Category Archives: Stem Cell Therapy

Pain.

It gnawsat you. It drains you. It becomes the focus of your life.

Experiencing a few pain-free moments can be euphoric; it makes you realize how long youve been living with aches and pain. You might wonder how you can find a solution to relieve the pain and regain your freedom from discomfort.

Regenerative Spine and Pain Institute on how to lesson your pain.

Dr. Ronak Patel at Regenerative Spine and Pain Institute wants you to know there are two new revolutionary answers to pain relief.

Both platelet-rich therapy otherwise known as PRP and stem cell therapy give patients new hope by using the bodys powerful healing power to accelerate the battle against pain. Dr. Patel has seen incredible success implementing these cutting-edge treatments on hundreds of patients suffering from pain-related issues.

So if you are suffering fromany of the ailments below, theres a lifeline.

Heres the best news: Neither PRP or stem cell therapy involves drug use with side effects or any surgical procedures.

Both PRP and stem cell treatments use the bodys own healing resources to repair diseased or damaged tissue and the results are quite remarkable.

PRP therapy involves injecting concentrated platelets and growth factors into damaged tissue to stimulate the faster growth of new healthy cells. Platelets are cells that prevent and stop bleeding. If a blood vessel is damaged, the body sends signals to our platelets to get on the job and start the healing. Some call platelets the bodys natural bandage.

So how does PRP therapy work? Its basically drawing a one small vial of blood from the patient and then using a centrifuge to turn it into a potent and concentrated form of platelets. It is then injected back into the patient. Think of it as a boost of your own blood only superpowered.

Recovery time for PRP therapy is far shorter than for surgery. Patients usually experience soreness for a week or so, but the gradual improvement soon begins. Unlike a steroid shot, which gives you immediate relief and quickly wears off, a PRP patient will see pain symptoms improve over a period of months, and up to 80 percent of patients will see relief for up to two years.

Stem cell therapy can be an even more powerful way to harness the bodys healing power. Stem cells are the building blocks for every cell in our body. These powerful cells can be harvested to produce powerful new cells to fight inflammation and disease.

For those suffering from osteoarthritis, stem cell therapy has proven very effective. Thats because the stem cells may help develop new cartilage cells and suppress inflammation. Stem cells can be harvested through a sample of body fat or bone marrow or be harvested from donated umbilical cord tissue.

And yes, you can even augment PRP therapy with stem cell therapy for an even bigger boost!

Stop wondering if youll have to live with your pain forever. Contact Regenerative Spine and Pain Institute today at 609-269-4451 or go to http://www.njpaindoc.com to book an appointment and learn more.

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Regenerative Spine and Pain Institute: Treating Pain with PRP and Stem Cell Therapy - Community News

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today announced the presentation of new long term follow-up data and health-related quality of life scores of patients treated with omidubicel at the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO), being held in Houston, Texas.

These data reinforce our commitment to advance transformational cell therapy research and underscore the potential of our NAM technology platform. Our lead stem cell therapy candidate, omidubicel, addresses the unmet need for patients with hematologic malignancies, demonstrated by the robust and growing body of encouraging clinical evidence, including the long-term follow up data and quality of life improvement, said Ronit Simantov, M.D., Chief Medical Officer of Gamida Cell. As we approach the PDUFA date of January 30, 2023, and upon potential FDA approval, we are prepared to execute our plan that ensures access to those patients who can benefit from omidubicel as quickly as possible.

The long-term, durable clinical benefit of omidubicel was observed at three years across a patient population that typically has a poor prognosis. A study titled, Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials, highlighted long-term follow-up of 105 patients transplanted with omidubicel between 2006-2020 (median follow-up of 22 months). The data demonstrated an overall survival and disease-free survival of 63% (95% CI, 53%-73%) and 56% (95% CI, 47%-67%) at three years, respectively, as well as durable long-term hematopoiesis and immune competence. Learn More

Overall well-being health-related quality of life scores for patients treated with omidubicel demonstrated clinical benefit compared to standard of care. A study titled, Health-Related Quality of Life Following Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel Versus Standard Umbilical Cord Blood featured an analysis of 108 patients that completed validated health-related quality of life (HRQL) surveys on screening and days 42, 100, 180, and 365 post-transplant. Measures of physical and functional well-being and other HRQL scores were more favorable with omidubicel. These data suggest clinically meaningful and sustained improvements in physical, functional, and overall well-being compared to umbilical cord blood transplantation. Learn More

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types including stem cells and natural killer cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cells product candidates (including omidubicel), regulatory filings submitted to the FDA (including the potential timing of the FDAs review of the BLA for omidubicel), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of Gamida Cells product candidates (including omidubicel), and Gamida Cells expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cells goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on May 12, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.

1CIBMTR 2019 allogeneic transplants in patients 12+ years with hematological malignancies.2Gamida Cell market research

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Long-Term Data from Omidubicel Phase 3 Trial Demonstrates Overall Survival and Sustainable Durable Outcomes for Patients with Blood Cancers at the...

New therapies have improved outcomes for patients in frontline treatment for Hodgkin lymphoma, and research now is addressing how subsequent therapies can meet needs of patients who progress after early lines of treatment.

When a patient has progressed through chemotherapytheyve had a stem cell transplant, theyve had brentuximab vedotin [Adcetris; Seagen] and theyve had PD-1 blockade, [physicians] are unsure of what treatments are available, Alex F. Herrera, MD, associate professor in the Division of Lymphoma, Department of Hematology and Hematopoietic Cell Transplantation at City of Hope in Duarte, California, said in an interview with The SOHO Daily News before the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO 2022).

Most patients with Hodgkin lymphoma can be cured by chemotherapy and radiotherapy, but the 20% to 25% of patients who do not respond can be as difficult to treat as those with other cancers, says Herrera. This means reducing the number of patients who relapse is crucial. Two major breakthroughs in this setting have been the introduction of brentuximab vedotin and PD-1 blockade, both of which have advanced to being used in the early lines of therapy in the United States. As the standard becomes using novel agents like brentuximab vedotin in frontline therapy, and maybe someday PD-1 blockade in frontline therapy, it even makes this a more pressing need to find therapies that work after a patient has progressed on those therapies, Herrera says.

Herreras presentation at SOHO 2022 discusses approaches to subsequent therapy for patients who have received brentuximab vedotin and PD-1 blockade. He says that although they have improved the outcomes of patients with Hodgkin lymphoma, patients who are not cured by initial therapies are now often resistant to these treatments as well. More durable responses are also needed for those who do benefit from these therapies, since it is a minority of patients who will have a long-term durable response from these immunotherapies when they are used alone.

Hodgkin lymphoma is a disease that typically affects younger patients, says Herrera. If a patient is resistant to therapy, our goal is to be able to not just get a patient in response, but [to keep] a patient in response for as long as we can.

A wide range of options could offer survival benefit to patients with relapsed disease. Because few new agents have been approved for patients with relapsed disease after brentuximab vedotin and PD-1 blockade, clinical trials play a major role in offering patients the best available care.

Emerging Therapies

New agents such as antibody-drug conjugates (ADCs) could offer a next-line approach. Whereas brentuximab vedotin targets CD30, camidanlumab tesirine (ADCT-301) is another ADC that targets CD25, which is also located on or around Hodgkin lymphoma cells. A phase 2 trial (NCT04052997) is investigating this ADC in patients who previously received brentuximab vedotin and an antiPD-1 agent.

Extending the length of benefit from antiPD-1 agents and overcoming resistance to immune checkpoint inhibitors is an important area of investigation due to the major role antiPD-1 agents now play in treating Hodgkin lymphoma. One potential approach is combining PD-1 blockade with an epigenetic-based or other targeted therapy. These include hypomethylating agents such as decitabine and azacitidine as well as histone deacetylase inhibitors such as vorinostat (Zolinza; Merck) and entinostat.

A study conducted in China (NCT03250962, NCT02961101) showed a significantly longer duration of response and favorable efficacy with the addition of decitabine to the antiPD-1 agent camrelizumab for patients with Hodgkin lymphoma who were PD-1 nave as well as those who were resistant to prior antiPD-1 therapy.1,2 Herrera is helping lead a similar study (NCT05162976) at City of Hope evaluating azacitidine plus nivolumab (Opdivo; Bristol Myers Squibb) to assess how a hypomethylating agent can improve response to immunotherapy. He is also the principal investigator of a phase 1 study (NCT03150329) of vorinostat (Zolinza; Merck) combined with pembrolizumab (Keytruda; Merck) in patients with Hodgkin lymphoma and other lymphoma types.

Herrera says targeted therapy that can extend the duration of benefit from immunotherapy would be an ideal approach in patients with low disease burden or fewer symptoms, because they are not in need of a fast-acting regimen and can benefit from the greater tolerability of immunotherapy. When a patient is resistant to immunotherapy, if we can re-sensitize them with something thats reasonably well tolerated and get them another year or two of response, [and] buy them that time, that might be a valuable option, he says.

For patients with higher disease burden who are more heavily symptomatic, chemotherapy may be the best approach to get a strong, rapid response. Research has also shown that PD-1 blockade can cause patients to become more sensitive to subsequent treatments,3 meaning chemotherapy could be used in combination with immunotherapy or afterward in patients who previously progressed on therapies such as the ABVD combination regimen (doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, and dacarbazine).

Drugs already in use such as lenalidomide (Revlimid; Bristol Myers Squibb), everolimus (Afinitor; Novartis), and temsirolimus (Torisel; Pfizer) can also have a role as subsequent therapies when patients need to achieve a strong anticancer response to improve their outcomes, according to Herrera.

Cellular Therapies

Cellular therapies such as chimeric antigen receptor (CAR) T-cell therapy have made an impact in non-Hodgkin lymphoma and other hematologic malignancies, providing long-term durable remissions in many patients who had few options remaining. Herrera says CD30-targeted CAR T-cell therapies are promising for Hodgkin lymphoma, although they are not yet approved, and several CAR T-cell trials such as the phase 2 CHARIOT study (NCT04268706) are under way. A lingering question for him is whether CAR T-cell products will lead to durable responses in the relapsed population that is resistant to chemotherapy, immunotherapy, and CD30-targeted ADCs.

Herrera says he and his colleagues look for patients who may benefit from trials of cellular therapies that evaluate CAR T cells or natural killer T cells that can result in a durable response. We are trying to refer patients [to trials] or give patients these types of cellular therapies when they are available, he said.

Although the advances in the field have deemphasized the need for patients to receive an allogeneic stem cell transplant, Herrera anticipates it may still have a role as survival is now extended for patients who now may receive 5 or more prior lines of therapy. Four or 5 years later, then you have all these patients who are now progressing on these later-line therapies and they need something else, he says. I think understanding the role allogeneic stem cell transplant may play isa bit of an unresolved question that probably is going to need to be explored again.

There is a range of options available to patients. Theres a lot of drug development happening. And I think that early results are promising and exciting, Herrera says. My talk is about just opening that door and showing folks that theres a lot out there that were studying and that is possible.

REFERENCES:

1. Nie J, Wang C, Liu Y, et al. Addition of low-dose decitabine to anti-PD-1 antibody camrelizumab in relapsed/refractory classical Hodgkin lymphoma.J Clin Oncol. 2019;37(17):1479-1489. doi:10.1200/JCO.18.02151

2. Liu Y, Wang C, Li X, et al. Improved clinical outcome in a randomized phase II study of anti-PD-1 camrelizumab plus decitabine in relapsed/refractory Hodgkin lymphoma.J Immunother Cancer. 2021;9(4):e002347. doi:10.1136/jitc-2021-00234

3. Rossi C, Gilhodes J, Maerevoet M, et al. Efficacy of chemotherapy or chemo-anti-PD-1 combination after failed anti-PD-1 therapy for relapsed and refractory Hodgkin lymphoma: a series from Lysa centers. Am J Hematol. 2018;93(8):1042-1049. doi:10.1002/ajh.25154

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Research Shows Promise for Directing Later Lines of Therapy for Hodgkin Lymphoma - Targeted Oncology

NEW YORK, Oct. 02, 2022 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today that it has submitted to the U.S. Food and Drug Administration (FDA) substantial new information on clinical and potency assay items identified in the Complete Response Letter (CRL) received from FDA in September 2020 to the Biologics License Application (BLA) for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD).

Mesoblast has maintained an active dialog with the FDA since receiving the CRL, and the substantial new information submitted to the Investigational New Drug (IND) file for remestemcel-L in the treatment of children with SR-aGVHD, as guided by FDA, represents a major milestone in the Companys complete response to the FDA. Remestemcel-L has been granted Fast Track Designation and BLA Priority Review from the FDA.

Survival outcomes have not improved over the past two decades for children or adults with the most severe forms of SR-aGVHD.1-3 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children.

The submission summarizes controlled data providing further evidence of remestemcel-Ls ability to save lives, said Dr. Silviu Itescu, Chief Executive of Mesoblast. Additionally, the improved process controls we have put in place to assure robust and consistent commercial product, together with a potency assay that predicts consistent survival outcomes, makes remestemcel-L a compelling treatment for these children.

About Steroid-refractory Acute Graft Versus Host Disease Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, including about 20% in pediatric patients.4,5 SR-aGVHD is associated with mortality as high as 90% and significant extended hospital stay costs.6,7 There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Mesoblast Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome. Rexlemestrocel-L is in development for advanced chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

References / Footnotes

Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or 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 differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblasts preclinical and clinical studies, and Mesoblasts research and development programs; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblasts ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including BLA resubmission), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblasts product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblasts product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblasts ability to enter into and maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblasts expenses, future revenues, capital requirements and its needs for additional financing; Mesoblasts financial performance; developments relating to Mesoblasts competitors and industry; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Mesoblast Submits New Information to FDA IND File in Response to Items in the CRL to the Remestemcel-L BLA for SR-aGVHD - GlobeNewswire

Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel known as exa-cel seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia.

The U.S. Food and Drug Administration (FDA) cleared Vertexand CRISPR to begin a rolling submission of exa-cels BLA a formal request therapy manufacturers make for regulatory approval to introduce a new biologic product in the market.

That submission is planned to begin this November, with completion likely to occur by the end of the first quarter of 2023.

We are pleased to have concluded our exa-cel pre-submission meetings with regulators and are excited that FDA has granted a rolling review, Nia Tatsis, PhD, executive vice president and chief regulatory and quality officer at Vertex, said in a press release.

A rolling submission means the companies can submit sections of the application for review as soon as they are ready, rather than waiting until each and every section is complete prior to submission, as is typically done.

CRISPR and Vertex gained access to this type of review after they met with the FDA as part of a fast track program that aims to speed a therapys development and get it to patients sooner.

SCD and beta thalassemia are two genetic diseases that occur when the body makes either a faulty version of hemoglobin, or none or too little of it. Hemoglobin is the protein in red blood cells that is responsible for oxygen transport.

Not having enough proper hemoglobin causes anemia, which occurs when there is a shortage of red blood cells in the blood.

Exa-cel, formerly known as CTX001, uses hematopoietic stem cells, or blood cell progenitors, that are taken from a patients own peripheral blood. These cells are modified in the lab in such a way that they make high levels of fetal hemoglobin. This version of hemoglobin, produced during fetal development, is more effective at carrying oxygen than its adult counterpart.

When these cells are given back to the patient, in the form of a stem cell transplant, they are expected to drive the production of fetal hemoglobin. This in turn is expected to help ease anemia, lower the need for blood transfusions, and reduce the frequency of painful vaso-occlusive crises (VOCs) that occur in SCD.

The modification is made with the aid of the gene-editing tool CRISPR/Cas9. It uses an RNA molecule that guides an enzyme to a specific point in the DNA sequence of a gene of interest. The enzyme cuts open the DNA sequence at a specific point and removes some of its building blocks.

In exa-cel, this is done in a gene that provides instructions to make BCL11A, a protein that shuts off the production of fetal hemoglobin some time after birth. The modification stops BCL11A from being made, turning the production of fetal hemoglobin back on.

The therapy is being tested in multiple clinical trials as a potential one-time therapy for patients with either SCD or beta thalassemia.

We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease, and one that holds potential to transform the lives of patients with sickle cell disease or beta thalassemia, said Tatsis.

Data from an open-label Phase 1/2/3 clinical trial, called CLIMB121 (NCT03745287), showed that a single dose of exa-cel increased the levels of fetal hemoglobin and prevented VOCs in SCD patients. The ongoing trial, now fully enrolled, involves patients ages 1235 with severe SCD.

The reported side effects were consistent with those of busulfan, a medicine that is used as part of a conditioning treatment regimen carried out in preparation for the stem cell transplant.

A similar clinical trial, CLIMB111 (NCT03655678), is evaluating the safety and efficacy of one-time exa-cel in patients with beta thalassemia of about the same age.

Two other open-label Phase 3 clinical trials CLIMB151 (NCT05329649) and CLIMB141 (NCT05356195) are enrolling patients ages 511 with SCD or beta thalassemia at two locations in the U.S. and Italy. There are plans to extend recruitment to patients as young as age 2 at a later date.

All patients who participated and received exa-cel in any of these clinical trials will have the chance to enter CLIMB-131 (NCT04208529), a follow-up study that will evaluate the long-term safety and efficacy of the therapy for up to 15 years.

In addition to fast track status, exa-cel received regenerative medicine advanced therapy (RMAT) and orphan drug designations from the FDA.

By the end of this year, the companies plan to submit an application requesting marketing approval of exa-cel in Europe. The therapy holds orphan drug status from the European Commission and priority medicines (PRIME) designation from the European Medicines Agency.

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Companies to Seek BLA Approval of Gene-editing Therapy for SCD |... - Sickle Cell Anemia News

Joint instability is a condition that can lead to pain, reduced mobility, and other complications. It can affect people of all ages and genders, but its more common in people with osteoarthritis or rheumatoid arthritis. Many different treatments are available for joint instability today.

Some of the most popular options include medications like glucosamine and NSAIDs, as well as physical therapy and exercise. New treatments are also being developed, including stem cell therapies that aim to improve joint stability. As technology evolves, we will likely see more future breakthroughs in joint instability treatment.

There are several potential benefits of joint instability treatments in the short term, including pain relief and improved quality of life. However, the long-term goal is to improve joint function and prevent future injuries.

What are joint instability treatments, and how do they work?

Joint instability is a common condition that can cause pain and discomfort. The most common type of joint instability is caused by damage to the ligaments or tendons that support the joint. This kind of joint instability can be caused by trauma, such as a fall, or repetitive motions that stress the ligaments or tendons.

As mentioned, joint instability can also be caused by arthritis, which can damage the cartilage that cushionsthe joint. Regardless of the cause, joint instability can be a debilitating condition that limits a persons ability to move freely.

There are several treatment options for joint instability. Physical therapy is often recommended to strengthen muscles and ligaments around the joint. Your doctor may also recommend surgery to repair damaged ligaments or tendons. In some cases, joint replacement is your best bet for recovery. Regardless of your treatment option, it is important to remember that joint instability requires proper medical care.

What are the risks and potential side effects associated with joint instability treatments?

While some people believe treating joint instability is risky and can cause serious side effects, the truth is that they are safe and effective when used as directed. The most common side effect of joint instability treatments is mild discomfort or tenderness at the injection site.

In rare cases, people may experience more serious side effects such as infection, nerve damage, or blood clots if the treated area is not properly sterilized before the treatment. Overall, joint instability treatments are safe and effective when used as directed by a doctor.

How much do they cost on average, and what kind of insurance coverage is available for them?

When it comes to joint instability treatments, many different options are available. For example, some patients opt for physical therapy, while others prefer surgery. In addition, various insurance plans will cover joint instability treatments and costs depending on your treatment type. An example of this would be Assurance IQ senior insurance plans.

However, most joint instability treatments will cost thousands of dollars on average. This is a significant expense, and its crucial to ensure that you have the right insurance coverage before you begin any treatment. Otherwise, you could be left with a hefty bill that you may not afford to pay.

How long do the effects of joint instability treatments last, and are there any long-term risks or side effects associated with them?

While no long-term side effects are associated with joint stabilization treatment, some people can develop a condition called Osteoarthritis. This condition causes the joints to become stiff and painful over time, and it can cause other issues, such as impaired mobility and pain in other joints.

Its important to note that Osteoarthritis does not occur overnight. As people age, the cartilage in their joints starts to wear down, creating more space for water and debris to settle into. Eventually, this creates an environment where bacteria can thrive, leading to Osteoarthritis. While there is no cure for Osteoarthritis, joint stabilization therapy can help reduce pain and improve range of motion while youre still young.

Is surgery better than joint instability treatments for some people with joint problems?

There are several different options for treating joint instability, and the right choice will depend on several factors. For example, some people may be able to manage their joint instability with simple, low-cost treatments such as stretching exercises or using a support brace. In contrast, others may need more invasive, expensive procedures such as surgery.

Depending on your joint problem and overall health, you may find that one approach is clearly better than another. If you have any concerns about your knee condition, you must talk to your doctor about the best course of action for you. Theres no doubt that surgery is the most effective treatment option for joint instability but it can also be the most costly.

While surgery is the only way to get relief from painful and debilitating symptoms caused by arthritic joints, other non-invasive and less expensive treatment options are available if you dont need surgical intervention. These include joint injections and stem cell therapy, which can help relieve pain and improve your quality of life.

Conclusion

Depending on the underlying cause of your joint instability condition, you may be able to treat it with various treatments. The goal of all joint instability treatments is to restore the normal function and structure of your joints. However, each treatment option has its own pros and cons, so its important to talk to your doctor about which is right for you.

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The Truth Behind Joint Instability Treatments - Medical News Bulletin