide, was also started on day 1 for 21 days every 28 days for 8 cycles at most. A starting dose of 2.5 mg of lenalidomide per day was given to the first 10 patients. If no dose limiting toxicity was observed at the time of a first interim analysis, the next 10 patients were to be treated with 5 mg per day. In case of no DLT after a second interim analysis, the remaining 30 patients were treated with 5 mg per day.
The primary end point of the study was to evaluate safety including the number, types, and severity of adverse events (AEs), graft versus host disease (GvHD) and hospitalization. The secondary end point of the study examined safety in regard to response, duration of response, and overall survival.
Findings revealed there to be an overall response rate of 56% (n = 28). The time to complete response was 113 days seen in the 50% of patients with a median of 4 cycles. A total of 20 patients with CR had received DLI. Of the patients included, 6% showed a partial response. The median OS for all patients was 21 months and the 1-year OS rate was 65%. Patients who achieved remission had a superior survival versus 9.7 months.
In regard to toxicity, almost a third of the patients had either grade 3 or 4 neutropenia (30%) or thrombopenia (38%) at the time of study entry. Of the 275 treatment cycles, 246 included 89% of patients receiving the combination therapy, and in 29 cycles, lenalidomide was omitted in 5 patients (11%).
Lenalidomide-related AEs which were grade 3 or higher were seen in some patients including 38% of patients (n = 19) having to be hospitalized at some point in the duration of the trial. Grade 3 AEs seen in patients included infections (20%), febrile neutropenia (10%), gastrointestinal disorders (6%), and nervous system disorders (6%). Grade 4 AEs consisted of infections (4%) and febrile neutropenia (2%).
Looking at GvHD, 30% of the patients suffered from acute GvHD and 38% chronic GvHD. Time to GvHD onset was approximately 112 days the majority of patients with GvHD (77%, n = 20) had received DLI.
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StemCyte, a leading regenerative cell therapy company, has submitted its application for a biologics license (BLA) for its allogeneic umbilical cord…
Posted: March 25, 2022 at 2:10 am
DetailsCategory: DNA RNA and CellsPublished on Wednesday, 23 March 2022 13:40Hits: 516
The BLA has been accepted for quality review and StemCyte anticipates licensure in 2023
BALDWIN PARK, CA, USA I March 22, 2022 I The BLA (Biologics License Application) for the "HPC-Cord Blood" product, was submitted to FDA on January 7th, 2022, and StemCyte was officially notified on March 8th, 2022, that the submission was acceptable to enter the biologics license quality review process.
The "HPC-Cord Blood" is an umbilical cord blood hematopoietic stem cell product intended for unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system. Since the first successful use of umbilical cord blood to treat Fanconi Anemia patients in 1988, there have been more than 40,000 successful umbilical cord blood transplants worldwide for the treatment of diseases of the hematopoietic and immune systems as well as inborn metabolic diseases.
In the past 20 years, StemCyte has provided more than 2,200 cord blood units for transplantation.
In the past 20 years, StemCyte has provided more than 2,200 cord blood units for transplantation to 1 in 20 patients worldwide who receive umbilical cord blood transplants. StemCyte's products consistently meet the quality standards of international accrediting bodies and are recognized and trusted as safe and effective by at least 350 transplant centers around the world including such well-known medical centers as: Duke University Hospital, UCLA Medical Center, Taiwan Chang Gung Memorial Hospital, National Taiwan University Hospital.
StemCyte is a regenerative cell therapy company that is developing cell therapy pipeline products as well as providing both public and private umbilical cord blood banking services. In addition to the US FDA approved Phase II multi-national and multi-center human clinical trial for its investigational spinal cord injury treatment, the cell therapy product lines also include several other human clinical trials in progress outside the US for treatment of acute ischemic stroke, chronic stroke, and cerebral palsy. Development of a cancer immunotherapy is planned to begin this year. StemCyte's cord blood bank is multi-ethnic with the matching rate for patients from different ethnic groups relatively high compared with other public cord blood banks. StemCyte considers umbilical cord blood banking its core competence, while actively pursuing new regenerative cell therapy indications. StemCyte's mission is to continue to develop its unique cord blood banking capabilities to achieve the goal of delivering lifesaving therapies to patients who suffered degenerative and other life-threatening diseases.
SOURCE: StemCyte
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StemCyte, a leading regenerative cell therapy company, has submitted its application for a biologics license (BLA) for its allogeneic umbilical cord...
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Stem Cell Research in India Size and Outlook 2022, Manufacturing Demand, Industry Growth, Production Volume, Future Dynamics with Challenges and…
Posted: March 25, 2022 at 2:10 am
Kenneth Research published a report onStem Cell Research in Indiawhich gives detailed analysis of the present market landscape. The report which is evaluated for the forecast period, i.e.,2022-2031, provides an in-depth analysis of the market, based on size, volume, latest trends, opportunities and challenges associated.
U.S. Market recovers fast; In a release on May 4th 2021, the U.S. Bureau and Economic Analysis and U.S. Census Bureau mentions the recovery in the U.S. International trade in March 2021. Exports in the country reached $200 billion, up by $12.4 billion in Feb 2021. Following the continuous incremental trend, imports tallied at $274.5 billion, picked up by $16.4 billion in Feb 2021. However, as COVID19 still haunts the economies across the globe, year-over-year (y-o-y) average exports in the U.S. declined by $7.0 billion from March 2020 till March 2021 whilst imports increased by $20.7 billion during the same time. This definitely shows how the market is trying to recover back and this will have a direct impact on the Healthcare/ICT/Chemical industries, creating a huge demand forStem Cell Research in IndiaProducts.
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According to the statistics by the World Bank, the current health expenditure (% of GDP) around the world increased from 9.08% in 2001 to 9.84% in 2018. Additionally, the current health expenditure per capita (current US$) increased from USD 492.99 in 2001 to USD 1110.84 in 2018. Moreover, growing concern for deaths caused due to various diseases and the need for treatment that can lower the crude death rate, which in the year 2019 recorded close to 7% (per 1000 people), are also anticipated to drive the market growth during the forecast period.
Final Report will add the analysis of the impact of COVID-19 on this industry.
The stem cell research in India is in its nascent stage and is gradually on a growth path of acceptance by people. The market is still in a phase of conducting research to establish itself as one of the best therapies for the widely prevalent incurable lifestyle diseases. Awareness campaigns and doctors are playing a key role educating people and especially would-be parents about the benefits associated with preserving stem cells.
The report begins with the introduction section which offers a brief insight of the concept of stem cell therapy and banking, the conventional sources of stem cells and the current and future therapeutic solutions for the most risky diseases. It then moves to the market overview section which provides an insight of the Indian stem cell therapy market, with highlight on the market size and growth. It also covers the market size and growth prospect of the stem cell banking market in India. In addition to these, the report gives a snapshot of the current and expected stem cell banking customers.
An analysis of the drivers explains the factors for growth of the industry that include favourable regulatory environment, high patient population, stem cell application in drug development, rising consumer awareness, a rise in medical tourism and an increase in research and development expenditure to ensure stem cell therapy as a viable treatment for the numerous diseases. The key challenges include high cost of therapy, capital intensive market and high development costs of stem cell resulting in slow progress of the market.
The next section of the report provides the progressive stages that the stem cell research is undergoing and the respective business prospect for each of the stages of business involved. The report then covers the key trends that are being observed in the stem cell research. Collaborations and partnerships are becoming a trend that is helping in healthy progress of the business, investments are pouring in from government, international organizations and pharmaceutical companies, serious efforts are being undertaken to raise awareness standards regarding stem cell therapy and the move is being popularized by stem cell banks by providing easy financing opportunities for banking stem cells.
Significant developments have emerged wherein new sources of stem cells have been identified dental stem cells and menstrual blood stem cells which have been discussed along with its benefits in comparison to cord blood stem cells.
The competition section gives overview of stem cell banks and stem cell research organizations in the country. The report highlights the features of the major players operating in the market in detail. It includes elaborate profile of the major players in the market along with their financial analysis. Porters Five Forces Analysis has been incorporated for a brief but effective understanding of the market scenario.
Thereafter, the report has a section on the recent developments that have taken place in the recent past in the stem cell research area followed by a section on strategic recommendations created after a thorough analysis of the industry. The strategic recommendations section focuses on some effective strategic decisions which can be taken up by companies to increase their market shares like public private partnerships, taking up measures to increase the number of cord blood samples and collaborations to a smooth progress of the stem cell research that will have a positive impact on the therapeutic segment.
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The Market report answers the following questions:
What is the aim of the report?
What aspects regarding the regional analysis Market are included in this report?
Based on what factors are the key market players assessed in this report?
What is the key information extracted from the report?
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Stem Cell Research in India Size and Outlook 2022, Manufacturing Demand, Industry Growth, Production Volume, Future Dynamics with Challenges and...
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SQZ Biotechnologies Announces $2 Million Grant From the National Institutes of Health to Develop a Novel, Scalable Cell Replacement Therapy for…
Posted: March 25, 2022 at 2:10 am
WATERTOWN, Mass.--(BUSINESS WIRE)--SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, today announced that it has been awarded a $2 million SBIR Phase II grant from the National Institute of General Medical Sciences, a division of the National Institutes of Health. Awarded through a competitive process, the two-year grant will support the development of cell engineering methods that are designed to reprogram a patients own immune cells directly into dopamine-producing neurons, a potential novel therapeutic approach for the treatment of Parkinsons disease.
Directly creating dopamine-producing neurons by reprogramming a patients own immune cells would be a major breakthrough and could support a new Parkinsons disease treatment paradigm, said Jonathan Gilbert, Ph.D., Vice President and Head of Exploratory Research at SQZ Biotechnologies. Unlike alternative allogeneic cell replacement approaches in development for Parkinsons disease, by using a patients own cells, treatment might not require chronic immunosuppression. Moreover, in altering cell fate with RNA-based cell engineering methods, no changes to the genome are likely to occur that could carry long-term risks.
Reprogramming a patients cells to replace lost or diseased cells has significant therapeutic potential. Beyond Parkinsons Disease, applications for cell replacement therapies include Multiple Sclerosis and Type 1 diabetes. However, traditional expensive, time-intensive, and inefficient cell reprogramming methods has hindered clinical progress and patient impact.
At the 2021 International Society for Stem Cell Research annual meeting, the company presented preclinical data showing that proprietary Cell Squeeze technology can be used to generate neurons from induced human pluripotent stem cells through the delivery of an mRNA encoding for a fate-specifying transcription factor.
With the support of the NIH grant, and building upon our experience in multiplex engineering of immune cells, SQZ researchers will attempt to generate dopaminergic neurons directly from somatic cells. The Cell Squeeze technology may allow for a unique complex combination of transcription factors, dosing, and timing.
About SQZ BiotechnologiesSQZ Biotechnologies Company is a clinical-stage biotechnology company focused on unlocking the full potential of cell therapies for patients around the world and has active programs in Oncology, Autoimmune and Infectious Diseases, as well as additional exploratory initiatives to support future pipeline growth. The companys proprietary Cell Squeeze technology offers the unique ability to deliver multiple biological materials into many cell types to engineer what we believe can be a broad range of potential therapeutics. With demonstrated production timelines under 24 hours and the opportunity to eliminate preconditioning and lengthy hospital stays, our approach could significantly broaden the therapeutic range and accessibility of cell therapies. The companys first therapeutic applications seek to generate target-specific immune responses, both in activation for the treatment of solid tumors and infectious diseases, and in immune tolerance for the treatment of autoimmune diseases. For more information, please visit http://www.sqzbiotech.com.
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements relating to events our platform development, our product candidates, project funding, preclinical and clinical activities, progress and outcomes, development plans, manufacturing, clinical safety and efficacy results, therapeutic potential, market opportunities and disease prevalence. These forward-looking statements are based on management's current expectations. Actual results could differ from those projected in any forward-looking statements due to several risk factors. Such factors include, among others, risks and uncertainties related to our limited operating history; our significant losses incurred since inception and expectation to incur significant additional losses for the foreseeable future; the development of our initial product candidates, upon which our business is highly dependent; the impact of the COVID-19 pandemic on our operations and clinical activities; our need for additional funding and our cash runway; the lengthy, expensive, and uncertain process of clinical drug development, including uncertain outcomes of clinical trials and potential delays in regulatory approval; our ability to maintain our relationships with our third party vendors and strategic collaborators; and protection of our proprietary technology, intellectual property portfolio and the confidentiality of our trade secrets. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K, as updated by our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2021 and other filings with the U.S. Securities and Exchange Commission could cause actual results to differ materially from those indicated by the forward-looking statements. Any forward-looking statements represent management's estimates as of this date and we undertake no duty to update these forward-looking statements, whether as a result of new information, the occurrence of current events, or otherwise, unless required by law.
Certain information contained in this press release relates to or is based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party sources to be reliable as of the date of this press release, we have not independently verified, and we make no representation as to the adequacy, fairness, accuracy or completeness of any information obtained from third-party sources.
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