New Viral Vector Improves Sickle Cell Gene Therapy – DocWire News

A unique, improved viral vector for use in treating sickle cell disease with gene therapy has recently been created by researchers from the National Institutes of Health (NIH). When tested in animal models, this vector was able to incorporate correct genes into bone marrow stem cells up to 10 times more efficiently than current vectors. This new viral vector also has a carrying capacity up to six times higher than the conventional vector, as per the researchers. The use of this new technique could potentially make gene therapy more effective and prevalent in treating sickle cell disease, which affects roughly 100,000 people in the US and millions globally. The NIH teams findings were published on October 2 in the journal Nature Communications.

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, said senior author John Tisdale, MD, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder.

In gene therapy, the viral vector serves as a delivery vehicle that leverages the viruss innate ability to infiltrate host cells and administer genetic material. The vector is modified to carry a beneficial gene that will induce therapeutic effects in the patient, often by counteracting a genetic mutation.

Patients with sickle cell disease have an inherited mutation in the beta-globin gene, resulting in a faulty hemoglobin structure that yields sickle-shaped red blood cells. This shape causes the blood cells to stick to blood vessel walls, causing pain, anemia, blockage, organ damage, and premature death. Gene therapy for sickle cell involves the modification of bone marrow hematopoietic stem cells. These blood-producing cells are altered to possess a normal copy of the beta-globin gene in a lab and are then reinfused into the patient, ultimately inducing the production of healthy red blood cells.

Though this approach has been effective, Tisdale notes that there is always room for improvement in such treatments. He compares this new viral vector to a new model of a car that is easier and more scalable to produce.

Researchers have been developing these beta-globin vectors in a reverse structural orientation for over 30 years. This approach entails that the genes incorporated into the virus are translated from right to left by the enzymes, analogous to a sentence being read backward. This is done due to the sensitive expression of intron 2, a key molecular component of the vector that is required for high beta-globin expression. This intron gets excluded during the normal vector preparation process if it is not oriented in this reverse manner.

Gene therapy studies that incorporate these reverse-oriented vectors for sickle cell disease and beta-thalassemia, a similar inherited blood disease, have been encouraging thus far. The researchers note that this complicated gene translation process has made both the preparation of the vector and the gene-transfer efficiency more challenging, however.

Roughly 10 years ago, Tisdale worked with Naoya Uchida, MD, PhD, a staff scientist in his lab, to find an improved beta-globin delivery vehicle. They found a unique way to leave the intron 2 intact by creating a new forward-oriented beta-globin vector. Unlike the previously used, reverse-oriented vector, this novel vehicle is read left to right in typical fashion. Tisdale notes that this simplifies the translation of this genetic information into a tangible protein compound, like beta-globin.

These unique vectors were used in mouse and monkey models, with the results being compared to reverse-oriented vectors. The NIH team found that their vector delivered a viral load of up to six times more therapeutic beta-globin genes than the traditional vector and had four to ten times higher transduction efficiency (ability to incorporate therapeutic genes into repopulating bone marrow cells).

In addition, these new vectors remained in place four years after transplantation, supporting the longevity of this approach. This forward-oriented vector can also be produced in greater quantities than the traditional vector, cutting time and costs that come with industrial production.

Our lab has been working on improving beta-globin vectors for almost a decadeand finally decided to try something radically differentand it worked, Tisdale concluded. These findings bring us closer to a curative gene therapy approach for hemoglobin disorders.

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New Viral Vector Improves Sickle Cell Gene Therapy - DocWire News

AIDS Pathogens: How Do They Multiply In The Body? – Science Times

Staff ReporterOct 04, 2019 06:23 AM EDT

To improve the treatment of a disease, scientists must know its root. A European research team led by Prof. Christian Eggeling from the Friedrich Schiller University Jena, the University of Oxford, and the Leibniz Institute of Photonic Technology (Leibniz IPHT) has succeeded in using high-resolution imaging to make the spread of theHI virusbetween living cells visible by the millisecond. The team has also identified the molecules the virus needs to replicate itself. Using theSTEDsuper resolution fluorescence microscopy, the research team was able to provide proof of the lipid environment where the AIDS pathogen replicates itself.

"Thestudyprovides a method of investigation to prevent the multiplication of such HI virus in the body," Eggeling said. The results of their study were published in the Science Advances journal on October 2, 2019.

In the study, the researchers discussed how they focused on the slice through which the Human Immunodeficiency Virus (HIV) emerges after the cell has been infected. They looked into the plasma membrane of the cell from which it emerged. The protein Gag was used as a marker to coordinate the process involved in the maturation process of the virus.

"We have identified that the decisive process of replication of infected cells happen where the protein Gag accumulates," Christian Eggeling explains. Looking further into this site, the researchers were able to identify that the HI virus interacts with certain lipids. Although it has been identified before, the study provides proof that the interaction happens both in infected and living cells in the body.

"This new discovery allows us to put together an antiviral drug,"Eggelingnoted. Another crucial discovery is the identification of molecules that the HI virus needs to leave one cell to infect another. With technology, this process can be followed, allowing scientists to prevent the spread of the virus from happening.

The team is now looking at developing antibodies that attack these identified molecules to suppress the virus. "The team wants the antibodies to work its magic medically, but they also want to find out the biophysical interaction that happens to further enhance their efficiency," Eggeling added.

With the use of fluorescence microscopy as a tool, the team was able to follow the labeled molecules and tracked them in real time. It ledthe team to understanding how diseases develop in the molecular level. Eggeling is closely working with physicians and biologists to discuss how these methods can further be used for easy disease detection. More importantly, he aims to make the process more accurate to hopefully prevent the diseases from spreading.

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AIDS Pathogens: How Do They Multiply In The Body? - Science Times

Identification of more than 200K Cancer Neoantigens Could Lead to New Cancer Vaccines – Clinical OMICs News

Scientists at Arizona State Universitys Biodesign Institute now report on the identification of more than 200,000 cancer neoantigens, which could feasibly lead to the development of broad-spectrum cancer vaccines, as well as tumor type-specific treatments or patient-personalized vaccines. In a cancer cell, it turns out that all levels of information transfer from DNA to RNA to protein become more error-prone, said research lead Stephen Albert Johnston, Ph.D., center director and professor, Biodesign Center for Innovations in Medicine. We proposed that these mistakes made in cancer cells may also be the source to make a cancer vaccine.

Johnston and his team at the Biodesign Institute have spent more than a decade working towards the goal of developing a universal vaccine that can prevent cancer. They report on their latest studies in Scientific Reports, in a paper titled, RNA Transcription and Splicing Errors as a Source of Cancer Frameshift Neoantigens for Vaccines.

The success of checkpoint inhibitor therapy against cancer is largely attributed to activation of the patients immune response to tumor neoantigens that result from DNA mutations in the cancer cells, the authors explained. However, while checkpoint inhibitor immunotherapies are revolutionizing how we treat cancer, about 5080% of patients with even the most responsive tumor types wont respond well to treatment. A surprising finding in the analysis of these patients was that one of the best correlates of response has been the total number of neoantigens in the tumor, the team stated. The realization that these DNA mutations have such immunological importance has accelerated research efforts to develop personal cancer vaccines. Its a promising approach, but in reality, a major problem is that the majority of tumors will not have enough neoantigen-generating mutations to sustain development of a personalized vaccine.

With this in mind, the researchers set out to look for an alternative source of neoantigens that could possibly broaden the scope of neoantigen-based cancer vaccines. They were particularly interested in how disrupted RNA processes can lead to the production of frameshift (FS) mutated peptides, and the exposure of these peptides to the immune system. As they pointed out, In the process of becoming a tumor, not only does the DNA mutation rate increase with faster cell divisions, but also there is a disruption of basic cellular functions, including RNA transcription, splicing, and the quality control system on peptides.

They reasoned that frameshift variants produced by errors in RNA processing might be a source of cancer neoantigens, and they also assumed that there is a general increase in error rates in cancer cells. For the most part these errors can be managed and cleaned up by the cells own quality control machinery. However, as cancer progresses, these mutated peptides can build up and swamp the cells ability to deal with them, so aberrant proteins are then exposed and recognized by the immune system.

These overwhelm the quality control systems of a cell, producing mistakes in RNA and proteins that are released from the cancer cell, and the immune system can respond to, said Johnston.

To quickly identify frameshift and splicing mutations, Johnstons research team designed an array to detect all possible predicted frameshift peptides that any tumor cell could potentially produce. They custom-build this frameshift array, which ended up containing almost 400,000 frameshift peptides, and screened these against the blood samples of cancer patients (and healthy samples as a control) to look for antibodies against the peptides.

We analyzed the specific IgG reactivities to these FSPs in 64 noncancer control samples and a total of 85 cancers from five different late-stage cancer types with 17 samples each (LC: lung cancer, BC: breast cancer, GBM: glioblastoma, GC: gastric cancer, PC: pancreatic cancer) and 12 stage I pancreatic cancer samples, the authors noted.

This approach is less complex than extracting, purifying, and then sequencing tumor DNA, which is the typical starting point for the development of personal cancer vaccines. Personal cancer vaccines are complicated and expensive, said Johnston. Also, only about 40% of tumors have enough mutations in the DNA to make a vaccine from. We discovered that even cold tumors at the DNA level make lots of mistakes at the RNA level. And the mistakes we focus on are frameshift peptides which are much more immunogenic than the point mutations used in personal cancer vaccines. Most importantly, we can make off-the shelf vaccines for therapeutic or even preventative vaccines which will be much less expensive.

The results of their screens indicated that all five cancer types, with the exception of glioblastoma, had significantly more peptides reacting with antibodies in the cancer patients than controls. There were also three basic patterns seen among patients with each cancer type. First, the vast majority of the frameshift peptides (6980%) were personal, or unique to that individual. Second, about 16% to 19% of the positive peptides were shared between two samples within the same cancer type, and thirdly, 1.56.9% were shared between three or more samples (with gastric cancer having the highest, at 6.9%). Strikingly, one of the hardest to treat cancers, glioblastoma, had the greatest potential for personalized vaccines. Of the 17 glioblastoma patient samples studied, each patient had 5,800 frameshift peptides, and of these, 4,500 were unique to that patient.

The team wanted to see how the frameshift mutations compared between early- and late-stage cancers. A comparison of the 20,000 peptides that they identified in late-stage and stage 1 pancreatic cancer showed little overlap, implying that a vaccine for early-stage cancer would have to be different to that for late-stage cancer.

Interestingly, studies in mouse models found that the newly discovered antigens were protective against both breast cancer and melanoma. Johnstons group has pioneered genetic immunization using gene gun technology, which they used for their experiments to shoot gold nanoparticles containing the most promising vaccines. In a typical experiment, six-week-old mice received one genetic immunization in the pinna of the ear. After four weeks, they were challenged with cancer-causing cells, and then twice received booster shots, two days apart. The results showed that the prototype vaccines could all significantly delay or even prevent tumor growth or progression. Most importantly, in the mouse vaccine challenges, they found that pooling multiple frameshift peptides resulted in a more effective vaccine, with additive effects further delaying tumor growth.

The team has used their work to date as the basis for a large preclinical trial, in dogs, funded by the Open Philanthropy Project, which is evaluating a vaccine candidate designed to be a broadly protective, prophylactic pan-cancer vaccine. Johnston has also established a spinout company, Calviri, to continue cancer vaccine development.

From their screening results and analyses of the different cancer samples, and the mouse cancer vaccine challenges, the Johnston group now has a top 100 peptide list for each of the five human cancers. We strongly believe that the data presented, as well as more to be submitted, support bringing FS antigen cancer vaccines to clinical trial, the authors wrote. We have recently initiated a large dog clinical trial of a pan-cancer prophylactic vaccine and will soon submit protocols for both dog and human therapeutic trials of cancer-type specific vaccines.

Johnston acknowledged that even optimistically it would be five to 10 years before human use. However, he concluded, This is probably the only approach to a broadly preventative cancer vaccine, so we feel we have to try it. The implications of success would be quite largefor dogs and people.

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Identification of more than 200K Cancer Neoantigens Could Lead to New Cancer Vaccines - Clinical OMICs News

XIST-Promoter Demethylation as Tissue Biomarker for Testicular Germ Cell Tumors and Spermatogenesis Quality – Beyond the Abstract – UroToday

One of the most remarkable characteristics of germ cell tumors is that they are developmental cancers, meaning that they closely resemble phenomena that occur during embryonic and germ cell development. It is only natural, then, that comprehensive knowledge about developmental biology drives the process of uncovering relevant disease biomarkers with a high likelihood of actual clinical use. This was the case for the classical serum markers AFP and HCG (secreted during embryogenesis), pluripotency factors (such as OCT3/4 and SOX2/17) and embryonic microRNAs (miR-371a-3p), which proved to be true biomarkers of germ cell neoplasms.

Pursuing this strategy, the evolutionary well-known event of X-chromosome inactivation in mammalian cells also resulted in another putative biomarker: XIST. This long non-coding RNA inactivates extra X-chromosome material in female cells, a process that is retained in testicular germ cell tumors as they have a super numerical X-chromosome constitution (because of the initial polyploidization step). Hence, in this work we explored and validated a demethylated XIST fragment (i.e., related to expression of the gene) as a biomarker of these tumors. Two different quantitative methodologies were applied, both with high sensitivity, including high-resolution melting analyses. Importantly, this biomarker was particularly useful in Seminomas, for which informative serum markers commonly used in the clinic are often detected in the normal range. Therefore, the demethylated XIST fragment in serum/plasma could be a promising biomarker for the clinical management of these patients.

Besides applications in the germ cell tumor field, we also demonstrated a valuable use of the demethylated XIST fragment for assessing spermatogenesis extent in testicular parenchyma samples. XIST has been shown to be only and specifically expressed in males during spermatogenesis when the germ cells enter meiosis. This is in line with the higher amount of demethylated XIST promoter identified by us in this study, being of relevance because infertility is a frequent side effect from cancer treatments, with a severe impact on cancer survivors quality of life. This novel finding may help to overcome the limitations of the time consuming and often inaccurate Johnsens score as evaluated by Pathologists to estimate spermatogenesis efficiency in clinical practice. Accordingly, we are pursuing an evaluation of our findings in seminal plasma samples.

Written by:Joo Lobo, MD, Resident in Pathology, PhD student, Cancer Biology & Epigenetics Group, Research Center,Portuguese Oncology Institute of Porto (IPO-Porto) & PhD student in Molecular Pathology and Genetics, Lecturer in Pathology, Master Degree in Medicine,Biomedical Sciences Institute Abel Salazar, University of Porto (ICBAS-UP) & PhD student, Looijenga Group,Princess Mxima Center (PMC) for Pediatric Oncology, Utrecht.

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XIST-Promoter Demethylation as Tissue Biomarker for Testicular Germ Cell Tumors and Spermatogenesis Quality - Beyond the Abstract - UroToday

Perioperative Targeted Therapy Or Immunotherapy In Non-Small-Cell Lung | OTT – Dove Medical Press

Huanlan Sa,1,* Peng Song,2,* Kewei Ma,3 Yong Gao,1 Li Zhang,2 Deqiang Wang1

1Department of Pain Management, Affiliated Hospital of Binzhou Medical University, Binzhou 256603, Peoples Republic of China; 2Department of Respiratory Medicine, Peking Union Medical College Hospital, Chinese Academy of Medical Science & Peking Union Medical College, Beijing 100010, Peoples Republic of China; 3Department of Oncology, Cancer Center, The First Hospital of Jilin University, Changchun, 130021, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Li ZhangDepartment of Respiratory Medicine, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, Peoples Republic of ChinaTel +8618811630866Email zhanglipumch@aliyun.com

Deqiang WangDepartment of Pain Management, Affiliated Hospital of Binzhou Medical University, Binzhou 256603, Peoples Republic of ChinaTel +8615066918983Email wangdeqiangbz@163.com

Abstract: Targeted therapy and immunotherapy have changed the treatment modes for advanced non-small cell lung cancer (NSCLC), moving from second-line to first-line treatment and significantly extending patients survival. Surgery and chemoradiotherapy remain the main treatment options for patients with locally advanced lung cancer, but recurrence and metastasis still occur in some patients. The survival rates of conventional perioperative chemotherapy among NSCLC patients have increased by only 5%. Therefore, more studies have begun to explore targeted and immune neoadjuvant/adjuvant therapies in early-stage and locally advanced NSCLC, and the relevant clinical research data have shown good efficacy and safety profiles. This article summarizes several clinical studies of critical importance.

Keywords: non-small-cell lung cancer, targeted therapy, immunotherapy

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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2 Years Later, Retired Toms River Teacher Meets Bone Marrow Donor – wobm.com

It was more than two years when in a Hometown View segment I shared a story about Lael McGrath who I described at that time as a very active retired 2nd grade teacher from Toms River. Her story centered on learning in August of 2016 that she had acute myeloid leukemia with a recommendation that she undergo a bone marrow transplant as it was her best chance for a full recovery. She had that procedure done in December of 2016 and the segment I aired had to do with a thank you party she was hosting for the family and friends who made up her devoted support group. That party also served as the Be the Match swabbing event as McGrath sought those who might consider being a donor as clearly she knew the importance of that.

So lets move forward to last week when Lael emailed me to say that more than two years later she was going to meet her bone marrow donor in person. Actually, let me backtrack just a bit. After McGrath was diagnosed with AML she was referred to the Blood and Marrow Transplant Program at Rutgers Cancer Institute of New Jersey and RWJ University Hospital where they determined she was a candidate for a stem cell transplant. Unfortunately, none of her five siblings were a match so through the help of RWJ and the Be the Match donor registry the search begana successful one. As I said earlier McGrath had the surgery in December of 2016 and now almost three years later she is in remission and the grandmother of six has returned to that active lifestyle which includes running and yoga.

Now I can go on with the update. As in cases like this, the identity of the donor must remain anonymous for two years and after that period Lael learned that her lifesaver was 21-year old Wiebke Rudolph of Germany who placed herself on a donor registry at the age of 18. The two began communicating and last week just before McGraths 68th birthday they met face-to-face in what was an emotional get-together. Rudolph stayed at McGraths Toms River home and they were treated to a special reception last Friday at the hospital in New Brunswick which also included Laels doctors and other members of her care team.

Clearly Wiebke has now become an unofficial member of the McGrath family and this is one of those stories with a very happy ending. It also brings added attention to the importance of being a donor and for more you can visit http://www.bethematch.org.

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Man with terminal cancer battles back from coma to marry his childhood sweetheart – Mirror Online

A 26-year-old man told he had just 12 months to live pushed his wedding forward to marry the love of his life.

Dan Cassidy, from Accrington, was diagnosed with aggressive strain of leukaemia in March this year.

He had many things he wanted to do before he died, the the most important one was wedding his soul-mate Darcy Young.

The couple had been together 10 years and were able to push their wedding forward to tie the knot in July after they learned of Dan's tragic terminal cancer diagnosis.

"It was a perfect day," Dan told LancsLive .

"I wasn't feeling too good on the day, I don't know if that was the cancer or the nerves.

"I still had a great time but I couldn't be myself."

Dan was previously diagnosed with cancer in 2016 when he started to feel unwell while at a friend's barbeque.

He said: "I tried to stand up to go to the toilet and I just couldn't. I kept feeling a pain down my legs.

"My friend's wanted me to go out with them but once I was in the taxi I had to go back, I was in so much pain."

He continued: "We went to the hospital the next day and I was told I had leukaemia."

The doctor said that Dan had probably had the condition for two years and that the pain he was experiencing was from his white blood cells fighting aggressively against the cancer.

"He was in so much agony," said Darcy.

"He had dialysis to clean the blood but, whether it was the procedure or not, that caused him to have a bleed on the brain.

"He was transferred to Blackburn hospital and was put into an induced coma."

Things got worse for Dan as the doctors had to cease his cancer treatment while they dealt with the bleed.

But somehow he pulled through.

Following another bleed, brain surgery, strokes, rounds of chemotherapy and radiotherapy, Dan had a stem cell transplant from his mum and was discharged.

"Just before Christmas 2016, I came out of hospital," he said.

"Everything was working fine, I was feeling better and the cancer was in remission."

It was at this point that the 26-year-old decided that he wanted to ask Darcy a special question.

He said: "We have been together for 10 years, so we were due to get married.

"Obviously after I got out of hospital I just thought: life is so short."

Dan took Darcy to her grandparents' favourite place, Shireburn Arms, for a meal while her family decorated her room in secret.

He then gave her a box of chocolates with the words: 'Will you marry me?' iced onto them.

"He asked with chocolates," said Darcy.

"Thats how he asked me to be his girlfriend when I was 15."

The couple planned to marry in November 2019 and Dan continued to have regular check ups every three months.

In March this year Dan came out in a rash and was later told by a doctor that his cancer had returned.

"At the next appointment they said it had come back really aggressively," said Dan.

"They told me I had 12 months to live, that everyone that has had this before doesn't live past 12 months."

Darcy says the consultant was crying when she delivered the news to them.

"It didn't give us much hope," she said.

"She must have to deliver news like that all the time. I think I was in denial at first, I just thought: this can't be happening again."

Once Dan got his diagnosis the wedding became the main focus for the couple and they tied the knot in Clitheroe, in front of their family and friends.

Darcy said: "I didn't know if he would make it to the wedding but he did and it was so lovely.

"It was amazing to have all of our family and friends there to celebrate.

"All the venues and all the businesses we wanted to use made it happen for us - we couldn't ask for anything more."

The couple now live together with their dog Teddy, they are trying to enjoy the present day.

Darcy said: "We just want to seize the moment, every minute we can."

The couple want to share their story in the hope that more people will become aware of leukaemia and sign up to stem cell donor lists.

They continue to raise money for the charity Anthony Nolan who keep a register of stem cell donors.

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Man with terminal cancer battles back from coma to marry his childhood sweetheart - Mirror Online

Maryland Biotech Just Raised $85 Million to Advance its Intelligent Cell Therapy Platform – BioBuzz

October 3, 2019

Arcellx, a privately-held biopharmaceutical company, today announced that it has raised $85 million in an oversubscribed Series B financing. Proceeds will be used to advance the Companys ARC-T + sparX programs, including clinical development of a bivalent BCMA-targeted cell therapy in multiple myeloma, and a CD123-targeted therapy in acute myeloid leukemia. The Series B will also fund earlier stage ARC-T + sparX programs for patients with solid tumors and diseases outside oncology.

Arcellxwas recently featured as one of Five Companies That Are Changing the Landscape for Cell and Gene Therapy and is led by founder David Hilbert, PhD and a veteran team of top-caliber scientists and executives. Earlier this year Arcellx raised $27M in Series A funding from several high caliber investment firms that include NEA, Novo, Takeda Ventures and SR One.

According to the press release;Participants in the Series B include both existing and new investors to Arcellx. New investors Aju IB and Quan Capital co-led the round, followed by Mirae Asset Venture Investment, Mirae Asset Capital, LG Technology Ventures, JVC Investment Partners, and certain funds managed by Clough Capital Partners, L.P. Existing investors Novo Holdings, S.R. One Limited, NEA and Takeda Ventures also participated in the financing.

Concurrent with the financing, Hugo Beekman, Partner at Aju IB, and Lewis (Rusty) Williams, M.D., Ph.D., Venture Partner at Quan Capital, have joined the Arcellx board of directors.

The financial and strategic support from our investors allows Arcellx to accelerate development of a robust pipeline of ARC-T + sparX programs for patients in need, commented David Hilbert, Ph.D., President and Chief Executive Officer of Arcellx. As impressive as conventional CART therapies have been, their safety and efficacy profiles are challenged by severe toxicities, high rates of relapse, and challenging target selection in the solid tumor setting. The ARC-T + sparX platform addresses these concerns by placing ARC-T cells under the control of one or more sparX proteins that uniquely determine how the ARC-T cells recognize tumor, and the speed with which ARC-T cells kill tumor. In the coming months we will begin clinical testing of our lead BCMA-targeted therapy in multiple myeloma.

Rusty Williams, M.D., Ph.D., commented, Arcellx hasreached a positive inflectionin its novel platform and pipeline with the potential to improve efficacy and safety. We are excited to support the company as it advances new cell therapies with the potential to deliver better outcomes for patients.

Hugo Beekman, Partner at Aju IB, commented, Arcellx has invented a differentiated cell therapy platform with ARC-T + sparX that allows simultaneous and sequential targeting of multiple tumor antigens. The ability of sparX proteins to reprogram the specificity of ARC-T cells has the potential to address the high incidence of tumor relapse, as well as the inherent diversity of tumor antigens expressed within solid tumors. The features of this platform, along with scalable and efficient manufacturing processes, are intended to facilitate the Companys development of new therapies in oncology, and more broadly, in autoimmune disease and the transplant setting.

Their vision is to provide cancer patients with adaptive gene cell therapies that are readily silenced, activated, and reprogrammed in order to combat the complexity of human disease. Their technology platform aims to achieve improved efficacy through the reprogramming of the immune systems tumor-targeting receptors to address both newly diagnosed and relapsed cancers. Arcellxs technology will extend to solid tumors as well as autoimmune indications.

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Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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Maryland Biotech Just Raised $85 Million to Advance its Intelligent Cell Therapy Platform - BioBuzz

Regulator raids cell therapy makers too often to little avail – Korea Biomedical Review

In the wake of the recall of cell gene therapy Invossa-K, the Ministry of Food and Drug Safety conducted a special inspection of six cell therapy developers. The raid came less than a year since the regulator made an urgent inspection in October last year.

According to industry sources, the ministry started the inspection in early September to enhance the regular monitoring after the cancellation of the Invossa license.

In October last year, the ministry had a special inspection of four cell therapy makers, suspecting that the companies poorly managed allogeneic cell therapies.

Some observers questioned the effectiveness of the inspection because the raid last year could not detect any problem in Kolon Life Sciences management of cell therapy Invossa.

However, six months after the inspection, Kolon found that Invossa contained a cell ingredient different from the authorized label.

If another company has a similar problem, the ministry will find it difficult to identify it even if it carries out an inspection, observers said.

When the ministry had a pharmacist inspection last year, Kolon Life Science had no problem. The government must have been hard to know the mislabeling of Invossa because such pharmacist inspection was based on the data provided by the company, an official at a cell therapy developer said.

The Invossa recall has also toughened regulations on cell therapies. The food and drug safety ministry revised the Regulation on Approval Review for Biologic Products on Sept. 9 to make cell therapy developers mandatorily submit the results of Short Tandem Repeat (STR) testing when seeking approval for cell and gene therapies.

The new rules came after Kolon Life Science said the company belatedly found the mislabeling because it had never conducted STR testing before it applied for the Invossa license.

The government indeed tightened the control over cell therapies and related rules after the Invossa recall, an industry source said. The government is inspecting other companies belatedly, however, as it did so last year.

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Regulator raids cell therapy makers too often to little avail - Korea Biomedical Review

Global Personalized Cell Therapy Market Research Report (2019-2028) by Future Trend, Growth Rate, Opportunity, Industry Experts Analysis – Sound On…

New York City, NY: October 3, 2019 Published via (Wired Release) Global Personalized Cell Therapy Market Research Report is an in-depth and professional document 2019:

A newly published market study by MarketResearch.biz, titled Global Personalized Cell Therapy Market, is built up with a step by step analysis from expert research. The report provides accurate estimation, improvement criterias, action plans, and root ways. It has covered emerging market trends, key challenges, restraints, opportunities, future growth potentials, competitive outlook, and regional outlook, and value chain analysis. The top players/vendors of the global market are further covered in the report. The report presents a pin-point breakdown of Personalized Cell Therapy on the basis of type, applications, and research regions. The latest data has been presented on the revenue numbers, product details, and sale of key companies.

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It aims to help customers in the decision making process. The manufacturers data is covered that includes shipment, price, revenue, gross profit, and business distribution. With this report, all the manufacturers and the vendors will be in aware of the threats, shortcomings that the market will offer in the next few years. The current and prospective growth of the market for 2019-2028 is also captured. Graphical data is integrated in the form of charts, diagrams and tables making the report well organized and understandable for the professionals.

We Have includedVital insights into Personalized Cell Therapy Market Competition and StrategiesofCompetitors :

Cytori Therapeutics Inc, Bellicum Pharmaceuticals Inc, Saneron CCEL Therapeutics Inc, MolMed S.p.A., Vericel Corporation, Oxford Nanopore Technologies, Cell Medica, MediGene AG, TxCell

Outlook ofPersonalized Cell Therapy Market Segmentation:

Global personalized cell therapy market segmentation, by technique:

Platelet TransfusionsBone Marrow TransplantationPacked Red Cell TransfusionsOrgan TransplantationGlobal personalized cell therapy market segmentation, by therapeutic area:

Cardiovascular DiseasesNeurological DisordersInflammatory DiseasesDiabetesCancer

Regional Coverage:

All the regions and countries of the world are covered that also shows a regional development status, Personalized Cell Therapymarket size, volume, and value, as well as price data. The global demand for the Personalized Cell Therapy market has been fragmented across several regions such as

Americas (United States, Canada, Brazil, and Mexico)

Middle East and Africa (Egypt, South Africa, Israel, Turkey, GCC Countries)

Europe (Germany, France, UK, Italy, Russia Spain)

APAC (China, Japan, Korea, Southeast Asia, India, Australia)

South America (Brazil, Colombia, Argentina, etc.)

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Main Features of The GlobalPersonalized Cell TherapyMarket Research Report:

The report provides market values and anticipated growth rate of the global Personalized Cell Therapy market for all years till 2028.

The report highlights the actual drivers of the market by considering risks and identifying and testing new tactics, manufacturing cost, raw material cost, downstream buyers, labor cost, and market channels.

The report assessed the market segments and provides the relative contribution to the development of the global Personalized Cell Therapy market.

The report offers coverage of the competitive nature of the market and discusses various marketing strategies to stay ahead in the competition.

Its an essential tool to check the feasibility of a new project and geographical expansion of the company.

Browse Full Summary Research Report ofPersonalized Cell TherapyMarket:https://marketresearch.biz/report/personalized-cell-therapy-market/request-sample

There are 9 Chapters to deeply display the global Personalized Cell Therapy market

Chapter One: Global Personalized Cell Therapy Market Overview

1.1 Personalized Cell Therapy Preface

Chapter Two: Global Personalized Cell Therapy Market Analysis

2.1 Personalized Cell Therapy Report Description

2.1.1 Personalized Cell Therapy Market Definition and Scope

2.2 Personalized Cell Therapy Executive Summary

2.2.1 Personalized Cell Therapy Market Snapshot, [Segment 1]

2.2.2 Personalized Cell Therapy Market Snapshot, [Segment 2]

2.2.3 Personalized Cell Therapy Market Snapshot, [Segment 3]

2.2.4 Personalized Cell Therapy Market Snapshot, [Region Segment]

2.3 Personalized Cell Therapy Market Opportunity Analysis

Chapter Three: Global Personalized Cell Therapy Market Dynamics

3.1 Drivers

3.2 Restraints

3.3 Opportunities

3.4 Trends

Chapter Four: Global Personalized Cell Therapy Market Segment Analysis, by [Segment 1]

4.1 Personalized Cell Therapy Overview

4.2 Personalized Cell Therapy Segment Trends

4.3 Personalized Cell Therapy Market Share and Forecast, and Y-o-Y Growth

Chapter Five: Global Personalized Cell Therapy Market Segment Analysis, by [Segment 2]

5.1 Personalized Cell Therapy Overview

5.2 Personalized Cell Therapy Segment Trends

5.3 Personalized Cell Therapy Market Share and Forecast, and Y-o-Y Growth

Chapter Six: Global Personalized Cell Therapy Market Segment Analysis, by [Segment 3]

6.1 Personalized Cell Therapy Overview

6.2 Personalized Cell Therapy Segment Trends

6.3 Personalized Cell Therapy Market Share and Forecast, and Y-o-Y Growth

Chapter Seven: Global Personalized Cell Therapy Market Segment Analysis, by [Region Segment]

7.1 Personalized Cell Therapy Overview

7.2 Personalized Cell Therapy Regional Trends

7.3 Personalized Cell Therapy Market Share and Forecast, and Y-o-Y Growth

Chapter Eight: Global Personalized Cell Therapy Market Company Profiles

8.1 Companies

8.1.1 Company Overview

8.1.2 Product Portfolio

8.1.3 Financial Overview

8.1.4 Key Developments

8.1.5 SWOT Analysis

Chapter Nine: Global Personalized Cell Therapy Market

9.1 Research Methodology

9.2 About Us

It further demonstrates a comprehensive view of the marketplace with subsequent information. The latest mechanical enhancements and new releases delivered in the report will help customers settle on taught business decisions and complete their requisite executions in the future. In the conclusion part of this report, you will find research findings, market size, worldwide market share, consumer needs along with customer preference change, and data source.

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Global Personalized Cell Therapy Market Research Report (2019-2028) by Future Trend, Growth Rate, Opportunity, Industry Experts Analysis - Sound On...