Category Archives: Stem Cell Therapy

publication date: Oct. 18, 2019

The UCLA Jonsson Comprehensine Cancer Center has launched a CAR T-cell immunotherapy trialthat will attack cancer cells by simultaneously recognizing two targetsCD19 and CD20that are expressed on B-cell lymphoma and leukemia.

By launching a bilateral attack instead of using the conventional single-target approach, researchers are hoping to minimize resistance and increase the life expectancy for people diagnosed with these cancers.

One of the reasons CAR T cell therapy can stop working in patients is because the cancer cells escape from therapy by losing the antigen CD19, which is what the CAR T cells are engineered to target, Sarah Larson, a health sciences clinical instructor in hematology/oncology at UCLA Health and the principal investigator on the trial, said in a statement One way to keep the CAR T cells working is to have more than one antigen to target. So, by using both CD19 and CD20, the thought is that it will be more effective and prevent the loss of the antigen, which is known as antigen escape, one of the common mechanisms of resistance.

Up to two-thirds of the patients who experience relapse after being treated with the FDA-approved CD19 CAR T-cell therapy develop tumors that have lost CD19 expression. UCLA researchers are identifying and testing new strategies like this one so many more patients can benefit from the therapy.

In preclinical studiesled byYvonne Chen, an associate professor of microbiology, immunology, and molecular genetics at UCLA and the sponsor of the trial, the team was able to show that by simultaneously attacking two targets, the engineered T cells developed in her lab could achieve a much more robust defense compared to conventional, single-target CAR T cells against tumors in mice.

Chens team designed the CARs based on the molecular understanding of the CARs architecture, the antigen structure and the CAR/antigen binding interaction to achieve optimal T cell function. This design helps the T cells have dual-antigen recognition to help prevent antigen escape.

Based on these results, were quite optimistic that the bispecific CAR can achieve therapeutic improvement over the single-input CD19 CAR thats currently available, said Chen, who is also the co-director of the Jonsson Cancer Centers Tumor Immunology Program and a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.

This first-in-humans study will evaluate the therapy in patients with non-Hodgkins B-cell lymphoma or chronic lymphocytic leukemia that has come back or has not responded to treatment. The goal is to determine a safe therapeutic dose.

Patients enrolled in the trial will have their white blood cells (T cells) collected intravenously then reengineered in the laboratory so the T cells can produce tumor-specific receptors (CARs), which allow the T cells to recognize and attack the CD19 and CD20 proteins on the surface of tumor cells. The new smarter and stronger T cells are then infused back into the patient and primed to recognize and kill cancer cells.

The trial is currently only offered at UCLA.

Results from STELLAR trial in MPM published in The Lancet Oncology

Novocure said the results from the STELLAR trial were published inThe Lancet Oncology.

The STELLAR trial was a prospective, single-arm trial including 80 patients that studied the use of Tumor Treating Fields, delivered via the NovoTTF-100L System, in combination with pemetrexed plus cisplatin/carboplatin as a first-line treatment for patients with unresectable, locally advanced or metastatic malignant pleural mesothelioma.

Data showed a median overall survival of 18.2 months (95 percent CI, 12.1 months-25.8 months) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. One- and two-year survival rates were 62.2 percent (95 percent CI, 50.3 percent-72.0 percent) and 41.9 percent (95 percent CI, 28.0 percent-55.2 percent), respectively. No serious systemic adverse events were considered to be related to the use of NovoTTF-100L. The most common mild to moderate adverse event was skin irritation beneath the transducer arrays.

The STELLAR trial demonstrated encouraging overall survival results with no increase in systemic toxicity observed in MPM patients treated with Tumor Treating Fields and standard chemotherapy, Giovanni Luca Ceresoli, head of pulmonary oncology at the Humanitas Gavazzeni Hospital in Bergamo, Italy, and principal investigator in the STELLAR trial, said in a statement. The median overall survival of 18.2 months is impressive given that MPM is a tumor with a dismal prognosis and few effective therapeutic options.

Median progression free survival was 7.6 months (95 percent CI, 6.7 percent-8.6 percent) for patients treated with NovoTTF-100L and pemetrexed plus cisplatin or carboplatin. There was a 97 percent disease control rate in patients with at least one follow-up CT scan performed (n=72). 40 percent of patients had a partial response, 57 percent had stable disease and 3 percent had progressive disease.

IASLC invites comments on Multidisciplinary Recommendations for Pathologic Assessment of Lung Cancer Resection Specimens Following Neoadjuvant Therapy

The International Association for the Study of Lung Cancer announced an open comment period for the IASLC Multidisciplinary Recommendations for Pathologic Assessment of Lung Cancer Resection Specimens Following Neoadjuvant Therapy paper.

The paper has been made available hereto provide an opportunity for public review of new draft recommendations. The open comment period runs from Oct. 14 to Nov. 7.

With the recent growing number of neoadjuvant therapy clinical trials for non-small cell lung cancer, there is a great need for standardization of specimen processing since major pathologic response has consistently been shown to be an important prognostic indicator.

The purpose of the paper is to outline detailed recommendations on how to process lung cancer resection specimens and to define pathologic complete response including major pathologic response and pathologic complete response following neoadjuvant therapy.

Currently there is no established guidance on how to process and evaluate resected lung cancer specimens following neoadjuvant therapy in the setting of clinical trials and clinical practice, Giorgio Scagliotti, past president of the IASLC and co-author of the paper, said in a statement. There is also a lack of precise definitions on the degree of pathologic response, including MPR or pCR.

IASLC is making an effort to collect such data from existing and future clinical trials. These recommendations are intended as guidance for clinical trials, although it is hoped they can be viewed as suggestions for good clinical practice outside of clinical trials, to improve consistency of pathologic assessment of treatment response.

The recommendations were developed by the IASLC Pathology Committee in collaboration with an international multidisciplinary group of experts in medical oncology, thoracic surgery and radiology.

We are crossing an exciting period of preclinical and clinical research around thoracic oncology. Targeted therapies and immunotherapy have greatly improved survival expectations in advanced disease and we believe they can equally generate benefit in the systemic therapy of earlier stages of the disease, Scagliotti said in a statement. Our initiative aims to use rigorous experimental conditions to analyze tissue specimens, collected in the context of already performed or ongoing neoadjuvant studies with targeted therapies and immunotherapy, to generate a diagnostic algorithm to be used in all subsequent studies in order to accelerate the scientific information about the clinical benefit produced by the neoadjuvant approach.

Expert second opinion improves reliability of melanoma diagnoses

Getting a reliable diagnosis of melanoma can be a significant challenge for pathologists.The diagnosis relies on a pathologists visual assessment of biopsy material on microscopic slides, which can often be subjective.

Of all pathology fields, analyzing biopsies for skin lesions and cancers has one of the highest rates of diagnostic errors, which can affect millions of people each year.

Now, a study led by UCLA researchers, has found that obtaining a second opinion from pathologists who are board certified or have fellowship training in dermatopathology can help improve the accuracy and reliability of diagnosing melanoma, one of the deadliest and most aggressive forms of skin cancer.

A diagnosis is the building block on which all other medical treatment is based,Joann Elmore, a professor of medicine at the David Geffen School of Medicine at UCLA and researcher at the UCLA Jonsson Comprehensive Cancer Center, said in a statement.All patients deserve an accurate diagnosis. Unfortunately the evaluation and diagnosis of skin biopsy specimens is challenging with a lot of variability among physicians.

In the study, led by Elmore and colleagues, the value of a second opinion by general pathologists and dermatopathologists were evaluated to see if it helped improve thecorrect diagnostic classification.

To evaluate the impact of obtaining second opinions, the team used samples from the Melanoma Pathology Study, which comprises of 240 skin biopsy lesion samples. Among the 187 pathologists who examined the cases, 113 were general pathologists and 74 were dermatopathologists.

The team studied misclassification rates, which is how often the diagnoses of practicing US pathologists disagreed with a consensus reference diagnosis of three pathologists who had extensive experience in evaluating melanocytic lesions. The team found that the misclassification of these lesions yielded the lowest rates when first, second and third reviewers were sub-specialty trained dermatopathologists. Misclassification was the highest when reviewers were all general pathologists who lacked the subspecialty training.

Our results show having a second opinion by an expert with subspecialty training provides value in improving theaccuracy of thediagnosis, which is imperative to helpguide patients to the most effective treatments, said Elmore, whois also the director of the UCLA National Clinician Scholars Program.

Elmore is now studying the potential impact of computer machine learning as a tool to improve diagnostic accuracy. She is partnering with computer scientists who specialize in computer visualization of complex image information, as well as leading pathologists around the globe to develop an artificial intelligence (AI)-based diagnostic system.

Michael Piepkorn of the University of Washington School of Medicine is the studys first author. Raymond Barnhill of the Institut Curie is the co-senior author.

The study was published in JAMA Network Open and supported by NCI.

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UCLA opens CAR T-cell trial focused on the most common types of lymphoma, leukemia - The Cancer Letter Publications

Delegates from the American Academy of Stem Cell Physicians recently traveled to the Bahamas to meet with the Governor General Sir Cornelius Alvin Smith to discus medical industry safety standards.

While in the Bahamas the delegates attended a 3 day regenerative health summit for educators, experts, professionals and learners where in doctors from all over the world arrived to discuss the future of regenerative medicine and stem cell therapy.

Dr. Krutchkoff announced the launch of the AASCPs Research Consortium dedicated to supporting research to validate therapies and promote compliance within the industry. During the HEALinc Summit Dr. Farschian and Dr. Krutchoff, the American Academy of Stem Cell Physicians delegates, held a workshop to teach physicians about a pathway to compliance when utilizing cell therapies in their practices.

The AASCP was created to advance research and the development of therapeutics in regenerative medicine, and aims to serve as an educational resource for scientists, physicians, and the public in diseases that can be caused by physiological dysfunction that are ameliorable to medical treatment.

The HEALinc Summit was organized by Dr. Desiree Cox, CEO of the HEALinc Future of Health Innovation Summit and The HEALinc Innovation Incubator which is a movement based organization with a mandate to champion a future health paradigm where medical therapy is driven by personal health and regenerative medicine by incorporating ongoing, real world capture, predictive analytics, and education.

Dr. Cox currently chairs the National Stem Cell Ethics Committee for the Bahamas, and has been leading health innovation and the development of the stem cell and regenerative industry since 2015 in the Bahamas which is a fast track test bed for real world studies in stem cell, regenerative medicine, and medical devices among others.

During the HEALinc Summit leading international experts spoke on hot topics in stem cell therapy, longevity and vitality, regeneration, resilience and adaptability, precision health, epigenetic and nutrition, personal wellness and optimal health, regenerative ventures/investment and much more.

"Within the Bahamas, health innovations in cellular and gene therapies, regenerative medicine, precision medicine and digital health can be tested ethically and safely in a regulated environment, so that the data obtained from the research can be used in later applications for FDA or EMA approval, says Dr. Cox.

Dr. AJ Farshchian, spokesperson for AASCP said, "It was a great honor meeting the Governor General in the Bahamas. We discussed safety standards currently practiced in the industry and how to improve the industry moving forward."

Dr. Adam Shwani, a member of the board for the American Academy of Stem Cell Physicians, added, "It is so important to bring industry partners together from all over the world to develop a standard that promotes patient safety and encourages growth in the field of regenerative medicine.

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AASCP Delegates Meet With The Governor General Of The Bahamas - Anti Aging News

Company Announcement

Copenhagen, Denmark; October 18, 2019 Genmab A/S (Nasdaq: GMAB) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending broadening the existing marketing authorization for DARZALEX (daratumumab) in the European Union. The recommendation is for the use of DARZALEX in combination with lenalidomide and dexamethasone (Rd) as treatment for newly diagnosed adult patients with multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). The Type II variation application, based on the Phase III MAIA (MMY3008) study, was submitted to the EMA by Janssen Pharmaceutica NV in March 2019. In August 2012, Genmab granted Janssen Biotech, Inc. an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

We are encouraged that the CHMP are recommending a broadening of the current DARZALEX marketing authorization in the European Union to include DARZALEX in combination with lenalidomide and dexamethasone as a possible treatment for patients newly diagnosed with multiple myeloma. This would give patients another treatment option, in addition to the already approved combination of daratumumab plus bortezomib, melphalan and prednisone in this same setting, said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the MAIA (MMY3008) studyThe Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either treatment with daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or treatment with lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone is administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is progression free survival.

About multiple myelomaMultiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 16,830 new patients were expected to be diagnosed with multiple myeloma and approximately 10,480 people were expected to die from the disease in the Western Europe in 2018.2 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX (daratumumab)DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy6. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adults with relapsed or refractory multiple myeloma and in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit http://www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a persons own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis, NKT-cell lymphoma and B-cell and T-cell ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

About Genmab Genmab is a publicly traded, international biotechnology company specializing in the creation and development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999, the company has two approved antibodies, DARZALEX (daratumumab) for the treatment of certain multiple myeloma indications, and Arzerra (ofatumumab) for the treatment of certain chronic lymphocytic leukemia indications. Daratumumab is in clinical development for additional multiple myeloma indications, other blood cancers and amyloidosis. A subcutaneous formulation of ofatumumab is in development for relapsing multiple sclerosis. Genmab also has a broad clinical and pre-clinical product pipeline. Genmab's technology base consists of validated and proprietary next generation antibody technologies - the DuoBody platform for generation of bispecific antibodies, the HexaBody platform, which creates effector function enhanced antibodies, the HexElect platform, which combines two co-dependently acting HexaBody molecules to introduce selectivity while maximizing therapeutic potency and the DuoHexaBody platform, which enhances the potential potency of bispecific antibodies through hexamerization. The company intends to leverage these technologies to create opportunities for full or co-ownership of future products. Genmab has alliances with top tier pharmaceutical and biotechnology companies. Genmab is headquartered in Copenhagen, Denmark with core sites in Utrecht, the Netherlands and Princeton, New Jersey, U.S.

Contact: Marisol Peron, Corporate Vice President, Communications & Investor Relations T: +1 609 524 0065; E: mmp@genmab.com

For Investor Relations: Andrew Carlsen, Senior Director, Investor RelationsT: +45 3377 9558; E: acn@genmab.com

This Company Announcement contains forward looking statements. The words believe, expect, anticipate, intend and plan and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with pre-clinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmabs most recent financial reports, which are available on http://www.genmab.com and the risk factors included in Genmabs final prospectus for our U.S. public offering and listing and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at http://www.sec.gov. Genmab does not undertake any obligation to update or revise forward looking statements in this Company Announcement nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law.

Genmab A/S and/or its subsidiaries own the following trademarks: Genmab; the Y-shaped Genmab logo; Genmab in combination with the Y-shaped Genmab logo; HuMax; DuoBody; DuoBody in combination with the DuoBody logo; HexaBody; HexaBody in combination with the HexaBody logo; DuoHexaBody; HexElect; and UniBody. Arzerra is a trademark of Novartis AG or its affiliates. DARZALEX is a trademark of Janssen Pharmaceutica NV.

1 American Cancer Society. "Multiple Myeloma Overview." Available at http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma.Accessed June 2016.2 Globocan 2018. Western Europe Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/926-western-europe-fact-sheets.pdf Accessed March 20183 Globocan 2018. World Fact Sheet. Available at http://gco.iarc.fr/today/data/factsheets/populations/900-world-fact-sheets.pdf. Accessed December 2018.4 American Cancer Society. "How is Multiple Myeloma Diagnosed?" http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed June 20165 DARZALEX Prescribing information, September 2019. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761036s024lbl.pdf Last accessed September 20196 DARZALEX Summary of Product Characteristics, available at https://www.ema.europa.eu/en/medicines/human/EPAR/darzalex Last accessed October 20197De Weers, M et al. Daratumumab, a Novel Therapeutic Human CD38 Monoclonal Antibody, Induces Killing of Multiple Myeloma and Other Hematological Tumors. The Journal of Immunology. 2011; 186: 1840-1848.8 Overdijk, MB, et al. Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma. MAbs. 2015; 7: 311-21.9 Krejcik MD et al. Daratumumab Depletes CD38+ Immune-regulatory Cells, Promotes T-cell Expansion, and Skews T-cell Repertoire in Multiple Myeloma. Blood. 2016; 128: 384-94.10Jansen, JH et al. Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking.Blood. 2012; 120(21): abstract 2974.

Company Announcement no. 50CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122

Genmab A/SKalvebod Brygge 431560 Copenhagen VDenmark

Original post:
CHMP Issues Positive Opinion Recommending DARZALEX (Daratumumab) in Combination with Lenalidomide and Dexamethasone in Frontline Multiple Myeloma -...

The landmark approvals of Yescarta and Kymriah have spurred unprecedented advancements in the market. The launch of these breakthrough therapies has bolstered cash inflow for innovation, thereby driving the growth.

New York, Oct. 16, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "T-Cell Therapy Market Size, Share & Trends Analysis Report By Modality, By Therapy, By Indication And Segment Forecasts, 2019 - 2026" - https://www.reportlinker.com/p05822974/?utm_source=GNW

Expansion of the market for T-cell therapy significantly relies on shifting preference from first-line stem cell transplants and chemotherapy to third-line CAR T-cell therapy.Moreover, the ever-expanding plethora of medical conditions for which the T cell therapies is projected to bode well for the market growth.

Rise in oncological disorders is projected to drive interest as well as investments in the T-cell therapy market in near future.

In contrast with the small-molecule landscape, engineered T cells market landscape is distinguished by an extensive network that encompasses several entities marked by connections academically, financially, and via technology licensing. Research bodies, and manufacturers, and regulators engage in assessing the long-term efficacy and safety of therapies to ensure safe access to patients.

By far, the antigen challenge and linked toxicity concerns have impeded the development of CAR T therapies in non-hematological malignancies. Market players are applying a data-driven approach of exploring this space to mitigate the challenge and expand the usage of T-cell therapy in indication type such as brain cancer and melanoma.

Further key findings from the report suggest: In the coming years, the number of hospitals implementing CAR T therapies is expected to increase, thereby driving the commercialized business model Research-based business modality accounted for the highest revenue generation over the past years, attributed to the presence of several research programs CAR-T cell therapy market share accounted for the larger revenue share of the overall T-cell therapy market in 2018 owing to the highest investment by sponsors in this therapy type Presence of approved products for B cell lymphoma and acute lymphocytic leukemia has resulted in the dominance of hematological malignancies in the market Therapy for solid tumors is expected to emerge as the lucrative source of revenue generation in the forthcoming years Rising research activities in CAR T-cell therapy for solid tumors, particularly, for brain & central nervous system and melanoma, is expected to benefit the key players North America led the global market in 2018 owing to the presence of a large number of cancer centers engaging in research activities. Moreover, U.S.-based pharma companies like Pfizer Inc. and Celgene Corporation have shifted their focus from conventional drug development to T-cell therapy space Asia Pacific is estimated to witness the fastest growth with China at the forefront. China has surpassed the number of CAR T clinical trials conducted in U.S. Moreover, Novartis is strategizing to secure approval for Kymriah by China regulatory authorities in the forthcoming years Gilead Sciences, Novartis AG, TCR2 Therapeutics Inc., Celgene Corporation, Sorrento Therapeutics, bluebird bio, and Fate Therapeutics are some key players operating in the market. They engage in mergers and acquisitions with therapy developers. Acquisition of Juno Therapeutics by Celgene in January 2018 and Kite Pharma by Gilead in August 2017 are some notable examples of acquisitions in this marketRead the full report: https://www.reportlinker.com/p05822974/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global T-cell therapy market size is expected to reach USD 7.51 billion, expanding at a CAGR of 15.4% by 2026 - Yahoo Finance

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IRVINE, Calif., Oct. 15, 2019 /PRNewswire/ -- AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, announced today that the Companys ROOT of SKIN line of rejuvenating skincare products sold out in its tenth appearance on the Japanese home shopping channel QVC Japan.

ROOT of SKIN is AIVITAs proprietary skincare line for skin rejuvenation, made possible through the same proprietary knowledge and expertise used to develop its cutting-edge stem cell therapies. SourceCode Technology, the proprietary actives complex in ROOT of SKIN, contains the complete set of factors and supporting biological components present in young, healthy skin.

AIVITA Biomedical uses 100% of the proceeds from ROOT of SKIN sales to support the treatment of women with ovarian cancer.

Outstanding market traction, a product that truly works, and a benevolent use of proceeds, said Hans Keirstead, AIVITAs Chief Executive Officer. This sets the stage for global expansion of our ROOT of SKIN product line.

About ROOT OF SKIN

ROOT of SKIN is a rejuvenating line of skincare products fueled by an unrelenting pursuit for advancements in life-changing and life-saving treatments. Harnessing breakthroughs in stem cell therapy, AIVITA Biomedical developed a technology that does more than just boost regeneration. The patented actives complex SourceCode Technology renews, repairs and protects, just as your skin did at its youngest and healthiest stage. Rich with every biological component healthy skin needs for development, and free of any unnecessary ingredients. All proceeds support treatment of women with ovarian cancer.

About AIVITA Biomedical

AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products.

View original content to download multimedia: http://www.prnewswire.com/news-releases/root-of-skin-skincare-products-sell-out-in-tenth-consecutive-appearance-on-qvc-japan-300938926.html

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ROOT OF SKIN Skincare Products Sell Out in Tenth Consecutive Appearance on QVC Japan - Associated Press

DUBLIN--(BUSINESS WIRE)--The "Global Longevity and Anti-Senescence Therapy Market" report has been added to ResearchAndMarkets.com's offering.

Global longevity and anti-senescence market will witness rapid growth over the forecast period (2018-2023) owing to an increasing emphasis on Stem Cell Research and increasing demand for cell-based assays in research and development.

An increasing geriatric population across the globe and rising awareness of antiaging products among generation Y and later generations are the major factors expected to promote the growth of global longevity and anti-senescence market. Factors such as a surging level of disposable income and increasing advancements in anti-senescence technologies are also providing traction to the global longevity and anti-senescence market growth over the forecast period (2018-2023).

Senolytics, placenta stem cells and blood transfusions are some of the hot technologies picking up pace in the longevity and anti-anti-senescence market. Companies and start-ups across the globe such as Unity Biotechnology, Human Longevity Inc., Calico Life Sciences, Acorda Therapeutics, etc. are working extensively in this field for the extension of human longevity by focusing on the study of genomics, microbiome, bioinformatics, and stem cell therapies, etc. These factors are poised to drive market growth over the forecast period.

The report provides analysis based on each market segment including therapies and application. The therapies segment is further sub-segmented into Senolytic drug therapy, Gene therapy, Immunotherapy, and Others. Senolytic drug therapy held the largest market revenue share in 2017. The fastest growth of the gene therapy segment is due to the Large investments in genomics.

Report Scope

The scope of this report is broad and covers various therapies currently under trials in the global longevity and anti-senescence therapy market. The market estimation has been performed with consideration for revenue generation in the forecast years 2018-2023 after the expected availability of products in the market by 2023.

The global longevity and anti-senescence therapy market has been segmented by the following therapies: Senolytic drug therapy, Gene therapy, Immunotherapy and Other therapies which includes stem cell-based therapies, etc.

Revenue forecasts from 2028 to 2023 are given for each therapy and application, with estimated values derived from the expected revenue generation in the first year of launch.

The report also includes a discussion of the major players performing research or the potential players across each regional longevity and anti-senescence therapy market. Further, it explains the major drivers and regional dynamics of the global longevity and anti-senescence therapy market and current trends within the industry.

The report concludes with a special focus on the vendor landscape and includes detailed profiles of the major vendors and potential entrants in the global longevity and anti-senescence therapy market.

The report includes:

Key Topics Covered

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market Overview

Chapter 4 Global Longevity and Anti-senescence Market by Therapy

Chapter 5 Global Longevity and Anti-senescence Market by Application

Chapter 6 Global Longevity and Anti-senescence Market by Region

Chapter 7 Industry Structure in Longevity and Anti-senescence Market

Chapter 8 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/zy7jt

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Global Longevity & Anti-Senescence Therapy Market Review 2017-2018 and Forecast to 2023 - ResearchAndMarkets.com - Business Wire

In AML, the role of ecotropic virus integration site-1 (EVI1) expression is debated. To date, results of studies have been mixed with only some studies demonstrating EVI1 expression related to poorer survival. In a meta-analysis published in the Annals of Hematology, researchers set out to uncover the predictive capability of this marker.

As a malignant disorder in hematology usually with a poor prognosis, AML needs an accurate prediction of prognosis to indicate protocoling the appropriate therapy regimens for patients hoping for survival improvement, wrote authors, led by Xia Wu, Department of Hematology, West China Hospital, Sichuan University, Sichuan Province. Molecular markers increasingly play an utmost significant role in the diagnosis and risk stratification of AML.

In the current meta-analysis, Wu et al. mined 11 studies for 4767 AML patients with intermediate cytogenetic risk (ICR), according to National Comprehensive Cancer Network (NCCN), International System for Human Cytogenetic Nomenclature (ISCN), or European leukemia network (ELN) guidance. The findings indicated that EVI1 expression negatively impacted OS (HR = 1.73, 95%CI 1.432.11) and event=free survival or EFS (HR = 1.17, 95%CI 1.051.31). Furthermore, EVI1 was a negative predictor of prognosis in patients with normal cytogenetics (NC) and younger patients (< 60 years).

Importantly, the investigators noted that due to location, altered EVI1 most often accompanies 3q26 rearrangements. However, it remains to be elucidated whether increased EVI1 expression is related to AML outcomes in those without 3q mutations. On a related note, higher levels of EVI1 may affect AML subgroups differently, which, according to the authors, is of utmost significance for clinical physicians.

In other findings, EVI1H expression was rarely found with NPM1, FLT3-ITD and DNMT3A mutations. Wu et al point to these mutations and mutations as avenues of further research.EVI1 is a transcription factor on chromosome 3. It was first discovered two decades ago in murine models. It has stem cell specific expression patterns and mediates growth of hematopoietic stem cells, and plays a role in AML, myelodysplastic syndrome (MDS), and CML.

The investigators suggested that the findings of the current study could assist clinicians with risk stratification and treatment decisionsespecially because most patients are NC.

EVI1, which also goes by MECOM, encodes a 145 kDa-unique zinc finger that attaches with DNA. This transcription factor is hypothesized to interfere with granulocyte and erythroid cell differentiation, as well as promotion of megakaryocyte breakdown, to aid with the differentiation and proliferation of hematopoetic stem cells.Several drug targets for EVI1 have been suggested such those involved in leukemogenesis and stem cell maintenance. Examples include the transcription factor Pre-B Cell leukemia Homeobox 1 (PBX1) and Phosphatase and Tensin Homolog (PTEN), which is a tumor suppressor gene. However, none of these targets have proven related to EVI1-deregulated AML.

Per the authors the current study had several limitations. First, most studies in the meta-analysis were observational and not randomized-controlled trials. Second, the sample contained cases of therapy-related AML and secondary AML, which have a worse prognosis and could thus confound results. Third, limited OS data precluded the ability to study AML patients without 3q alterations. Fourth, the studies were highly heterogeneous in a clinical sense.

Reference

Wu X et al. Prognostic significance of the EVI1 gene expression in patients with acute myeloid leukemia: a meta-analysis. Annals of Hematology. 2019 Sep 3. doi: 10.1007/s00277-019-03774-z.

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The Prognostic Importance of EVI1 Expression - Cancer Network

Mogrify Ltd (Mogrify), a UK company aiming to transform the development of life-saving cell therapies, recently announced the initial close of its Series A funding. The Company raised $16M USD in this round, bringing the total investment to over $20M USD to date. The funding will support internal cell therapy programs, and the development and out-license of novel IP relating to cell conversions of broad therapeutic interest. Mogrify is also actively recruiting, and will increase headcount to 60 scientific, operational and commercial staff located at its state-of-the-art facility on Cambridge Science Park.

The funding round was led by existing investor Ahren Innovation Capital (Ahren), an investment fund co-founded by leading UK scientific entrepreneurs, supporting transformational companies at the cutting edge of deep science and deep tech. Parkwalk, the largest EIS growth fund manager, backing businesses with IP-protected innovations creating solutions to real-world challenges, 24Haymarket, an early investor in Mogrify and a prolific early-stage investment syndicate in deep technology and the life sciences, and the University of Bristol Enterprise Fund III, also contributed to the fundraise.

Mogrify has developed a proprietary direct cellular conversion technology, which makes it possible to transform (transmogrify) any mature human cell type into any other without going through a pluripotent stem cell- or progenitor cell-state. The Company is deploying this platform to develop novel cell therapies addressing musculoskeletal, auto-immune, cancer immunotherapy, ocular and respiratory diseases as well as generating a broad IP position relating to cell conversions that exhibit safety, efficacy and scalable manufacturing profiles suitable for development as cell therapies.

Mogrify is commercializing its technology platform via a model that includes development and out-license of internally developed cell therapy assets, development and license of novel cell conversion IP, and the formation of joint-ventures to exploit the platform and/or novel cell conversion IP in non-core areas.

Mogrify launched in February 2019, announcing $3.7M USD seed funding from Ahren, 24Haymarket and Dr. Darrin M. Disley, OBE and went on to secure grants from Innovate UK and SBRI Healthcare.

Mogrify also strengthened its management and scientific teams and relocated a 20-strong workforce to its new headquarters at TusParks Cambridge Bio-Innovation Centre on Cambridge Science Park in May. It has now begun recruiting up to 40 additional commercial, operational and scientific roles to support its expanding pipeline of internal programs, as well as supporting numerous biotech and pharma collaborators in developing novel IP to underpin existing and new cell therapy programs.

Dr. Darrin M. Disley, OBE, CEO, Mogrify, said: Following the recent announcement of Dr. Jane Osbourn, OBE, as Chair of Mogrify, I am delighted we have been able to make an initial close of this fundraising round, with the backing of both existing and new investors. Due to the significant interest, we have been able to secure this growth-funding without engaging in a protracted and distracting fund-raising process. Having now raised over $20M, we can focus on delivery of our business strategy with the support of an aligned investor group. We will continue to engage with high-caliber investors with computational biology and cell therapy domain expertise as part of our on-going investor relations and capital markets strategy.

Alice Newcombe-Ellis, Founder and Managing Partner, Ahren Innovation Capital, said: Mogrifys technology is well positioned to disrupt the global cell therapy market. The Company has grown rapidly since February, appointing a world-class management team and delivering strongly against its business plans. We look forward to supporting Mogrify as it continues to go from strength to strength.

Alastair Kilgour, Chief Investment Officer, Parkwalk, said: We are delighted to be supporting the team at Mogrify, many of whom have been involved successfully with companies we have previously invested in, in this investment round. The science and technology base Mogrify are building is truly unique and disruptive. If successful, the positive effect on patient outcomes across a wide range of diseases will be staggering.

Alice Newcombe-Ellis, Founder and Managing Partner of Ahren, and Alastair Kilgour, Chief Investment Officer, Parkwalk, both join Mogrifys board of directors along with Dr. Karin Schmitt, the Companys Chief Business Officer.

For more information, visit https://mogrify.co.uk/investors/

About MogrifyMogrify has developed a proprietary direct cellular conversion technology, which makes it possible to transform (transmogrify) any mature human cell type into any other without going through a pluripotent stem cell- or progenitor cell-state.

The platform takes a systematic big-data approach to identify, from next-generation sequencing and gene-regulatory networks, the transcription factors (in vitro) or small molecules (in vivo), needed to convert a cell. By bypassing the stem cell-stage of cell transformation, Mogrify simultaneously addresses challenges associated with efficacy, safety and scalability.

Mogrify is deploying this platform to develop novel cell therapies addressing musculoskeletal, auto-immune, cancer immunotherapy, ocular and respiratory diseases as well as generating a broad IP position relating to cell conversions that exhibit safety, efficacy and scalable manufacturing profiles suitable for development as cell therapies.

Uniquely positioned to address a cell therapy market estimated to be $35B USD by 2023, Mogrify is commercializing its technology via IP licensing, product development, and drug development. Based in Cambridge, UK, the Company has raised over $20M USD funding from Ahren Innovation Capital, Parkwalk, 24Haymarket, Dr. Darrin M. Disley, OBE and the University of Bristol Enterprise Fund III. For more information, visit http://www.mogrify.co.uk

About Ahren Innovation CapitalAhren LP is an investment fund that supports transformational companies at the cutting edge of deep science and deep tech. The technologies of its Founding Partners are today valued more than $100B combined.

A group of highly diverse, creative and original thinkers leading their domains, Ahren believes in taking considered risk that will deliver superior rewards capturing a generational opportunity to provide smart capital to deep technology pioneers.

With a philosophy espousing the importance of relationships and trust, Ahren provides long-term capital and support to exceptional founders and teams, empowering them to achieve the unimaginable.

Ahren Innovation Capital was founded by Alice Newcombe-Ellis, together with Science Partners Sir Shankar Balasubramanian, Professor John Daugman, Professor Zoubin Ghahramani, Professor Steve Jackson, Professor Andy Parker, Sir Venki Ramakrishnan, Lord Martin Rees and Sir Gregory Winter. For more information, visit http://www.ahreninnovationcapital.com

About ParkwalkParkwalk is the largest growth EIS fund manager, backing world-changing technologies emerging from the UKs leading universities and research institutions. With 250M of assets under management, it has invested in over 100 companies across its flagship Parkwalk Opportunities EIS Fund as well as the award-winning enterprise and innovation funds Parkwalk manages for the Universities of Cambridge, Oxford and Bristol.

Parkwalk invests in businesses creating solutions to real-world challenges, with IP-protected innovations, across a range of sectors including life sciences, AI, quantum computing, advanced materials, genomics, cleantech, future of mobility, MedTech and big data.

For more information, visit http://parkwalkadvisors.com

About 24Haymarket24Haymarket is a premium deal-by-deal investment platform focused on high-growth businesses, investing up to 5M in any company. 24Haymarkets Investor Network includes several highly experienced private equity and venture capital investors, seasoned entrepreneurs and senior operators. We invest our own capital in direct alignment with entrepreneurs and typically seek Board representation to actively support their growth agenda. Since its inception in 2011, 24Haymarket has invested in more than 50 high-growth businesses. For more information, visit http://www.24haymarket.com

The University of Bristol Enterprise Fund III (Managed By Parkwalk)The University of Bristol Enterprise Fund is an early stage investment fund backing scientific and technological companies spun out of the University of Bristol or being supported by the Universitys SETsquared incubator. For more information, visit http://parkwalkadvisors.com/fund/university-of-bristol-enterprise-fund.

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Mogrify Raises Additional $16M To Advance Its Mission To Transform The Development Of Life-Saving Cell Therapies - Clinical Leader

Oct 15, 2019

The Equine Medical Research Fund was created in 2014 to provide consistent funding for equine medical research, which has historically been underfunded. A $1.00 starter fee was added to each USEA recognized event entry beginning in 2014, and at years end a majority of those dollars were distributed through the Morris Animal Foundation, which assists the USEA with selecting studies to fund, monitoring the funds, and reporting on progress. Morris has decades of experience in reviewing grant applications from universities and research institutions and has a team of veterinarians who spend thousands of volunteer hours assessing which studies are most likely to impact horse welfare. Ultimately, a committee from the USEA comprised of riders and veterinarians will determine which studies the USEA dollars will target.

This years committee recommendation is a bit different as the committee is recommending spreading funds among three entities - the Stephen Teichman Farrier study, a Grayson Jockey Club Research Foundation study, and two Morris Animal Foundation Studies.

Through these partnerships, the USEA is helping to address the major problem of equine research underfunding while maintaining control over how member dollars are distributed. Although the committee is always interested in recommending studies that concern general horse welfare, this year we took a closer look at recommending studies that were geared to solving issues that concern performance horses. The goal of our selection philosophy is to provide USEA positive PR through funding a variety of institutions and studies that show our support for horse care, welfare, and rehabilitation.

In 2017, $46,900 dollars were distributed between five different studies on different topics pertaining to equine health, from improving regenerative therapies to studying gaps in the equine genome related to tendon health.

In 2018, $44,858 dollars were distributed between four different studies on a variety of topics including insulin's relation to laminitis, stem cell therapies, equine asthma, and joint infections.

At the USEA Board of Governors meeting in August, the Equine Medical Research Committee recommended the funding of four studies using the $39,581 collected in 2018 and the Board approved their recommendation. Below are additional details about the four selected studies.

Non-Invasive Evaluation of Host-Microbiota Interactions Canaan Whitfield-Cargile, Texas A&M

Summary: This study aims to develop a non-invasive platform to serve as a diagnostic test for gastrointestinal inflammation prior to severe disease and to reveal how bacteria in the gut influence horse health.

Description: One of the most important and common classes of diseases affecting horses are gastrointestinal (GI) diseases including colic and colitis. Despite tremendous research efforts to both diagnose and prevent these diseases, they remain very common and frequently are not diagnosed until severe disease is present. One area that is gaining recognition as an important player in equine GI health and disease is GI bacteria or the microbiota. The microbiota consists of all of the bacteria and other microorganisms in the GI tract. Alterations in the amounts and types organisms that make up the microbiota have been linked to both GI and non-GI diseases of horses including colic, colitis, laminitis, and obesity. These findings are part of the reason why there are a vast array of prebiotics, probiotics, and other products on the market that claim to influence the GI microbiota. Unfortunately, there is minimal evidence to support the efficacy of these products and a lack of understanding of how these products work. A major contributor to the lack of evidence and understanding of these products is related to an inability to monitor the health and function of the equine GI tract. It is clear when severe disease is present but currently impossible to monitor subtle changes. Similarly, while we can easily diagnose severe inflammation and microbiota changes during disease, we cannot capture the changes that occurred prior to clinical disease. These limitations result in capturing results of GI injury and disease but not causes. Consequently, there is great need for improved understanding of the causes of disease and early identification of horses at-risk of developing GI disease. In the absence of such knowledge, effective intervention strategies to reduce GI and GI-related health risks will remain elusive.

Horses and other animals naturally shed cells lining the GI tract on a daily basis. People, in fact, shed up to 1/3 of the cells lining their GI tract daily. We have developed a technique for examining the gene expression profile of these cells to monitor the health and function of the GI tract from fecal samples. We have shown that this approach offers a promising means to non-invasively examine the response of the GI intestinal tract to injury. Further, we have convincing preliminary data demonstrating that the gene expression profile arising from these cells mirrors the gene expression profile of the tissues within the GI tract of the horse and provides a global view of the health and function of the GI tract. This approach offers a highly attractive, non-invasive means of monitoring GI responsiveness to disease and interventions. Thus, our long-term goals are to 1) develop inexpensive, non-invasive diagnostic tests in order to document GI inflammation prior to the onset of severe disease in horses; and, 2) by examining host and microbiota data simultaneously we aim to elucidate the mechanisms of host-microbiota interactions in the context of health and disease. Ultimately, we aim to use this approach to identify specific pathway by which the host and microbiota interact so that this critically important interaction can be targeted therapeutically.

In order to achieve our goals, we will utilize a reversible model of GI inflammation with which we have extensive experience. We will induce GI inflammation with the non-steroidal anti-inflammatory drug (NSAID) phenylbutazone and noninvasively interrogate the response and response of the microbiota. Importantly, we will collect samples sequentially both before and after induction of inflammation in order to gain predictive and diagnostic information about GI inflammation in horses. This approach will have broad application to many equine intestinal disorders and will provide the much needed mechanistic insight into disease development and progression, thereby enabling us to develop effective prevention and treatment strategies for equine GI disease.

The total cost of this study is $46,756 in 2019 and $45,814 in 2020. The EMRC recommends $12,000 for funding this study.

Paraoxonase-1 Activity as a Marker for Diagnosis of Equine Metabolic Syndrome Gabriele Rossi, DVM, Ph.D., DECVCP Murdoch University

Summary: Researchers will investigate a new laboratory test to improve diagnosis of equine metabolic syndrome, a metabolic and hormonal disorder in horses.

Description: Equine metabolic syndrome (EMS) is characterized by abnormal insulin regulation, obesity, and susceptibility to laminitis. EMS is challenging to diagnose since laboratory tests fail to distinguish EMS from other common equine diseases. Human metabolic syndrome (HMS) shares similar laboratory features to EMS and a new serum biomarker has been proposed in human medicine to improve the diagnosis of HMS. Researchers will investigate if this new HMS test also can provide an accurate diagnosis for horses with EMS. A new efficient and cost-effective EMS test will help veterinarians better diagnose and manage this complex disease in horses.

The total cost of this study is $9,355. The EMRC recommends $9,355 to fund this study.

Cell-mediated immunogenicity and MHC expression regulation of TGF-beta2-treated mesenchymal stem cells for improved clinical use in the horse. Lauren V Schnabel, DVM, Ph.D., DACVS, DACVSMR North Carolina State University

Summary: Researchers will manipulate the expression of immune markers on stem cells to develop safer and more effective therapies for horses with musculoskeletal injuries.

Description: Stem cell therapies have the potential to improve the outcome of potentially severe and life-ending musculoskeletal injuries in horses. However, sometimes the recipients immune system will destroy transplanted stem cells if the immune system perceives the introduced cells as foreign. To improve stem cell treatment success, researchers will examine if a novel cell culture treatment can be used to prevent the immune system from destroying donor stem cells. This study will help researchers better understand the cellular regulation of the molecules that initiate immune responses and advance the development of convenient and effective off-the-shelf stem cell therapy.

The total cost of this study is $131,479. The EMRC recommends $12,000 to fund this study. This is the second year that we are recommending funding this 3 year study.

Stephen Teichman Study

The goal is to determine the effects of front shoe surface modifications in the sport horse on the duration of breakover. We discussed and agreed to support this study during the May BOG conference call providing the publishing and professional fees were researched and found to be reasonable, which they were.

The total cost of this study is $9,640. The EMRC recommends USEA to provide $4,820 and an additional $4,820 is to be contributed by other donors.

This brings the total recommended research total to $38,175, leaving $1,406 of use at another time.

For an update on studies previously selected for funding by the USEA Equine Medical Research Committee, click here. To learn more about how the Equine Medical Research Fund got started, click here. For additional information about the Fund, click here.

Link:
Equine Medical Research Funding Receives USEA Board of Governors - United States Eventing Association

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (Rocket), a leading U.S.-based multi-platform gene therapy company, today announces data presentations at the upcoming European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress taking place October 2225, 2019, in Barcelona, Spain. Presentations at this years meeting include four oral presentations and one poster presentation related to Rockets lentiviral pipeline programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD), Pyruvate Kinase Deficiency (PKD), and Infantile Malignant Osteopetrosis (IMO).

Details for Rockets oral and poster presentations are as follows:Title: Towards Haematopoietic Stem Cell-Targeted Gene Therapy of Infantile Malignant OsteopetrosisSession Title: Skeletal Muscle & Bone Gene TherapySession Date: Wednesday, October 23, 2019Session Time: 5:30 PM 7:30 PM CESTRoom: 116-117

Title: Gene Therapy for Patients with Fanconi AnaemiaSession Title: Gene Therapy Clinical Trials IISession Date: Thursday, October 24, 2019Session Time: 8:30 AM - 10:30 AM CESTRoom: 113-117

Title: First Steps of a Lentiviral Gene Therapy Clinical Trial for Pyruvate Kinase DeficiencySession Title: Blood DiseasesSession Date: Thursday, October 24, 2019Session Time: 2:45 PM - 4:45 PM CESTRoom: 211

Title: Broad Applicability of NHEJ-Mediated Gene Editing to Correct Mutations in a Variety of Fanconi Anaemia GenesSession Title: New Approaches in Gene EditingSession Date: Friday, October 25, 2019Session Time: 9:00 AM - 11:00 AM CESTRoom: 113-115

Title: Stable Transduction of Long-Term HSCs Under Optimized GMP-Conditions for the Gene Therapy of LAD-I PatientsSession Title: Poster Session IISession Date: Thursday, October 24, 2019Session Time: 1:15 PM - 2:45 PM CESTPoster Number: P228

Full results from the ESGCT presentations will be available online at the conclusion of the presentation: https://www.rocketpharma.com/esgct-presentations/

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's first two clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO). For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Upcoming Presentations at the European Society of Gene and Cell Therapy Annual Congress - Business Wire