Stem Cell Clinic

AIVITA Biomedical to Present at Upcoming Regenerative Medicine, Oncology and Investor Conferences in November – PRNewswire

IRVINE, Calif., Nov. 1, 2019 /PRNewswire/ --AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, today announced that it will be presenting at the following regenerative medicine and investor conferences in November:

Society for the Immunotherapy of Cancer (SITC) Annual MeetingOral PresentationPresenter: Dr. Daniela Bota, MD, PhD, University of California, Irvine; AIVITA GBM Principal InvestigatorTitle: Phase II trial of therapeutic vaccine consisting of autologous dendritic cells loaded with autologous tumor cell antigens from self-renewing cancer cells in patients with newly diagnosed glioblastomaTime: November 6-10, 2019Location: Gaylord National Hotel & Convention Center, National Harbor, MD

The Regenerative Medicine Consortium of the Gulf Coast Consortia for Biomedical SciencesOral Presentation Presenter: Dr. Hans S. Keirstead, AIVITA Chairman and CEOTitle: Clinical and Commercial Application of Scaled Human Stem Cell DerivatesTime: November 8, 4:00 PM CTLocation: Bioscience Research Collaborative, Houston, TX

NYC Oncology Investor ConferenceOral Presentation Presenter: Dr. Hans S. Keirstead, AIVITA Chairman and CEO Title: AIVITA Corporate PresentationTime: November 12, 4:50 PM - 5:10 PMLocation: Rockefeller Center, New York, NY

Society for NeuroOncology Annual MeetingPoster PresentationTitle: Phase II trial of AV-GBM-1 (autologous dendritic cells loaded with autologous tumor associated antigens) as adjunctive therapy following primary surgery plus concurrent chemoradiation in patients with newly diagnosed glioblastoma.Time: November 20-24, 2019Location: JW Marriott Desert Ridge, Phoenix, AZ

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. All proceeds from the sale of AIVITA's skin care products support the treatment of women with ovarian cancer.

SOURCE AIVITA Biomedical, Inc.

Homepage

Read the original:
AIVITA Biomedical to Present at Upcoming Regenerative Medicine, Oncology and Investor Conferences in November - PRNewswire

Status of Proteins Housing DNA Controls How Cells Maintain their Identity – PRNewswire

NEW YORK, Oct. 31, 2019 /PRNewswire/ -- The inheritance, not only of DNA, but of changes to proteins that package it, maintains the identity of cells as they multiply, a new study finds.

Led by researchers at the NYU School of Medicine, the study sheds light on the process by which each cell transmits its identity to the next cellular generation as it multiplies by dividing in two during development. All cells have the same, complete set of DNA, but each is programmed to activate or silence certain genes, determining whether they become, for instance, heart cells instead of intestinal cells, researchers say.

Published online on October 31 in Cell, the new study confirms that mechanisms preserving cell identity are based on how DNA is packaged. With the nuclei of cells, chains of molecular DNA are known to be wrapped around sets of proteins called histones in repeating units called nucleosomes, all within the larger superstructure called chromatin.

Further, the "tails" of the histones are known to extend outside of this wrapping, making them available for chemical changes that determine whether a chromatin region is "open," with the DNA accessible, ortightly compacted, with the genes residing there silenced. The current study authors devised a method to track whether chemical modifications to histones in nucleosomes were accurately passed from a parent cell to the same DNA regions in the two cells it forms upon division.

"Based on our new method, experiments suggested that compaction based on histone modifications at key positions is a fundamental feature of inheritance in cells, with the related silencing passed on to keep genes from being expressed in the wrong context," says study first author Thelma Escobar, PhD, a postdoctoral scholar in the Department of Biochemistry and Molecular Pharmacology at NYU School of Medicine.

Mapping Inherited Nucleosome Changes

The research team developed a system in which "old" nucleosomes, those positioned throughout a parent cell's DNA, could be tagged before the DNA was copied as a prerequisite to cell division.

Researchers then used the specificity of the CRISPR-Cas9 gene editing system to attach a molecule called biotin but only to those nucleosomes present within a single gene of their choice. That in turn let them follow the fate of these marked nucleosomes in genes known to occur in either open or silent chromatin as the cell copied its DNA.

The new method revealed that only the nucleosomes from silent, compacted chromatin were inherited ending up at the same locale on the newly replicated DNA as they were in the parent DNA. The nucleosomes from the open chromatin were not inherited, and were distributed widely throughout the newly copied chromatin.

Based on their experiments, the authors argue that chemical modifications to histones that foster chromatin compaction are inherited as a fundamental property preserving a cell's identity. Open chromatin, on the other hand, did not rely on this "positional inheritance," as its DNA can be activated as needed by regulatory proteins that interacted with the DNA directly.

Furthermore, the research team found that only within compacted, silent chromatin could the proteins responsible for chemically modifying histone tails recognize ("read") modifications in parental nucleosomes and then duplicate ("write") them on newly forming nucleosomes.

The study also suggests that histones forming nucleosomes in silent chromatin may be escorted to the proper place in replicating DNA by novel chaperone proteins, which the researchers are currently seeking to identify.

"Strikingly, it may be through gene repression that histone modifications show a heritable gene regulatory system not present in more primitive cells like bacteria, potentially playing a role in the greater complexity of human cells," says senior study author Danny Reinberg, PhD, the Terry and Mel Karmazin Professor of Biochemistry and Molecular Pharmacology at NYU Langone Health. "At the same time, cancer arises in some cases from this very complexity as cells that randomly lose compaction due to a loss of histone chemical modifications see their usually silenced genes become active."

"In this way, cancer cells evolve to overcome treatments or limits on their growth, and we hope the current work will suggest new ways to stop this," says Reinberg, also an investigator with the Howard Hughes Medical Institute.

Along with Escobar and Reinberg, study authors from NYU Langone School of Medicine were Ozgur Oksuz, Ricardo Saldaa-Meyer, Nicolas Descostes, and Roberto Bonasio. Funding for the work was provided by the Howard Hughes Medical Institute and the National Cancer Institute grants 9R01CA199652-13A1, 3R01CA199652-14S1, and P30CA016087.

Contact: Gregory Williams, 212-404-3500, gregory.williams@nyumc.org

SOURCE NYU School of Medicine

https://med.nyu.edu/

Visit link:
Status of Proteins Housing DNA Controls How Cells Maintain their Identity - PRNewswire

Opportunities and Challenges in Cell and Gene Therapy Development – Genetic Engineering & Biotechnology News

Ger Brophy, PhDExecutive Vice PresidentBiopharma ProductionAvantor

Genuine progress is being made in the longstanding battle to effectively treat and control cancer. The National Cancer Institute projects that nearly five million more U.S. citizens are expected to survive cancer in 2029 than in 2019.1 Therapeutic tools such as next-generation sequencing and advances in immunotherapy are just two ways that fundamental scientific breakthroughs and innovative thinking are realizing the potential for new cancer treatments.

One of the most revolutionary breakthroughs in this new era is cell and gene therapy. At its most basic definition, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patients cells as a drug to treat disease. According to a paper published in the Journal of Gene Medicine, somewhere around 2600 gene therapy clinical trials had been undertaken in 38 countries around the world as of November 2017.2

These clinical trials demonstrate that the recent attention being paid to gene and cell therapy is not just hype. Some have noted that a select number of approved cell and gene therapies are for relatively small patient groups. However, its exciting to see the number of trials grow, especially when one considers this technologys ability to impact patients lives.

Its true that the number of patients receiving treatment is relatively small compared to other therapeutic regimens, but thats to be expected. Many of the biopharmaceutical researchers and manufacturers started with smaller, defined patient populations and, in particular, those with pediatric relapse refractory acute lymphoblastic leukemia. In part, these early efforts were directed at this type of cancer because the researchers wanted to deal with small populations that they understood well and, in many cases, had few or no other options for treatments.

The success of these initial efforts has led to broader programs targeting larger populationsstarting with leukemia and lymphoma. Ultimately, the most challenging opportunityand the one with the greatest potential for beneficial outcomesis multiple myeloma. If these patients begin to see benefits from cell and gene therapies, it will justify the incremental approach the industry has been taking.

The genuine, almost unprecedented potential for cell and gene therapy cannot be understated. For the first time, people are talking about curing these ruthless, relentless diseases. In a way never before possible, were taking control of and harnessing the patients own immune system to fight these cancers. Among the first patients treated for acute lymphoblastic leukemia, several are alive and thrivingfour, five, and six years later.

The game changer here is that cell and gene therapy uses the bodys own systems, either the cellular immune system or the ability to repair and replace defective or missing genes. CAR T-cell therapy is arguably among the most personalized medicines one can consider. The patients own T cells are extracted, modified, activated, expanded, purified, and returned to the patient.

Significant growth is underway in the size and sophistication of companies and organizations entering the cell and gene therapy markets. Many of the early movers in cell and gene therapy were small biotech startups. In some cases, their treatments were supported by major hospital centers.

Increasingly, weve all seen a greater interest from the major biopharma companies. Novartis was probably the biggest; it started earliest and was successful in getting approval for Kymriah. Since last year, weve seen several important acquisitions by Gilead and Bristol-Myers Squibb, and major biopharma companies are participating in large strategic partnerships in China. As companies of this size get involved, the hope is that they will leverage their increased breadth and depth to develop novel products, instigate new trials, and find ways to manufacture therapies at scale.

If the cell and gene therapy industry is to succeed, it must overcome challenges of two kinds: scalability and manufacturability. These challenges may be summarized in a set of questions: Can we manufacture cell and gene therapies at scale? If we can manufacture these treatments at scale, then can we do so safely? Can we do so at a reasonable cost so the populations that are affected by these diseases can access treatments?

With cell therapy, the single biggest point of variability is the patients own cells. By their very nature, these cells are individual to the patient, and their health implications for the patient should be considered as an integral part of cell processingat least as far back as the time of leukapheresis.

Variables and failure modes must be taken out of cell processing systems. We can standardize and miniaturize these systems, and we can enclose them so that theyre not exposed to failure modes. Also, we can improve technologies, like sterile fluid transfer, if we use excipient technology to further stabilize production. Finally, we can use analytical technology to understand the factors that contribute to a therapys success or failure.

Cell therapy producers and the companies that support and supply them need to become more innovative. In areas such as cell culture components, production chemicals, single-use technologies, sterile fluid transfer, and excipientsand the technology surrounding those process componentsthere is value to improving collaboration and trying new solutions to address the issues of manufacturability and scale.

We need to better analyze and understand the variability that comes from the research data, even at the early stages of these trials, and use it to correlate with clinical and process outcomes. Taking out manual steps as early as possible is important, as well as creating closed systems using sterile fluid transfer technologies.

One of the most significant challenges is finding solutions around side effects. As we understand how to provide a more efficacious dose, perhaps using fewer cells, some of the side effects of these drug therapies may improve. Furthermore, we must find scalable ways to reduce costs.

Ultimately, these drugs must be developed in a more cost-effective manner. Thats an area where technology providers and suppliers can play a significant role, by closing and automating systems and by understanding the contribution of labor and overhead and possible economies of scale from reducing processes.

There have been encouraging improvements in the way various regulatory groups have supported gene and cell therapy. To a certain degree, groups representing different regionsNorth America, Europe, and Asiahad been perceived to be setting precedents independently. More recently, it appears that regulatory bodies have been very open and collaborative in acknowledging that cell and gene therapies differ from more mature treatments such as biopharma drugs for cancer. The regulatory bodies have shown that they are willing to put the appropriate regulatory system into place to streamline the approval process and institute the ongoing monitoring of cell and gene therapies.

The U.S. FDAs support on CAR T-cell technologies is a good example. Regulators are allowing flexibility in the normal hierarchy of how clinical trials are performed, particularly in Phase II and III trials, but the companies must still address the FDAs postmarketing comments and safety issues.

Some have suggested that, ultimately, almost all cancer treatments will be based on gene and cell therapy approaches since they represent the most personalized form of treatment, which is, theoretically, the one with the highest potential for success.

Thats probably overly ambitious. Both large molecules and small molecules will continue to provide trusted, effective solutions with each type of drug product finding its niche. For example, large molecules are being developed for areas like neurodegeneration and are still offering great potential.

Its worth remembering that monoclonal-based therapies and biopharmaceuticals have really only started to make a significant impact in the last 15 to 20 years. Cell and gene therapies are just starting and have yet to make a significant market impact. But considerable effort is going into developing, understanding, and characterizing drug targets, as well as the development of technology to make targeted drugs in production-level volumes.

All these developments are exciting and offer a great deal of hope. It is clear that gene and cell therapies work and save lives. The challenge now is to scale their production. It is also clear that cell and gene therapies can emulate other therapeutic approaches that have transitioned from theoretical possibility to practical reality. As a similar transition occurs for cell and gene therapies, the production issues that need to be addressed will be seen more clearly, prompting action that will bring us to the next stage of development.

References1. Bluethmann SM, Mariotto AB, Rowland, JH. Anticipating the Silver Tsunami: Prevalence Trajectories and Comorbidity Burden among Older Cancer Survivors in the United States. Cancer Epidemiol. Biomarkers Prev. 2016; 25: 10291036.2. Ginn SL, Amaya AK, Alexander IE, et al. Gene therapy clinical trials worldwide to 2017: An update. J. Gene Med. 2018; 20(5): e3015.

Ger Brophy, PhD, is Executive Vice President, Biopharma Production at Avantor

Follow this link:
Opportunities and Challenges in Cell and Gene Therapy Development - Genetic Engineering & Biotechnology News

Tmunity Raises $75 Million in Series B Financing to Advance Next-Generation T-Cell Therapies – Business Wire

PHILADELPHIA--(BUSINESS WIRE)--Tmunity Therapeutics, in pursuit of its vision to save and improve lives by delivering the full potential of next-generation T-cell immunotherapy, closed a $75 million Series B financing. The financing was led by Andreessen Horowitz (also known as "a16z"), a venture capital firm that backs bold entrepreneurs building the future through technology and includes participation from a16zs Cultural Leadership Fund. Joining the Series B financing are Westlake Village BioPartners, Gilead Sciences, The University of Pennsylvania, Be The Match BioTherapies and BrightEdge, the philanthropic impact fund of the American Cancer Society.

The proceeds from the Series B will continue to fund ongoing and planned research, clinical development of product candidates, the continued build-out of the Companys proprietary, vertically-integrated viral vector and cell therapy product manufacturing, working capital and other general purposes. Since inception, Tmunity has raised $231 million.

We are fortunate to be funded by impressive investors who share our commitment to patients and our vision to dramatically change the way cancer is treated, said Usman Oz Azam, MD, President and Chief Executive Officer of Tmunity. We see ourselves leading the innovation of the future of oncology treatment by uniting our foundational competences in cell therapy with expertise in building new constructs, translating them and getting them into the clinic.

As part of the Series B financing, Jorge Conde, General Partner at a16z, will join the Companys Board of Directors. Mr. Conde leads a16zs investments that are at the cross section of biology, computer science and engineering.

"To win the war on cancer, we need smarter weapons. Tmunitys founders Carl June and Bruce Levine invented CAR-T, one of the most profound breakthroughs against cancer in recent history. Together with Oz Azam, who with his team, brought the first CAR-T therapy to market, the company has built a pioneering platform that has produced an unrivaled therapeutic pipeline with programs already in human clinical trials for both solid and liquid tumors. This is the dream team to deliver on the bold and promising mission to cure disease using engineered T-cells," said Conde.

About Tmunity Therapeutics

Tmunity is a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy to patients with devastating diseases. Integrating a foundational collaboration with the University of Pennsylvania (Penn) with the groundbreaking scientific, clinical, and manufacturing expertise, and the demonstrated track record of its founders (Carl June, MD; Bruce Blazar, MD; Bruce Levine, PhD; Yangbing Zhao, MD, PhD; Jim Riley, PhD; and Anne Chew, PhD), Tmunity represents a new center of gravity in translational T-cell medicine. Through the University of Pennsylvania, the Parker Institute for Cancer Immunotherapy and collaborations with the University of California San Francisco and Childrens Hospital of Philadelphia, the company is developing a diversified portfolio of novel treatments that exhibit best-in-class control over T-cell activation and direction in the body, with a focus in cancer and three programs currently in clinic development. With headquarters in Philadelphia, Tmunity utilizes laboratories and production facilities at Penn and its own dedicated cGMP manufacturing facility in East Norriton, PA, to pursue process improvement and production scale-up in support of clinical development of its T-cell therapies. For more information, visit http://www.tmunity.com and connect with us on social media at @TmunityTx and LinkedIn.

See more here:
Tmunity Raises $75 Million in Series B Financing to Advance Next-Generation T-Cell Therapies - Business Wire