Global Stem Cell and Primary Cell Culture Medium Market Dynamics, Production, Supply and Demand Forecast covered in the Latest Research Available at…

Stem Cells are a class of cells that have unlimited or immortal self-renewal ability, capable of producing at least one type of highly differentiated progeny cells. Primary Cells are cells that are cultured immediately after removal from the body. Stem Cell and Primary Cell Cultures are specialized systems, and as such developing and manufacturing media for these systems come with inherent complexities.

The global Stem Cell and Primary Cell Culture Medium market is valued at US$ xx million in 2020 is expected to reach US$ xx million by the end of 2026, growing at a CAGR of xx% during 2021-2026.

Access more details about this report at: https://www.themarketreports.com/report/global-stem-cell-and-primary-cell-culture-medium-market-research-report

(This is our latest offering and this report also analyzes the impact of COVID-19 on Stem Cell and Primary Cell Culture Medium market and updated by the current situation, especially the forecast)

The research report has incorporated the analysis of different factors that augment the markets growth. It constitutes trends, restraints, and drivers that transform the market in either a positive or negative manner. This section also provides the scope of different segments and applications that can potentially influence the market in the future. The detailed information is based on current trends and historic milestones. This section also provides an analysis of the volume of production about the global market and also about each type from 2015 to 2026. This section mentions the volume of production by region from 2015 to 2026. Pricing analysis is included in the report according to each type from the year 2015 to 2026, manufacturer from 2015 to 2020, region from 2015 to 2020, and global price from 2015 to 2026.

A thorough evaluation of the restrains included in the report portrays the contrast to drivers and gives room for strategic planning. Factors that overshadow the market growth are pivotal as they can be understood to devise different bends for getting hold of the lucrative opportunities that are present in the ever-growing market. Additionally, insights into market experts opinions have been taken to understand the market better.

The major players in the market include Merck, STEMCELL Technologies, Irvinesci, Cell Applications, Inc, Biological Industries, Miltenyi Biotec, Swiss Medica Clinic, Promocell, Creative Biolabs, Lifeline Cell Technology, ScienCell Research Laboratories, Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix), etc.

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Global Stem Cell and Primary Cell Culture Medium Market: Regional Analysis

The report offers in-depth assessment of the growth and other aspects of the Stem Cell and Primary Cell Culture Medium market in important regions, including the U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, Taiwan, Southeast Asia, Mexico, and Brazil, etc. Key regions covered in the report are North America, Europe, Asia-Pacific and Latin America.

The report has been curated after observing and studying various factors that determine regional growth such as economic, environmental, social, technological, and political status of the particular region. Analysts have studied the data of revenue, production, and manufacturers of each region. This section analyses region-wise revenue and volume for the forecast period of 2015 to 2026. These analyses will help the reader to understand the potential worth of investment in a particular region.

Global Stem Cell and Primary Cell Culture Medium Market: Competitive Landscape

This section of the report identifies various key manufacturers of the market. It helps the reader understand the strategies and collaborations that players are focusing on combat competition in the market. The comprehensive report provides a significant microscopic look at the market. The reader can identify the footprints of the manufacturers by knowing about the global revenue of manufacturers, the global price of manufacturers, and production by manufacturers during the forecast period of 2015 to 2019.

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Global Stem Cell and Primary Cell Culture Medium Market Dynamics, Production, Supply and Demand Forecast covered in the Latest Research Available at...

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Trusted By Experts MARK HYMAN, M.D. Thirteen-time#1 New York Times bestselling author When I decided to undergo stem cell therapy for a nagging injury, I put the word out to people I trust to direct me to the best of the best. Harry Adelson, N.D. was the name that kept coming back to me. The staff members at Docere Clinics are radiantly happy and refreshingly efficient. Dr. Adelson is professional, confident, and caring. Overall, I give Docere Clinics an A+ and enthusiastically recommend them. CARRIE DIULUS, M.D. Spine Surgeon As a Cleveland Clinic trained orthopedic spine surgeon, when I heard about stem cell injections being performed into intervertebral discs and epidurals, I had to see it with my own eyes. When I visited Docere Clinics and watched Dr. Adelson operate, what I discovered was pleasantly surprising. Dr. Adelson is a skilled spine injector, practices flawless sterile technique, is honest and upfront, and is completely professional. Im happy to call Dr. Adelson a trusted colleague and a friend. Vishen Lakhiani Founder Of Mindvalley, Entrepreneur & Education Activist, And Author Of The International Bestseller, The Code Of The Extraordinary Mind I had a painful sciatica that made me miserable. Based on recommendations from people I trust, I sought Dr. Harrys help. To be honest, I was somewhat skeptical because the pain was so intense. To my surprise, after treatment the pain resolved completely. I could hardly believe it! Additionally, the low back pain Id had for almost ten years was eliminated and to this day there is still not a trace of it. I really appreciate the heart of Docere Clinics and the amazing sense of comfort and kindness that surrounds the organization.

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Zaia Draws on Decades of Innovation in Infectious Disease for Breakthroughs in Gene Therapy – AJMC.com Managed Markets Network

Interview by Maggie L. Shaw

Known as a gene therapy pioneer, Zaia has spent almost40 years at City of Hope, in Duarte, California. He was first drawnby the promise of studying cytomegalovirus. Over the decades, hisgroundbreaking research has encompassed HIV/AIDS, cellular gene transfer therapy, immunotherapy, bispecific antibodies, andnow hyperimmune globulin for workers on the frontlines of thecoronavirus disease 2019 (COVID-19) pandemic.

Known as a gene therapy pioneer, Zaia has spent almost40 years at City of Hope, in Duarte, California. He was first drawnby the promise of studying cytomegalovirus. Over the decades, hisgroundbreaking research has encompassed HIV/AIDS, cellular gene transfer therapy, immunotherapy, bispecific antibodies, andnow hyperimmune globulin for workers on the frontlines of thecoronavirus disease 2019 (COVID-19) pandemic. Zaia was recentlyawarded $750,000 from the California Institute for RegenerativeMedicine to study the potential use of convalescent plasmain patients with COVID-19, as well as to create the COVID-19 Coordination Program to aid in this effort.1

Zaia spoke at length about the crucial connections betweenbasic research in HIV/AIDS and developments in gene therapy.This interview has been edited slightly for clarity.

EVIDENCE-BASED ONCOLOGY (EBO): We know that HIV does notelicit a protective immune response in the body. Do you think itis possible to overcome nature in this regard, to trick the immunesystem into fighting HIV to the degree that it can overcome it, suchas interrupting the binding of the virus to the CD4 receptor?

ZAIA: So, lets take that question apart. There is an immuneresponse in the body to HIV, but its just not protective. Thequestion is, why isnt it protective? And could you overcome thatdeficiency? So, one aspect to understand is the ability of the virusto continuously mutate.

If youre familiar with RNA replication, it doesnt have highfidelity, meaning that mistakes occur while copying the newstrand of RNA. Whereas DNA replication has high fidelity, meaningthat once you copy it, its virtually word-for-word precise with onlyan occasional mutation. So, whenever the virus makes 10,000 basepair copies, theres 1 mistake. But the virus is actually only 10,000base pairs long in terms of its RNA. So that means theres about 1naturally occurring mistake in every new virus. And since therecould be billions of new viruses made, there will be literally allthese mutations a day, some of which could help the virus survive.Since the barriers that put up against the virus for continuing itsreplication are limited (eg, immune response, antiviral medications),its not that hard to imagine that a mutation could occurthat gets around a specific barrier When this occurs, it is calledantigen escape or drug resistance.

The deeper understanding of this question is, in the immunerecognition of the virus, the T lymphocytes have a receptor forthe virus called the T-cell receptor; its really an antigen receptorthat can see a specific peptide on the surface of the virus or onthe infected cell. So, the T-cell receptor itself is the problem. Itsexerting this selection, but it is not very flexibleits rigid. Its anall-or-none thing. If the virus can mutate its protein slightly, thenthat peptide never fits into the receptor. Its kind of like a lock andkey. So, we need a T-cell receptor thats more resistant to antigenescape. Could you make an artificial receptor, called a chimericantigen receptor (CAR), that would better resist antigen escape?At City of Hope, weve been trying to make a T-cell receptor thatyou [could] paste on to the T cells genetically so they are better atresisting this inability to detect the mutated part of the virus.Is there a part of the virus that is resistant to mutation? Thereprobably is. The key surface protein is called gp120. And someantibodies are very broadly reactive to all viruses, all HIV viruses.So, a broadly neutralizing antibody can detect multiple different gp120s, all of which are slightly differentbut theres somecommon feature thats recognized by the broadly neutralizingantibodies. If you put that on a T cell, as a chimeric antigenreceptor, the T cell might be more resistant to antigen escape bythe mutating virus.

The other possibility is, what if you use the CD4 receptor? Thatsan almost immutable part of the virus biology, because if thevirus didnt bind to the CD4 receptor, it probably wouldnt be HIV.It would be a different virus, a different lentivirus. But there areCAR T-cell receptors that utilize not the antibody to find the virus,but the CD4 receptor itself to find the virus. In other words, if youput CD4 on the surface of a CD8 cell, it would find all the gp120because the CD4 and gp120 would bind to each other. So that isactually another concept that can be utilized, and thats currentlyin clinical trials at the University of Pennsylvania.2

So, in summary, I think the trick would be to utilize a modificationof a T-cell receptor that would avoid the ability of the virus tomutate around the classical T-cell receptor and allow the immunesystem to see the virus and to control it.

EBO: The holy grail of HIV research for nearly 40 years has beento produce a vaccine. The Thai trial (RV144)3 has been the onlytrial thus far to show that a preventive HIV vaccine is possible.What have been the barriers to reproducing these results? Why hasachieving the goal of developing an AIDS vaccine been so elusive?

ZAIA: Those are good questions. I dont think anyone knows [theanswers] for sure. But 2 factors are probably important. Again,you go back to the virus mutation issue. Its continuous, and now[its] in the presence of immune pressure placed on the virus bythe vaccine. Youll get selection for these mutations. So, I guessthe question is, did the vaccine make an immune response to themost immutable parts of the virus? Probably notvirus mutationis not the only answer to why vaccines fail. It seems to be somethingmore basic than that.

For example, do vaccines induce mucosal immunity? We have amucosal immunity to many viruses that come in contact with ourmucous membranes via nose, throat, etc. Well, HIV would be inthe mucous membranes of the genital tract and rectum, [usually].So, are these vaccines really making a mucosal immunity where its needed? Thats a possible explanation for why the vaccinesare not working.

An area that people just dont understand at the present timeis why you can make a vaccine for certain viruses that wouldnormally come through the respiratory tract and not be able tomake a vaccine for others. Its relevant to COVID. Will we be ableto make an immune response to COVID when we know that theinitial entry point is through the nasal passages? Thatll be themillion-dollar question.

EBO:Can you explain what a lentiviral vector is? Why is it that HIVcan be inactivated outside the body to be used as a safe lentiviralgene therapy, but we cant do the same with the virus internally?

ZAIA: A lentivirus has a certain structure and is made of RNA andprotein. And it fulfills the requirements from some taxonomiccommittee that defines what a lentivirus is. Basically,it is a virus that can do 1 thing very usefully: it canreverse the RNA to DNA, and the DNA can then beintegrated into the host DNA, become part of the host. And that integration is due to an enzyme calledintegrase. So, it has an RNA that also encodes for thisintegrase as well as reverse transcriptase; it turns RNA into DNA. That was a famous discovery at onepoint; it won the Nobel Prize. And so thats whatmakes it a lentivirus.

Now, you can inactivate it in the sense of makingit safe. It has only 9 major proteins, and thoseproteins are important in those elements that Ijust mentioned and in leading to its pathogenicity.You can remove them and still have some of theelements that you need. For example, you couldleave the integrase but remove other things, whichmay make the virus able to replicate and lead toAIDS. But now youd have an incomplete virus thatyou can put a gene into, and it can be delivered to the cell, because the virus can still get into the cell.And it can still have an integrase, which can helpyou integrate that message or that gene into the hostcell. But the virus cant replicate. It has all the otherparts of it that are needed, but replication has beenremoved. You basically neuter the virus by removingcritical genes. Its still allowed to be a good virusfor your useit can get it into the cell, deliver itspayloadbut it just cant replicate.

The question is, why cant we inactivate certainof these critical genes that are important forreplication? And, in fact, you canin vitro. You cancertainly put in inhibitors of all the various proteinsof a virus. Some of those are called small inhibitoryRNAs (siRNA), which are known to block specificallydifferent proteins of a virus and almost any virus.The question is, how do you deliver that siRNA to allcells that are infected? If you have trillions of cellsinfected, how would you get to the last one? Thatsthe issue. So, you can do it in a test tube, but you justcant do it in a human organism.

Now, you might ask, why are you able to getcertain things to work for acute lymphoblasticleukemia but not for HIV?

Well, I think its because the virus can becomelatent and invisible to most systems and theleukemia cannot. The leukemia is robustly growingand expressing all of its proteins and enzymes,and so we [can fight it with] chemicals and otherthings like CAR T cells, and they will destroy thosecells. The HIV is holed up in an inactive form, aso-called reservoir, and that reservoir is the problem.Once its in there, its like a snake in its hole. Youcannot get to it.

EBO: In a 2016 commentary,4 you discuss thefindings by Yang et al that the immune-mobilizingmonoclonal T-cell receptor, or ImmTAV, couldbe an effective new agent against HIV/AIDS dueto its bispecific antibodybindingproperties. It was shown to haveactivity against both p17-expressingactivated and resting CD4 cells. Is theagent proposed by Yang possible andwithout neurotoxicity?

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Zaia Draws on Decades of Innovation in Infectious Disease for Breakthroughs in Gene Therapy - AJMC.com Managed Markets Network

Mayo Clinic Study of Humanigen’s Lenzilumab Shows Rapid Recovery and Discharge in Severe and Critical COVID-19 Patients – Business Wire

BURLINGAME, Calif.--(BUSINESS WIRE)--Humanigen, Inc., (HGEN) (Humanigen), a clinical stage biopharmaceutical company focused on preventing and treating cytokine storm with lenzilumab, the companys proprietary Humaneered anti-human granulocyte macrophage-colony stimulating factor (GM-CSF) monoclonal antibody, announced data on the first clinical use of lenzilumab in 12 COVID-19 patients. The manuscript, titled First Clinical Use of Lenzilumab to Neutralize GM-CSF in Patients with Severe and Critical COVID-19 Pneumonia was published online at medRxiv.org (www.medrxiv.org/content/10.1101/2020.06.08.20125369v1). Patients showed rapid clinical improvement with a median time to recovery of five days, median time to discharge of five days and 100% survival to the data cut-off date. Patients also demonstrated rapid improvement in oxygenation, temperature, inflammatory cytokines and key hematological parameters consistent with improved clinical outcomes.

Dr. Zelalem Temesgen, Professor of Medicine at Mayo Clinic and one of the key authors of the study, said, Lenzilumab use was associated with improved clinical outcomes and oxygen requirement, with no reported mortality. We did not observe any treatment-emergent adverse events attributable to lenzilumab and it was well-tolerated. Based on the pathophysiology of cytokine storm following SARS-CoV-2 infection, along with work conducted at Mayo Clinic on GM-CSF depletion in CAR-T therapy, lenzilumab may offer a rational approach to ameliorate the consequences of cytokine storm in COVID-19.

Dr. Cameron Durrant, chief executive officer of Humanigen, stated, It is extremely encouraging to see this initial group of high-risk patients with severe and critical COVID-19 pneumonia show clinical improvement on lenzilumab, and at the data cut-off point, 11 of them discharged from the hospital. All 12 patients had at least one risk factor associated with poor outcomes, such as age, smoking history, cardiovascular disease, diabetes, chronic kidney disease, chronic lung disease, high BMI, and elevated inflammatory markers, with several patients having multiple such risk factors.

All patients were hospitalized in the Mayo Clinic system and had severe or critical pneumonia as a result of COVID-19. They were also viewed as being at high risk of further disease progression. All patients required oxygen supplementation and had elevation in at least one inflammatory biomarker prior to receiving lenzilumab. All patients had at least one co-morbidity associated with poor outcomes in COVID-19 and several patients had multiple co-morbidities: 58% had diabetes mellitus, 58% had hypertension, 58% had underlying lung diseases, 50% were obese (defined as a BMI greater than 30), 17% had chronic kidney disease and 17% had coronary artery disease. The median age was 65 years.

More details on the companys programs in COVID-19 can be found on the companys website at http://www.humanigen.com under the COVID-19 tab, and details of the Phase III potential registration study can be found at clinicaltrials.gov using ClinicalTrials.gov Identifier NCT04351152.

About COVID-19

COVID-19 is an infectious disease caused by SARS-CoV-2. COVID-19 has become a global pandemic, with almost 8 million confirmed cases and almost 450,000 deaths reported to date. Patients with severe cases of COVID-19 experience severe viral pneumonia that can progress to acute respiratory distress syndrome (ARDS), respiratory failure and death.

In severe and critical patients with COVID-19, published research suggests GM-CSF as the key link between pathogenic Th1 cells and inflammatory monocytes, which secrete additional GM-CSF1. Lenzilumab is a late clinical-stage, monoclonal antibody targeting GM-CSF, a pro-inflammatory cytokine up-regulated in the serum of COVID-19 patients2. The percentages of certain GM-CSF-expressing cells are significantly higher in the blood of ICU-admitted COVID-19 patients compared with healthy controls and are more pronounced in ICU-admitted COVID-19 patients versus non-ICU patients2.

1. Zhou Y, Fu B, Zheng X, et al. Aberrant pathogenic GM-CSF+ T cells and inflammatory CD14+CD16+ monocytes in severe pulmonary syndrome patients of a new coronavirus. Pre-Print. 2020. https://doi.org/10.1101/2020.02.12.945576.

2. Huang C, Wang Y, Li X, et al. Clinical features of patients infected with 2019 novel coronavirus in Wuhan, China. Lancet. 2020;395(10223):497-506. doi:10.1016/s0140-6736(20)30183-5.

About Humanigen, Inc.

Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of inflammation and cancers via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. We believe that our GM-CSF neutralization and gene-editing platform technologies have the potential to reduce the inflammatory cascade associated with coronavirus infection as well as the serious and potentially life-threatening CAR-T therapy-related side effects while preserving and potentially improving the efficacy of the CAR-T therapy itself, thereby breaking the efficacy/toxicity linkage. The companys immediate focus is to prevent or minimize the cytokine storm that precedes severe lung dysfunction and ARDS in serious cases of SARS-CoV-2 infection and also in combining FDA-approved and development stage CAR-T therapies with lenzilumab, the companys proprietary Humaneered anti-human-GM-CSF immunotherapy, which is its lead product candidate. A potential registrational Phase III study in COVID-19 patients is currently enrolling. The company is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T- cell engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat graft-versus-host disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). For more information, visit http://www.humanigen.com

Forward-Looking Statements

This release contains forward-looking statements. Forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct and you should be aware that actual events or results may differ materially from those contained in the forward-looking statements. Words such as "will," "expect," "intend," "plan," "potential," "possible," "goals," "accelerate," "continue," and similar expressions identify forward-looking statements, including, without limitation, statements regarding our expectations for the Phase III study and the potential future development of lenzilumab to minimize or reduce the severity of lung dysfunction associated with severe and critical COVID-19 infections or to be approved by FDA for such use or to help CAR-T reach its full potential or to deliver benefit in preventing GvHD. Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to, the risks inherent in our lack of profitability and potential need for additional capital to conduct the Phase III study and grow our business; our dependence on partners to further the development of our product candidates; the uncertainties inherent in the development and launch of any new pharmaceutical product; the outcome of pending or future litigation; and the various risks and uncertainties described in the "Risk Factors" sections and elsewhere in the Company's periodic and other filings with the Securities and Exchange Commission.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You should not place undue reliance on any forward-looking statements, which speak only as of the date of this release. We undertake no obligation to revise or update any forward-looking statements made in this press release to reflect events or circumstances after the date hereof or to reflect new information or the occurrence of unanticipated events, except as required by law.

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Mayo Clinic Study of Humanigen's Lenzilumab Shows Rapid Recovery and Discharge in Severe and Critical COVID-19 Patients - Business Wire

Cirmtuzumab Plus Ibrutinib Shows Activity and Tolerability in MCL and CLL – OncLive

The combination of the first-in-class humanized monoclonal antibody cirmtuzumab and the BTK inhibitor ibrutinib (Imbruvica) was found to be an active, well-tolerated regimen for patients with relapsed/refractory mantle cell lymphoma (MCL) and treatment-nave or relapsed/refractory chronic lymphocytic leukemia (CLL), according to data from a phase 1b/2 study (NCT03088878).

The trial enrolled patients with relapsed/refractory MCL, or relapsed/refractory or treatment-nave CLL or small lymphocytic lymphoma (SLL) who had measurable disease and had either limited or no prior treatment with BTK inhibitors. The trial is comprised of 3 parts: a phase 1 dose-escalation phase with cirmtuzumab (part 1), an expansion cohort (part 2), and phase 2 randomization of the cirmtuzumab/ibrutinib combination versus ibrutinib alone (part 3).

As of January 29, 2020, 12 patients with relapsed/refractory MCL were enrolled onto part 1 of the trial. All patients had stage III/IV disease at diagnosis and 25% had bulky tumor at the time of study entry. Fifty-eight percent of these patients had Mantle Cell Lymphoma International Prognostic Index risk scores of intermediate or high, and 83% had received 2 prior regimens. Additionally, a total of 34 patients with CLL were enrolled onto either part 1 of the trial (n = 18) or the part 2 expansion phase (n = 16).

Results presented during the 2020 ASCO Virtual Scientific Program by lead author HunJuLee, MD, showed a high tumor response rate with the combination in those with MCL, with an objective response rate (ORR) of 83% and a complete response (CR) rate of 58%. The initial median progression-free survival with the regimen was 17.5 months. Those who had received 1 to 10 months of prior ibrutinib also responded well to the combination; 2 patients achieved CRs and 2 achieved partial responses (PRs).

In patients with CLL/SLL, treatment with the combination led to an overall best response rate of 88% and a clinical benefit rate of 100%. One patient experienced a CR and has since remained in remission for over 8 months and off all CLL treatment. Three other patients achieved PRs with the regimen.

With regard to safety, the most common adverse effects (AEs) potentially related to the combination were fatigue, diarrhea, and contusion. One patient with MCL and 8 patients with CLL reported treatment-related severe AEs. However, the events were thought to be related to ibrutinib or the combination rather than cirmtuzumab alone. Grade 3 or higher AEs included atrial fibrillation (n = 5), pneumonia (n = 3), pericardial hemorrhage, pleural effusion, pyrexia, hyperkalemia, gastrointestinal hemorrhage, and staph infection (n = 1, each).

In terms of medium follow up, it's very short so I don't want to get too excited. The median follow-up was 8 months and the median PFS is 17 months, said Lee. We are very happy with the results we have, and we look forward to building on top of this. There are ways to get rid of MCL but some of the ways that you take have a lot of toxicities involved. It is really exciting that all of my patients did not have any toxicity.

In an interview with OncLive, Lee, an assistant professor of medicine in the Department of Lymphoma & Myeloma and the Jessica and Jeffrey Brue Endowed Professor of Lymphoma Research at The University of Texas MD Anderson Cancer Center, further discussed the interim results of the study examining cirmtuzumab plus ibrutinib and the next steps with this research in MCL and CLL/SLL.

OncLive: Could you provide some background oncirmtuzumaband the rationale to explore it in combination withibrutinibin this setting?

Lee: As you know,the holy grail of cancer therapy is to find a target on the cancer cell that is not expressed on normal cells. Ideally, with lymphomas we're looking for a target on the lymphoma cell that is different from the proteins expressed on the surface of normal B cells. We would like to treat the lymphoma without harming normal B cells. Preclinical studies have examined ROR1, a family of proteins that are expressed in embryonic protein during early life and [then] disappear. [Investigators have] found [that these proteins] were expressed on the lymphoma cells and leukemia cells.[Investigators thought that] if [ROR1] was expressed on malignant lymphoma and leukemia cells and not expressed on normal cells, then it would serve as a great target [for therapy].

Rituximab (Rituxan)was developed in the early 90s and it targets CD20, which is expressed on normal cells. As such, when you give treatments like rituximab, even though the agent is well-tolerated, it does have some collateral damage. Rituximab decreases lymphocyte counts and we do have patients who require intravenous immunoglobulin therapyfollowing prolonged rituximab maintenance [if they have] lowlymphocyte counts.When they observed this finding, investigators questioned whether this is something that can be targetable. However, before we can develop a treatment [we have to understand] what it does.

What they found was that it is 1 of the proliferative survival signals, that are complementary to another system of proteins called immunoglobulin and that is the B-cell receptor. We know that B-cell receptor targeting has been tremendous with the targeting of BTK with ibrutinib and idelalisib (Zydelig). In all these pathways, targeting has generated great treatment options for many of our patients. However, we know that the difficulty withibrutinib, idelalisib, the PI3K inhibitors, and BTK inhibitors, is that they don't [lead to an] 100% complete remission.

Where is it? How are these B cells surviving? What about the patients who are primary refractory? What about the patients who respond initially and then relapse? Where are these cells coming from and where's the survival? We're almost trying to play chess with these lymphoma cells because we know that they're using biological mechanisms to achieve a survival advantage.

[When the BTK is being blocked, these lymphoma cells] reroute the survival signal through another survival channel. We believe one of the mechanisms that this drug works by, is by blocking the ROR1, which is one of the other survival signals. When we gave ibrutinib in preclinical studies, [it was found to] increase the signaling pathway in the ROR1 pathway. If you block the BTK, the cells find an alternative route. The lymphoma and leukemia cells are very smart. They are able to take advantage of alternate sources of growth signals.That's where the idea came in, combined with the results from preclinical studies demonstrating that patients who had high levels of ROR1 had lower survival.

I'm not saying this is the only way that the lymphoma cells escape mechanism. We know that there's mutations in the BTK and so forth and so on. However, this may be one of the contributing factors that help these lymphoma and leukemia cells become resistant to BTK inhibition. Therefore, we feel that blocking this pathway will block both pathways, thus, leading to a deeper response, meaning more CRs.

Could you discuss the design of the trial and the dosing schedules that were investigated?

This was designed as an open-label, phase 1/2 study. We knew that BTK inhibition and the ROR1 works in a complimentary fashion. We already knew the dosing schedule for ibrutinib, but we did multiple-dose escalations to try to find the right recommended dose [forcirmtuzumab] in the phase 2 [portion of the] study. We had 2 mg, 4 mg, 8 mg, 16 mg, 3 kg, and a flat dosing of 300 mg and 600 mg. Ultimately, after the phase 1 study, went with the 600 mg flat dosing, and that was found to be very well tolerated.

This goes right into the safety issue. We're looking for any toxicity. One of the remarkable things about this protein is that many of the antibody-driven therapies have been very well tolerated, andcirmtuzumabhas also been very well tolerated. We did not see any grade 3 toxicity forcirmtuzumab.

Do anti-ROR1 antibodies have an established toxicity profile or is it still under investigation?

That is still under investigation, but in combination with the ibrutinib, we did not have any signal in terms of dose-limiting toxicities with the doses that we gave. Here, at The University of Texas MD Anderson Cancer Center, we gave the 2 mg through 18 mg per kilograms and the high and low flat dosing, and it was found to be very tolerated.

What did we see with efficacy?

The efficacy seen with the combination was very interesting. Two histologies were studied: MCL and CLL. I am leading the MCL side and Michael Choi, MD, of the University of California, San Diego Medical Center, is leading the CLL portion of this study.

The MCL portion showed tremendous, exciting signal on phase 1 and 2 of this study. The ORRs were north of 80%, and the real kicker, knock-your-socks-off data are the CR rates. We were able to get 58% CR rates for many of these very heavily pretreated patients. We had approximately 40% of patients who had undergone autologous stem cell transplantation; we have patients who failed ibrutinib; and we have patients who had received [prior] CAR T-cell therapy and responded. Those findings gets people excited. I was surprised at how good the response was, given that usually toxicity and response go hand in hand. When you get more responses, you usually have very high toxicity. However, we really did not see any grade 3 toxicities.

Tons of CAR T-cells are being developed, but they come with fairly heavy prices. The headlines are big, but I know behind [the scenes], because I take care of these patients. It's not easy; if a patient is 70 years of age, theyre barely getting into the clinic. [When they ask for a CAR T], I get nervous; it is tough. But with this [combination], the majority of my patients [did not even feel that they were on a treatment]. This is very exciting for our patients.

The ORRs were north of 80%, and there was a 58% CR rate. We have 12 patients treated. Part 2 is open and it's accruing very nicely so we're trying to get more patients to enroll. This space is very tight; I'm sure you're aware that MCL is a tough place now because of the success achieved by Michael Wang, MD, of the University of Texas MD Anderson Cancer Center, with ibrutinib and CAR T. We're competing against some big agents, which are very effective. However, it's [a good thing] for our patients, because they have many treatment options that are available.

In terms of CLL, we did see high response rates; however, we did not see the robust CR rates that we saw in MCL; thats one thing to note. They're up to part 2 and that part of the trial is enrolling much faster than MCL. CLL is the most common lymphoid malignancy in North America so investigators are readily finding patients. We're hoping that the enrollment for the MCL portion can get going and will show robust data.

Is there anything that you would like to add?

There was a rapid attainment of CR rates observed. Many of these patients have had hyper-CVAD, autologous stem cell transplantation, and CAR T-cell therapies, among others. [These were not patients who had only received 1 line of prior] therapy. If you start using ibrutinib earlier in treatment, you get higher levels of CRs and higher levels of PFS. However, these are patients that are heavily pretreated with fairly aggressive histologies and behavior. If you look at the waterfall plot, you can clearly see a rapid drop in the tumor sizes of the patients with MCL.

Many more patients were included in the CLL [portion of the study] and they do have a response, but the majority of these responses were PR, so they were not able to attain CR. Although they do have a response, it is not as dramatic as the MCL population.

If you look at the PFS curves for MCL they look nice. It's a very limited population with very limited follow up, so this will need to be followed up for much longer.

This is going to be leading to a very exciting time forcirmtuzumab and ibrutinib as we enroll for part 2 [of the trial], which is going to be the efficacy signal that we will be seeing moving forward. Additionally, other agents are being developed for the ROR1 targets, but the efficacy that we saw in our study with our limited number of patients is very exciting.

Lee HJ, Choi MY, Siddiqi T, et al. Clinical activity of cirmtuzumab, an anti-ROR1 antibody, in combination with ibrutinib: interim results of a phase Ib/II study in mantle cell lymphoma (MCL) or chronic lymphocytic leukemia (CLL). J Clin Oncol.2020;38(suppl 15):8036. doi:10.1200/JCO.2020.38.15_suppl.8036

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Cirmtuzumab Plus Ibrutinib Shows Activity and Tolerability in MCL and CLL - OncLive

Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies…

89% of evaluable patients (17/19) with transfusion-dependent -thalassemia who do not have a 0/0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207

Data from exploratory analyses of HGB-207 show improved markers of blood cell production and bone marrow function in patients who achieved transfusion independence

85% of patients (11/13) with a 0/0 genotype or IVS-I-110 mutation in HGB-212 have been transfusion-free for at least 7 months

bluebird bio, Inc. (Nasdaq: BLUE) today announced that new data from ongoing Phase 3 studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent -thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal (10.5 g/dL). These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

"With more than a decade of clinical experience evaluating gene therapy in patients with transfusion dependent -thalassemia across a wide range of ages and genotypes, we have built the most comprehensive understanding of treatment outcomes in the field," said David Davidson, M.D., chief medical officer, bluebird bio. "Seeing patients achieve transfusion independence and maintain that positive clinical benefit over time with robust hemoglobin levels reflects our initial vision of the potential of beti-cel. The accumulating long-term data demonstrating improvements in bone marrow histology, iron balance and red cell biology support the potential of beti-cel to correct the underlying pathophysiology of transfusion-dependent -thalassemia."

A total of 60 pediatric, adolescent and adult patients across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies as of March 3, 2020. In studies of beti-cel, transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

TDT is a severe genetic disease caused by mutations in the -globin gene that results in significantly reduced or absent adult hemoglobin (HbA). In order to survive, people with TDT maintain Hb levels through lifelong, chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

"Patients with transfusion-dependent -thalassemia do not make enough healthy red blood cells and cannot live without chronic transfusions; for patients that means a lifetime of necessary visits to a hospital or clinic and reliance on an often unreliable blood supply, which compounds the challenges of managing this disease," said presenting study author Professor John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK. "These results showing patients free from transfusions and maintaining near-normal hemoglobin levels after treatment with beti-cel is a positive outcome for people living with transfusion-dependent -thalassemia. In addition, we now have more data that provide further evidence that most of these patients have a measurable improvement in markers of healthy red blood cell production."

Beti-cel is a one-time gene therapy designed to address the underlying genetic cause of TDT by adding functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). This means there is no need for donor HSCs from another person, as is required for allogeneic HSC transplantation (allo-HSCT). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived Hb, at levels that eliminate or significantly reduce the need for transfusions.

As of March 3, 2020, all 23 patients in HGB-207 were treated and have been followed for a median of 19.4 months. These patients ranged in age from four to 34 years, including eight pediatric (<12 years of age) and 15 adolescent/adult (>12 years of age) patients. Only 19 patients were evaluable for transfusion independence; four additional patients do not yet have sufficient follow-up to be assessed for transfusion independence.

Eighty-nine percent of evaluable patients (17/19) achieved transfusion independence, with median weighted average total Hb levels of 11.9 g/dL (min-max: 9.4 12.9 g/dL) over a median of 19.4 months of follow-up to date (min-max: 12.3 31.4 months). These 17 patients previously required a median of 17.5 transfusions per year (min-max: 11.5 37 transfusions per year).

Improved iron levels, as measured by serum ferritin and hepcidin levels (proteins involved in iron storage and homeostasis), were observed and trends toward improved iron management were seen. Over half of patients stopped chelation therapy, which is needed to reduce excess iron caused by chronic blood transfusions. Seven out of 23 patients began using phlebotomy for iron reduction.

Analysis of Healthy Red Blood Cell Production

In exploratory analyses, biomarkers of ineffective erythropoiesis (red blood cell production) were evaluated in patients who achieved transfusion independence in HGB-207.

The myeloid to erythroid (M:E) ratio in bone marrow from patients who achieved transfusion independence increased from a median of 1:3 (n=17) at baseline to 1:1.2 (n=16) at Month 12. Improvement of the M:E ratio, the ratio of white blood cell and red blood cell precursors in the bone marrow, suggests an improvement in mature red blood cell production. Images illustrating the bone marrow cellularity at baseline, Month 12 and Month 24 are available in the EHA25 presentation (abstract #S296): "Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study".

Additionally, biomarkers of erythropoiesis continue to demonstrate a trend toward normalization in patients who achieved transfusion independence, including improved levels over time of erythropoietin, a hormone involved in red blood cell production; reticulocytes, immature red blood cells; and soluble transferrin receptor, a protein measured to help evaluate iron status. The continued normalization of red blood cell production over time among some patients who achieved transfusion independence supports the disease-modifying potential of beti-cel in patients with TDT.

Northstar-3 (HGB-212) Efficacy

As of March 3, 2020, 15 patients (genotypes: 9 0/0, 3 0/ +IVS1-110, 3 homozygous IVS-1-110 mutation) were treated and had a median follow-up of 14.4 months (min-max: 1.124.0 months). Median age at enrollment was 15 (min-max: 4 33 years).

Six of eight evaluable patients achieved transfusion independence, with median weighted average total Hb levels of 11.5 g/dL (min-max: 9.5 13.5 g/dL), and continued to maintain transfusion independence for a median duration of 13.6 months (min-max: 12.2 21.2 months) as of the data cutoff.

Eighty-five percent of patients (11/13) with at least seven months of follow-up had not received a transfusion in more than seven months at time of data cutoff. These 11 patients previously required a median of 18.5 transfusions per year (min-max: 11.0 39.5 transfusions per year). In these patients, gene therapy-derived HbAT87Q supported total Hb levels ranging from 8.814.0 g/dL at last visit.

Betibeglogene autotemcel Safety

Non-serious adverse events (AEs) observed during the HGB-207 and HGB-212 trials that were considered related or possibly related to beti-cel were tachycardia, abdominal pain, pain in extremities, leukopenia, neutropenia and thrombocytopenia. One serious event of thrombocytopenia was considered possibly related to beti-cel.

In HGB-207, serious events post-infusion in two patients included three events of veno-occlusive liver disease and two events of thrombocytopenia. In HGB-212, serious events post-infusion in two patients included two events of pyrexia.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

In both Phase 3 studies, there have been no deaths, no graft failure, no cases of vector-mediated replication competent lentivirus or clonal dominance, no leukemia and no lymphoma.

The presentations are now available on demand on the EHA25 website:

About betibeglogene autotemcel

The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel (beti-cel; formerly LentiGlobin gene therapy for -thalassemia), marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO, including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Beti-cel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity and non-cardiac chest pain. Two serious adverse events (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration (FDA) granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of transfusion-dependent -thalassemia. Beti-cel is not approved in the U.S.

Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207) and NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel or LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

bluebird bio Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the COVID-19 pandemic and resulting impact on our operations and healthcare systems will affect the execution of our development plans or the conduct of our clinical studies; the risk that the efficacy and safety results observed in the patients treated in our prior and ongoing clinical trials of beti-cel may not persist; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated with additional patients in our ongoing or planned clinical trials or in the commercial context; the risk that the FDA will require additional information regarding beti-cel, resulting in a delay to our anticipated timelines for regulatory submissions, including submission of our BLA. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200612005084/en/

Contacts

Media:Catherine Falcetti, 339-499-9436cfalcetti@bluebirdbio.com

Investors:Ingrid Goldberg, 410-960-5022igoldberg@bluebirdbio.com

Elizabeth Pingpank, 617-914-8736epingpank@bluebirdbio.com

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Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies...

Cord Blood Banking Leader Cryo-Cell Enters into Patent Option Agreement with Duke University – Global Banking And Finance Review

OLDSMAR, Fla., June 11, 2020 Cryo-Cell International, Inc. (OTC:QB Markets Group Symbol: CCEL) (the Company), the worlds first private cord blood bank to separate and store stem cells in 1992, announced that effective June 9, 2020, the Company has entered into a patent option agreement with Duke University. The six-month exclusive option agreement gives Cryo-Cell an option to obtain a license to manufacture and sell products based on Dr. Joanne Kurtzbergs patents. Please see the Companys Form 8-K filed with the Securities and Exchange Commission on June 11, 2020 for more details.

David Portnoy, Chairman of the Board and Co-CEO, said, Cryo-Cell is honored to have entered into this option agreement with Dr. Kurtzberg and Duke University and believes that this is a significant step in the transformation of the Company. We look forward to Duke becoming a major shareholder and partner of the Company for many years to come.

We are excited to enter into this exclusive option agreement with Cryo-Cell and are looking forward to working together to bring novel cord blood and birthing tissue based cellular therapeutics to the clinic, said Joanne Kurtzberg, MD, who is the Jerome S. Harris distinguished professor of pediatrics and a pioneer in cell therapies based on umbilical cord blood. Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine.

About Cryo-Cell International, Inc.

Founded in 1989, Cryo-Cell International, Inc. is the worlds first private cord blood bank. More than 500,000 parents from 87 countries have entrusted Cryo-Cell International with their babys cord blood and cord tissue stem cells. In addition to its family bank, Cryo-Cell International has a public banking program in partnership with Duke University. Cryo-Cells public bank has provided cord blood for more than 600 transplantations and operates cord blood donation sites across the U.S in prominent hospitals such as CedarsSinai Hospital in Los Angeles and Baptist Hospital in Miami. Cryo-Cells mission is to provide clients with state-of-the-art cord blood and cord tissue cryopreservation services, raise awareness of the opportunity for expectant parents to bank or donate their babys cord blood and support the advancement of regenerative medicine. Cryo-Cell operates in a facility that is FDA registered, cGMP-/cGTP-compliant and licensed in all states requiring licensure. Besides being AABB accredited as a cord blood facility, Cryo-Cell was also the first U.S. (for private use only) cord blood bank to receive FACT accreditation for adhering to the most stringent cord blood quality standards set by any internationally recognized, independent accrediting organization. In addition, Cryo-Cell is ISO 13485:2003certified by TV, an internationally recognized, quality assessment organization. Cryo-Cell is a publicly traded company, OTCQB:CCEL. For more information, please visit http://www.cryo-cell.com.

Forward-Looking Statement

Statements herein the terms believes, intends, projects, anticipates, expects, and similar expressions as used are intended to reflect forward-looking statements of the Company. The information contained herein is subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated in such forward-looking statements or paragraphs, many of which are outside the control of the Company. These uncertainties and other factors include the impact of the COVID-19 pandemic on our sales, operations and supply chain, the success of the Companys global expansion initiatives and product diversification, the Companys actual future ownership stake in future therapies emerging from its collaborative research partnerships, the success related to its IP portfolio, the Companys ability to enter into a definitive license agreement with Duke, the Companys future competitive position in stem cell innovation, future success of its core business and the competitive impact of public cord blood banking on the Companys business, the Companys ability to minimize future costs to the Company related to R&D initiatives and collaborations and the success of such initiatives and collaborations, the success and enforceability of the Companys menstrual stem cell technology license agreements and umbilical cord blood license agreements and their ability to provide the Company with royalty fees, the ability of the reproductive tissue storage to generate new revenues for the Company and those risks and uncertainties contained in risk factors described in documents the Company files from time to time with the Securities and Exchange Commission, including the most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and any Current Reports on Form 8-K filed by the Company. The Company disclaims any obligations to subsequently revise any forward-looking statements to reflect events or circumstances after the date of such statements.

Contact: David Portnoy, Chairman and Co-CEO Cryo-Cell International, Inc. 813-749-2100 [emailprotected]

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Cord Blood Banking Leader Cryo-Cell Enters into Patent Option Agreement with Duke University - Global Banking And Finance Review

Judge Says FDA Can Stop Clinic from Selling Stem Cell …

In 2015, a stem cell clinic in Florida conducted a procedure on three women to treat their macular degeneration. Instead, it left each of them with severe vision loss. The tragedy has been held up as an example of the lack of regulatory oversight the US government has had over such outfits that offer unproven stem cell treatmentsand now, its an example of how that is changing.

On June 3, a federal judge ruled that the US Food and Drug Administration (FDA) is entitled to a permanent injunction against US Stem Cell, forcing the company to stop conducting procedures using a particular technique that involves isolating stem cells from clients fat.

The FDA also filed a suit against a California-based company Cell Surgical Network, which provides similar interventions, that is still pending in court.

Precedent from cases like this helps the FDA in future enforcement actions.

Stephanie Caccomo, FDA

The lawsuit itself wasnt surprising. The allegations werent surprising. And the judges conclusion wasnt very surprising, Andrew Ittleman, an attorney at Miami-based Fuerst, Ittleman, David & Joseph, a law firm that counts government compliance for stem cell and regenerative medicine companies as one of its key practice areas, tells The Scientist. If anything, people were wondering why it took so long.

Hundreds of stem cell clinics have popped up across the US and other countries in recent years, making promises with little evidence that their treatments can cure ailments that traditional medicine cannot. The clinics have often avoided FDA oversight by claiming that their procedures, which often use a patients own cells, are not subject to FDA regulations.

The agency has been cracking down on the industry, but it has only successfully obtained a judgment against a stem cell clinic once before. This latest ruling by Judge Ursula Ungaro of the United States District Court for the Southern District of Florida may represent a sea change in regulatory enforcement, and possibly open the door for the FDA to file suits against companies violating FDA guidelines for marketing stem cell treatments en masse, according to Ittleman.

This is a landmark decision because this is only the second time the FDA has obtained a judgment against a stem cell clinic, and the first judgment since FDA announced in 2017 the agencys risk-based enforcement priorities for regenerative medicine, FDA spokesperson Stephanie Caccomo tells The Scientist in an email.

Research on stem cell therapies has ballooned in recent years, and some procedures for certain blood disorders have even been FDA-approved, but most remain unproven as far as the FDA is concerned. Extracting fat cells using liposuction, processing them to extract stem cells (known as stromal vascular fraction cells or SVF), and injecting them into other areas of the body the strategy US Stem Cell useshas been an FDA target before. Some clinics provide treatments with stem cells derived from bone marrow, cord blood, or birth tissue.

Ittleman, who has represented clients sued by the FDA, doesnt believe the ruling will immediately affect clinics using other types of stem cells. The fat [derived stem cell treatment] has been really the one place where the FDA has been very clear for very long about its position. We dont necessarily have that clarity in other areas, he says. The ruling may inspire the FDA to target other unapproved stem cell treatments with litigation, he adds.

The three patients who lost all or most of their sight were the first (and only) three participants in a discontinued clinical trial US Stem Cell was running on the procedure. Afterward, the patients saw university-based ophthalmologists for treatment, and those doctors published a report in March of 2017 in the New England Journal of Medicine detailing the adverse effects on each individual and raising concern about stem cell clinics.

US Stem Cell failed to follow best practice in ophthalmology of operating on one eye first, and returning later for a second surgery on the remaining eye. This way, if there is an adverse reaction, the patient can still see with the untreated eye. But the company conducted both procedures simultaneously.

Shortly after the failed procedures, two of the patients settled lawsuits with US Stem Cell, but the company faced few other penalties. While it stopped offering fat-derived stem cell treatments for macular degeneration, it continued to provide services using SVF that it claimed could treat myriad ailments, from Parkinsons disease to chronic obstructive pulmonary disease (COPD).

The FDA sent a warning letter to US Stem Cell in August 2017 about marketing the unapproved products and violations to good manufacturing practices. But the company did not comply. Ittleman says they were really sticking their fingers in the FDAs eyes over the course of time saying, You dont regulate us.

In a written statement sent to The Scientist, US Stem Cell said, While we believe there is substantial evidence to prove the efficacy of this protocol, we must immediately comply with the court as we review the decision. A spokeswoman told TheNew York Times that the company plans to continue offering stem cell treatments derived from other tissue.

Precedent from cases like this helps the FDA in future enforcement actions, says Caccomo. The FDA will continue to take stepssuch as issuing warning letters or initiating court casesagainst clinics that abuse the trust of patients and endanger their health with inadequate manufacturing conditions or by manufacturing and promoting products in ways that make them drugs under the law, but which have not been proven to be safe or effective for any use.

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Judge Says FDA Can Stop Clinic from Selling Stem Cell ...

Saad Z. Usmani, on the Approval of Daratumumab and Hyaluronidase-fihj – Cancer Network

In an interview with CancerNetwork, Saad Z. Usmani, MD, FACP, hematologist and medical oncologist at Levine Cancer Institute, spoke about the recent approval of daratumumab and hyaluronidase-fihj (Darzalex Faspro) for use in adult patients with newly diagnosed or relapsed/refractory multiple myeloma.

The addition of this product now allows for subcutaneous dosing of daratumumab.

One of the concerns that medical oncologists have had is the long infusion time [of daratumumab], especially in the first cycle of treatment, said Usmani. The subcutaneous formulation of daratumumab brings a lot of convenience to the patients.

The combination of daratumumab and hyaluronidase-fihj is now approved for the following indications, of which intravenous daratumumab had already received approval:

[This] is going to be very important moving forward in the new COVID-19 pandemic environment where were trying to find innovative ways in which we can streamline the operations in the clinic, as well as make things more convenient for our patients and do it in a safe environment, Usmani said.

References:

FDA. FDA approves daratumumab and hyaluronidase-fihj for multiple myeloma. FDA website. Published May 1, 2020. fda.gov/drugs/drug-approvals-and-databases/fda-approves-daratumumab-and-hyaluronidase-fihj-multiple-myeloma. Accessed May 1, 2020.

This segment comes from the CancerNetworkportion of the MJH Life Sciences National Broadcast, airing daily on all MJH Life Sciences channels.

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Saad Z. Usmani, on the Approval of Daratumumab and Hyaluronidase-fihj - Cancer Network

R3 Stem Cell International Now Offering Enhanced Autism Stem Cell Program in Mexico – PR Web

Top Stem Cell Treatment for Autism in Mexico (888) 988-0515

SCOTTSDALE, Ariz. (PRWEB) May 18, 2020

R3 Stem Cell International is now offering an enhanced autism stem cell therapy program at its Tijuana regenerative clinic. There are two program options for patients and their families, which are all inclusive and represent extremely effective and safe cell counts.

While it is unclear exactly what causes Autism Spectrum Disorder (ASD), there are several recent clinical studies which have shown exciting outcomes for patients. Also, those studies have shown mesenchymal stem cell therapy for autism to be very safe as well.

R3 Stem Cell International's enhanced program provides umbilical cord derived stem cell therapy for patients, with total cell counts for the treatment ranging between 90 million up to 200 million live stem cells. The actual total administered depends on patient weight.

According to R3 International Medical Director Ramon De La Puerta, "We have seen exceptional outcomes for autism patients, which typically include increased cognitive abilities, interpersonal skills, less aggression and more. The biologics undergo quality assurance standards that exceed FDA regulations in the US, and no preservatives are used so cell viability is over 95%!"

The two all inclusive treatment options include either a five day stay, or several trips over a span of of a year. The all inclusive fee starts at $8975, and involves several IV therapies. Sedation is available with a highly qualified anesthesiologist if necessary, and parents are welcome to attend all treatment sessions.

As with all R3 Stem Cell International procedures, R3 takes care of concierge escort transportation from San Diego to the clinic, which is only 20 minutes from the SD International Airport. R3 will also help with travel logistics.

According to R3 CEO David Greene, MD, MBA, "Our enhanced Autism program offers an incredible opportunity for families desiring a safe, cost effective and clinically effective option for those who have not responded desirably with conventional treatments. Over 8 years of therapies, R3 has not seen any significant adverse events, and the patient satisfaction rate has been amazing!"

In order to find out if a person is a good candidate for stem cell therapy for Autism, R3 offers a free phone consultation for families simply by calling (888) 988-0515.

About R3 Stem Cell International: After 14,000 stem cell procedures in the US, R3 opened R3 Stem Cell International. With the first location in Tijuana, https://stemcelltreatmentclinic.com has been an incredible option for patients suffering from all types of chronic conditions such as kidney, liver, lung and heart issues. Additionally, neurologic issues respond well such as stroke, Alzheimer's, stem cells for diabetes and more.

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