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Category Archives: Cell Therapy

Global CAR-T Cell Therapy Market 2020 Opportunity Assessment, Analysis, Size, Share and Forecast Report to 2025 – Instanews247

Posted: January 5, 2020 at 3:44 am

The Global CAR-T Cell Therapy Market covers important aspects of this market concerning fundamental parameters. The report explains outline of the business range, concentrating on the overall industry, development possibilities, types and application. It brief CAR-T Cell Therapy summary of the market considering the current and future scenarios. It also provides information in terms of development and its capacities.

The CAR-T Cell Therapy industry analysis size, share, growth, trends, and forecasts 20202025. The CAR-T Cell Therapy report help to analysis players to improve their business strategies and helpful data. It shows key players in the worldwide market and trends about methodologies utilizing to separate themselves from other players. The analysis involves a broad outline of the CAR-T Cell Therapy market information on different particular divisions. The CAR-T Cell Therapy research report gives a pestal analysis rely upon the total market, available size, development scene, and analysis.

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Sorrento Therapeutics Inc., Legend Biotech (Genscript Biotech Corporation), Kite Pharma Inc. (Gilead Sciences Inc.), Pfizer Inc., Novartis International AG, Celgene Corporation, Mustang Bio Inc., CARsgen Therapeutics Ltd., Aurora Biopharma Inc

This CAR-T Cell Therapy report explores feasibility with an objective of educational new entrants in regards to the changes within the market. The description, thorough SWOT analysis & investment analysis is given which CAR-T Cell Therapy predictions are impending opportunities for its players.

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Global CAR-T Cell Therapy Market 2020 Opportunity Assessment, Analysis, Size, Share and Forecast Report to 2025 - Instanews247

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Lineage Cell Therapeutics Conducts Sale of Shares in OncoCyte Corporation – Business Wire

Posted: January 5, 2020 at 3:44 am

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced the pricing of the sale of 2,383,090 shares of common stock of OncoCyte Corporation at a price to buyers of $2.156 per share, representing the average closing price of OncoCyte common stock over the five trading days prior to the execution of the agreement. Net proceeds from the sale were approximately $5 million. The sale is expected to close by January 9, 2020, subject to certain closing conditions. Following the completion of the sale, Lineage will own approximately 6 million shares, which represents less than 10% of OncoCytes outstanding common stock. Based on the closing price of OncoCytes common stock on January 2, 2020, the value of Lineages remaining OncoCyte shares following the closing is approximately $13.5 million. Lineage has agreed not to sell additional shares of OncoCyte common stock until March 17, 2020 or unless the OncoCyte common stock price closes above $3.40.

Lineage is working diligently to become a leading cell therapy company and usher in a new branch of medicine, based on transplanting intact and differentiated cells into the body to restore activity lost to aging, injury, or disease, stated Brian M, Culley, CEO. To support our mission, we have deployed a financial strategy featuring intelligent cost-cutting alongside selectively selling our investments in OncoCyte and AgeX, companies which originated at Lineage prior to becoming independent public entities. Notably, we believe this most recent transaction involving OncoCyte will enable Lineage to have sufficient capital to support our core programs and will bridge us to the $21.6 million promissory note due to us in August 2020 from Juvenescence Ltd. Assuming the Juvenescence note is paid in cash and on time, we believe Lineages 2020 budget is now fully-funded and our available cash will extend well into 2021. Moving forward, we will continue to strategically assess and leverage all of the funding mechanisms at our disposal, including our remaining investments and potential partnership opportunities to support the development of our OpRegen, OPC1 and VAC2 programs, each of which may represent billion-dollar market opportunities in the emerging fields of cell therapy and immuno-oncology.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the timing and closing of Lineages sale of OncoCytes shares and Lineages planned spending for the year ending December 31, 2020. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Top 10 ALS Stories of 2019 – ALS News Today

Posted: January 5, 2020 at 3:44 am

Throughout 2019, ALS News Today brought you daily coverage of key findings, treatment developments, clinical trials, and other events related to amyotrophic lateral sclerosis (ALS).

As a reminder of what mattered most to you in 2019, here are the top 10 most-read articles of last year with a brief description of what made them interesting and relevant to the ALS community.

We look forward to reporting more news to patients, family members, and caregivers dealing with ALS during 2020.

No. 10 Experimental Gene Therapy Successfully Silences Key ALS Gene C9orf72, Preclinical Studies Show

A tale of two preclinical studies showed that a gene therapy candidate targeting a key ALS mutation in the C9orf72 gene was able to lessen the buildup of toxic RNA clumps and reduce the activity of the mutated gene in cells collected from a patient with frontotemporal dementia (FTD) and a mouse model of ALS.

Developed by uniQure,the therapy is designed to silence thedisease-causing gene. It works by delivering microRNAs (miRNAs) RNA molecules that regulate gene expression that target the mutated C9orf72s RNA for degradation. The results supported the continuation of uniQures gene therapy program in ALS and FTD, the company said.

No. 9 Altered Gut Microbiota in ALS Patients Could Drive Digestive Problems, Study Suggests

In the summer, a small study discovered an altered composition of gut microbes in people with ALS, which could drive digestive problems in those with the disease.

Using genetics, a research team in China found that fecal samples of people with ALS have an increase in harmful microbes of the phylum Firmicutes and a decrease in beneficial microorganisms called Bacteroidetes. The resulting poor gastrointestinal health may lead to a decline in the guts digestion and metabolism functions.

No. 8 Ibudilast-Rilutek Therapy Combo Closer to U.S. Patent for ALS and Other Neurodegenerative Diseases

At the beginning of the year, U.S. biopharmaceutical MediciNova received a notice of allowance stating that its request for a patent covering a combination of its investigational therapy ibudilast (MN-166) plus Rilutek (riluzole) was being consideredby the U.S. Patent and Trademark Office.

Ibudilast is a small molecule that reduces the activity of immune cells in the brain while supporting the growth of motor neurons, those lost in people with ALS. In a Phase 2 trial (NCT02238626), the treatment was found to work well in combination with Sanofis approved therapy Rilutek, improving patients functional activity, quality of life, and muscle strength.

The notice of allowance was the final step toward patent registration. Such registration will provide patent protection until November 2035 to the combination, for use in treating ALS and other neurodegenerative diseases.

No. 7 MediciNova Launches Phase 2b/3 Trial of Ibudilast for ALS

In June, MediciNova launched a Phase 2b/3 clinical trial to continue studying ibudilast as an add-on therapy to Rilutek. The multicenter, double-blind study (NCT04057898) will recruit approximately 230 participants, who will be randomly selected to receive either ibudilast plus Rilutek or a placebo plus Rilutek for 12 months.

The trials main goal is to study ibudilasts impact on ALS progression and functional disability by determining changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score between the beginning and end of the treatment period. Secondary objectives include changes in patients muscle strength, quality of life, and respiratory function. The study also will evaluate the safety and tolerability profile of ibudilast.

No. 6 FDA Approves Pivotal Phase 2b/3 Trial for Ibudilast in ALS Patients

Just a few months earlier, MediciNova had received approval from the U.S. Food and Drug Administration (FDA) to initiate this trial. Eligible patients must have had the disease for no more than 18 months and present just mild disability.

During the trial, patients will receive Rilutekfor at least 30 days before starting a regimen of either 100 mg per day of ibudilast or a placebo for a period of 12 months. Participants may then enter an extension phase in which they will be offered the ibudilast combination for an additional six months. Top-line data is expected by December 2021.

No. 5 Phase 3 Trial of NurOwn Cell Therapy Fully Enrolls 200 Patients, BrainStorm Announces

The Phase 3 trial evaluatingBrainStorm Cell Therapeuticss cell therapy candidateNurOwn completed patient enrollment in October. The 200 participants will receive three administrations of NurOwn, or a placebo, into the spinal canal every two months. The primary goals are to demonstrate the therapys safety and its ability to slow disease progression.

A cell-based therapy, NurOwn works by removing specific stem cells from patients and converting them to cells that produce molecules that promote nerve tissue growth and survival. The modified cells are then returned to the patient to stimulate nerve tissue growth. NurOwn aims to safely improve abilities like swallowing, speech, handwriting, and walking in people with ALS.

No. 4 High-dose Vitamin B12 May Improve ALS Prognosis if Started Early, Study Suggests

A long-term Phase 2/3 study examining vitamin B12 as a treatment for ALS found that ultra-high doses of methylcobalamin, the physiologically active form of this vitamin, may extend survival and slow the decline in functional capacity, compared with a placebo.

The study included 373 patients, diagnosed fewer than three years earlier, across 51 sites in Japan. However, the benefits were only seen in patients diagnosed less than one year before taking the supplements, and only a trend was observed.

No. 3 CuATSM Therapy May Slow ALS Progression, Improving Cognition and Respiration in Patients, Phase 1 Trial Shows

Results from a Phase 1 clinical trial (NCT02870634) showed that CuATSM a small molecule able to selectively deliver copper to cells with damaged mitochondria slowed disease progression and improved the respiratory and cognitive function of people with ALS.

Damaged mitochondria are considered a hallmark of several neurodegenerative diseases, including ALS, and delivering copper is thought to restore the health of these organelles. Developer Collaborative Medicinal Development (CMD) began testing CuATSM in patients with sporadic and familial ALS in 2016.CMD is now planning to launch a randomized, placebo-controlled clinical trial for CuATSM to confirm these results.

No. 2 Bacteria and Fungi Found in Central Nervous System of ALS Patients, Study Reports

In March, a study discovered several species of bacteria and fungi living in the central nervous system (CNS) of people with ALS, suggesting that patients have coexisting bacterial and fungal infections.

The study built on prior research suggesting that ALS might be caused by a fungal infection. Researchers then examined frozen CNS tissue from 11 ALS patients to assess whether bacterial infections accompany fungal infections. Bacterial DNA was found in different regions of the CNS and the presence of bacteria was confirmed in neural tissue samples.

No. 1 Nerve Injury May Trigger Onset, Progression of ALS Symptoms, Rat Study Suggests

Our most-read article of 2019reported that small nerve damage may serve as an ALS trigger. Such damage may accelerate motor symptoms in rats carrying a mutation in the SOD1 gene,one of the 40 genes associated with ALS development in humans.

While rats without the mutation completely recovered leg function four weeks after induced damage to the sciatic nerve, located in the leg, SOD1-mutated animals were unable to fully recover. These rats also lost function in the uninjured leg, likely as a consequence of sustained immune activation and more severe neurodegeneration.

The induced nerve damage mimics head injury and trauma in human patients, which could explain the higher prevalence of the disease among war veterans and professional athletes.

***

At ALS News Today, we hope these stories and our regular reporting throughout 2020 contribute to informing and improving the lives of everyone affected by ALS.

We wish all our readers a happy 2020.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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A First-in-Human Trial Offers Hope for CAR-T in Solid Tumors – Cancer Therapy Advisor

Posted: January 5, 2020 at 3:44 am

While chimeric antigen receptor T-cell (CAR-T) therapy has been making waves for the last few years as a novel treatment approach in cancer, the successes have remained mostly in the realm of blood cancers. This makes a first-in-human CAR-T clinical trial that targets a solid tumor growth receptor called MUC1* stand out. At the 34th Annual Meeting & Preconference Programs of the Society for Immunotherapy of Cancer, or SITC 2019, in November 2019, Minverva Biotechnologies shared an abstract offering some details behind this trial, which began in September 2019.1

Many clinical trials have tried to target the full-lengthMUC1 protein and have failed. The failures are likely due to the fact that MUC1is cleaved and released from the tumor surface, meaning any therapy that bindswith it goes too. When MUC1 is cleaved, however, the binding site for a crucialgrowth factor receptor that Minerva is calling MUC1* is revealed, said CynthiaBambdad, PhD, CEO of Minerva.

Leaving MUC1* exposed and unattended is like the pin beingpulled on the hand grenade, Dr Bamdad said. When a growth factor comes alongand binds to the MUC1* receptor, it causes the cancer cells to grow and becomeresistant to chemotherapy.

Minervas new investigational CAR-T therapy aims to swoop in and bind to the MUC1* receptor to block growth factors from accessing the MUC1 cleavage product. This binding and blocking action can, theoretically, stop a tumor from growing. The CAR-T therapy also aims to kill any mutant cancer cells in its vicinity and send out a call to the patients immune system to produce more like-minded CAR-T cells to shrink the tumor.

Its a very unique approach to MUC1, and thats why weemphasize that its different, said Matt Britz, head of business developmentat Minerva, referring to a line in the companys abstract that calls outprevious failed trials that looked only at full-length MUC1 and not the MUC1*receptor.

Up to now, CAR-T therapy has been so specifically successfulwith blood cancers because theyre malignancies of B cells, which CAR T-cellscan completely wipe out (it also can eliminate healthy B cells in the process,which can affect a patients ability to ward off infections). The patient mayget a sniffle more often, but is otherwise okay, Dr Bamdad remarked.

But this same approach doesnt work with solid tumors because they have no B-cell equivalent. Its been a challenge to find a solid tumor target that is not also present on healthy cells, which, if wiped out, could negatively impact a patients chances of survival.

Our approach is unique in that the CAR-T that we developedbinds to the part of MUC1* that is masked on healthy tissue, she said. TheCAR-T cells very selectively bind to and kill the tumor cells.

Dr Bamdad also said that MUC1*, which is expressedaberrantly in 95% of breast cancers, 83% of ovarian cancers, 78% of pancreaticcancers, and 71% of lung cancers, is the closest thing there is to a B-cellequivalent in solid tumors.

If this trial is successful, it will be a major step forward toward CAR-T cures for solid-tumor cancers, which make up 93% of all cancers, Dr Bamdad added.

Britz said that an obstacle in getting CAR-T therapy to gomainstream is that its still seen as being prohibitively expensive but heremains hopeful that as there are more successes in trials, there will be aparallel effort to bring the cost down. I think were in a paradigm shift inthe way that cancer is going to be treated, he said.

Reference

Bamdad C, Stewart A, Huang P, Smagghe B, Moe S, Swanson T,Jeon T, Page D, Mathavan K, Grant T, Herrup R. Poster presented at: the 34thAnnual Meeting & Preconference Programs (SITC 2019); November 610, 2019;National Harbor, MD. Abstract P150.

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Chronic PPI Use Linked to Declining Kidney Function Tops SPT Week in Review – Pharmacy Times

Posted: January 5, 2020 at 3:44 am

5. Mosunetuzumab Induces Complete Remissions in NHL After CAR T-Cell Therapy RelapseA novel, off-the-shelf bispecific antibody could serve as a new treatment option for patients with non-Hodgkin lymphoma who dont respond to CAR T-cell therapy. Read more.

4. Abemaciclib in Combination with Fulvestrant May Improve Care for Patients with HR+, HER2- Advanced Breast CancerAbemaciclib is a potent, oral cyclin-dependent kinase 4/6 inhibitor that has previously demonstrated statistically significant improvement in PFS and ORR in combination with endocrine therapy. Read more.

3. Microbes in Rectum Could be Key in Experimental HIV Vaccines Researchers aimed to understand whether microbes living in the rectum and vagina, sites of HIV transmission, interacted with experimental HIV vaccines similar to the HVTN 111 vaccine currently in early stage clinical trials in humans. Read more.

2. Tocilizumab Successful in Slowing Down Tuberculosis Bacillus Infection in Adult PatientsThe study led to insight on the process of tuberculosis bacillus infection as well as a potential drug treatment. Read more.

1. Chronic PPI Use Linked to Declining Kidney Function in Chronic Kidney DiseaseStudy findings presented at the American Society of Nephrologys Kidney Week 2019 suggest a link between chronic use of proton pump inhibitors and progression of kidney function decline in chronic kidney disease. Read more.

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Allogene Therapeutics to Present at the 38th Annual J.P. Morgan Healthcare Conference – Yahoo Finance

Posted: January 5, 2020 at 3:44 am

SOUTH SAN FRANCISCO, Calif., Jan. 02, 2020 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T) therapies for cancer, today announced that David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder, will present at the 38th Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 8:30 a.m. Pacific Time. The event will be held in San Francisco at the Westin St. Francis.

A live audio webcast of the presentation will be available on the Investors section of Allogenes website, http://www.allogene.com. A replay of the webcast will be available on the website for approximately 30 days. Please connect to the companys website at least 15 minutes prior to the start of the presentation to ensure adequate time for any software download that may be required to listen to the webcast.

AboutAllogene TherapeuticsAllogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T) therapies for cancer. Led by a world-class management team with significant experience in cell therapy, Allogene is developing a pipeline of off-the-shelf CAR T cell therapy candidates with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients. For more information, please visitwww.allogene.com, and follow @AllogeneTx on Twitter and LinkedIn.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability to develop allogeneic CAR T therapies for cancer and the potential benefits of AlloCAR T therapy. Various factors may cause differences between Allogenes expectations and actual results as discussed in greater detail in Allogenes filings with the Securities and Exchange Commission (SEC), including without limitation in its Form 10-Q for the quarter ended September 30, 2019. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Allogene Media/Investor Contact:Christine CassianoChief Communications Officer(714) 552-0326Christine.Cassiano@allogene.com

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What Does 2020 Have in Store for the Life Sciences? – BioSpace

Posted: January 5, 2020 at 3:44 am

With the books being closed on 2019, its time to prognosticate about what the next year will hold in the biotech and pharmaceutical industry. And, to paraphrase a famous song from the 1980s, grab a set of shades, because the future looks bright.

BioSpace spoke to leaders from various corners of the industry who provided their insights into what the coming year is likely to hold for their particular sphere. Below are predictions for three spheres within the industry for the coming year.

Oncology

Immuno-oncology has been one of the research cornerstones in cancer research and that will continue. However, in 2020, traditional targeted therapies will continue to be of importance. Stephen Gately, chief executive officer of TD2 (Translational Drug Development), an oncology-focused contract research organization, said there are still roles for traditional targeted therapeutics for specific indications. Following Mercks acquisition of ArQule, Inc. and its kinase inhibitor discovery and development programs for cancer treatment, predicted the industry might see a refocus on understanding cancer and how it can respond to different drugs. He said Merck would not have spent $2.7 billion for ArQules Phase II Brutons tyrosine kinase inhibitor ARQ 531 if there was not a potential upside for the therapy, particularly as a treatment for B-cell cancers. If trial protocols are well-established and there are stricter guidelines for patient selections, Gately said the use of certain inhibitors are vastly superior to immunotherapies in development.

When it comes to clinical trials, Gately predicted there could be a change in how those are organized. He said the goal for companies is to attempt to get their assets into trials as quickly as possible but noted there is a heated and competitive battle for trial sites. As companies attempt to elbow their way to the front of the line, Gately said it is possible that the industry will begin to see more opportunities for economic incentives used to convince the clinical trial sites to take up trials sooner. If that is established, Gately said it will become a game-changer.

Cell and Gene Therapies

This past year saw the approval of gene therapies that can address devastating diseases. And that is likely to continue in 2020. But, safety will still be paramount. Ena Cratsenburg, chief business officer of Ginkgo Bioworks, said over the course of the next year, we will see drug developers use synthetic biology to improve their ability to develop therapeutics with unprecedented levels of activity and control. In 2020, synbio companies will solidify their positions as key enablers in bringing the most innovative medicines to consumers, Cratsenburg said.

Mark Sawicki, chief commercial officer at Cryoport, developer of the first shippers certified for the delivery of human advanced therapies, said that as the cell and gene therapies continue to show their importance for treating new diseases, 2020 will usher in a record year for biologics license applications and marketing authorization applications. Sawicki suggested that over the course of the coming year, eight or nine companies will file such applications to gain regulatory approval of their products. In addition to the high number of filing companies, he suggested that there will be multiple new products launching with three or four generating revenues in 2020. By 2023, Sawicki predicts there will be at least 22 commercial launches in the space.

Jason Steiner, vice president of business development and strategy at gene editing company Synthego, said in 2020, the gap between the pace of innovation in the cell and gene therapy landscape and the infrastructure required for commercialization and deployment will continue to widen before it converges again.Steiner said the development of engineered cell therapies will continue to move toward non-viral approaches in order to boost engineering sophistication while decreasing the time and cost of traditional engineering. He added that manufacturing consideration of cell and gene therapies will continue to move farther upstream. They will be more tightly integrated with early development to avoid commercialization bottlenecks that are being caused by a rapid increase in the pace of development and regulatory approval cycles.

Real World Evidence

The importance of Real World Evidence (RWE) will continue to grow in 2020. Jane Reed, director of life sciences at U.K.-based Linguamatics, said the industry will continue to harness RWE in its drug development programs. RWE was not a topic of conversation in the industry 10 years ago, Reed said, but now there is significant value seen from what RWE brings to the table. RWE relates to the collection of information about a drugs safety and efficacy outside the structure of a clinical trial. The data can be collected from a variety of sources, including electronic health records, wearable devices, lab tests and more. The pharma industry will need to not only continue to collect the data, but learn to use it in a way that improves the outcomes of the patients it serves. Reed said the industry has to show they are listening to the patient community and be flexible. From there, the industry will need to feed that patient-driven data into drug discovery efforts. To boost the collection of RWE, Reed said the use of targeted social media research will also increase the amount of data that companies have at their fingertips. As companies within the pharmaceutical industry continue to dig into the depths of disease states, particularly those of rare diseases, Reed said the use of RWE will prove to be increasingly important.

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Global Musculoskeletal Disorder Stem Cell Therapy Market 2019 2024 Qualitative and Quantitative Assessment by Industry Analysis across the Value…

Posted: January 5, 2020 at 3:44 am

Global Musculoskeletal Disorder Stem Cell Therapy Market Growth (Status and Outlook) 2019-2024 Overview :

According to this study, over the next five years the Musculoskeletal Disorder Stem Cell Therapy market will register a xx% CAGR in terms of revenue, the global market size will reach US$ xx million by 2024

A newly revealed market research study titledGlobal Musculoskeletal Disorder Stem Cell Therapy MarketbyMRInsights.biz, studies market insights, list of significant key professionals, threats of new competitors and alternate products. The report presents in-depth analysis along with competitive insights, segmentation, and the market size information. In addition, it exploresMusculoskeletal Disorder Stem Cell Therapymarket size, trends, share, growth, development plans, growth opportunity, cost structure and drivers analysis.One part of this report contains production in which analysis on the production, revenue, gross margin of its main manufacturers in different regions from 2014 to 2019 has been covered. It also makes a prediction of its production and consumption in coming 2019-2024. The report provides a detailed analysis by the categorization of the global market on the basis of regions.

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Globally top leading manufacturers listed here: Osiris Therapeutics, NuVasive, Takeda (TiGenix), Medi-post,

Market region segmentation:

Furthermore, the SWOT analysis and strategies of each vendor are highlighted in the report which can be employed to create future opportunities.It sheds light on its applications, types, deployments, components, growths of this market. The research has also carried out analysis on upstream raw materials, equipment and downstream consumers. Next, it analyzes volume, utilization value, sale price, import and export in different regions from 2014 to 2019. On the basis of product, this report displays the production, revenue, price, market share and growth rate of each type, while for end use/application segment, this report focuses on the status and outlook for key applications.

The GlobalMusculoskeletal Disorder Stem Cell TherapyMarket Report Contains:

Next, the raw data collected through several sources has been processed using various mathematical and analytical tools and techniques in order to conclude the significant information related to future growth prediction trend for 2019 to 2024 time frame. At the end, research findings and conclusions specified in the report will help decision makers to take vital choices in the near future.

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There are 12 Chapters to deeply display the globalMusculoskeletal Disorder Stem Cell Therapymarket.

Chapter 1:Scope of the Report

Chapter 2:Executive Summary

Chapter 3:GlobalMusculoskeletal Disorder Stem Cell Therapyby Manufacturers

Chapter 4:Musculoskeletal Disorder Stem Cell Therapyby Regions

Chapter 5, 6, 7, 8 and 9:Americas,APAC,Europe,Middle East & Africa,Market Drivers, Challenges and Trends

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Global Musculoskeletal Disorder Stem Cell Therapy Market 2019 2024 Qualitative and Quantitative Assessment by Industry Analysis across the Value...

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Hepatitis C, cell therapy among breakthroughs in decade of progress – The Columbian

Posted: December 28, 2019 at 8:42 pm

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldnt be looking so far into the future without this decades extraordinary progress.

Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

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Hepatitis C, cell therapy among breakthroughs in decade of progress - The Columbian

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Lineage Cell Therapeutics Enters Into Three Separate License Agreements for Certain Intellectual Property and Assets – Business Wire

Posted: December 28, 2019 at 8:42 pm

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cellular therapies for unmet medical needs, today announced that it has entered into license agreements with three separate companies, each relating to different parts of Lineages intellectual property portfolio. All three companies have ongoing commercial operations, including with respect to their cell therapy-related assets. The aggregate up-front cash payment from the transactions was greater than one million dollars with additional cash and royalties due upon reaching certain development milestones or product sales.

Lineage is a leading cell therapy company with clinically-validated technology based on one of the largest and most comprehensive patent estates in cell therapy, stated Brian M. Culley, CEO. We believe our IP estate has increased in value in part due to advancements made by us and others in the field of cell therapy. As a result, we have placed a greater emphasis on completing business development transactions which can help fund our operations without having to raise capital at unattractive prices. We have entered into three separate agreements with entities in the cell therapy space for the license, development, manufacture and sale of products based on our broad cell therapy platform and technologies. Each of the agreements are with companies which have already commercialized cell therapy-related assets and are well-positioned to advance our programs. We believe these partnerships help validate the superiority of our well-characterized and NIH-approved human cell lines, which are not genetically manipulated, and provide an external endorsement of our inventions. We intend to provide further updates to our stockholders as we work with our new partners. We also are pursuing external partnership opportunities to support the development of our OpRegen, OPC1 and VAC2 programs, each of which may represent billion-dollar market opportunities in the emerging fields of cell therapy and immuno-oncology.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the evaluation of business development opportunities. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Lineage Cell Therapeutics Enters Into Three Separate License Agreements for Certain Intellectual Property and Assets - Business Wire

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