New York, Aug. 24, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Therapy Packaging Products and Services Market by Type of Therapy, Package Engineering Design, Scale of Operation and Geography : Industry Trends and Global Forecasts, 2021-2030" - https://www.reportlinker.com/p06130493/?utm_source=GNW Presently, more than 30 cell-based therapy products have received marketing approvals across various geographies, while more than 1,000 therapeutic candidates are under clinical evaluation. Considering the lucrative investment opportunity within this domain, several investors are actively supporting ongoing / future product development programs in this upcoming field of pharmacological interventions. In fact, there has been a 240% increase in investment activity, in terms of capital amount invested, between 2019 and 2020. However, the ultimate success of cell therapies is dependent on the safe and timely delivery of viable doses of therapeutic cells to the right patient. In this context, it is worth highlighting that drug product manufacturing, packaging and logistics operations involving cell therapies are both complex and challenging. Moreover, in order to ensure the stability of such products across the supply chain, specific temperature conditions (cryogenic, ambient or refrigerated) are required, in addition to specialized packaging material. Considering that most cell therapies are personalized, it is imperative that errors in labeling are avoided at all costs.

Given the need for costly and specialized equipment and the complexities associated with handling cell-based therapy products, innovators in the healthcare industry are likely to involve contract service providers for their drug product production, packaging and transportation requirements. Over time, regulators across the world, including the US Food and Drug Administration (FDA) and the International Air Transport Association (IATA), have established guidelines for the packaging, storage and transport of cell therapies. Since CMOs / CDMOs with the necessary capabilities to handle cell therapies are also likely to have established processes that comply with the current acceptable standards across different global regions, it is better for therapy developers to strategically partner with the aforementioned players instead of focusing on developing in-house capabilities. Presently, close to 60 companies claim to be offering cell therapy packaging services and associated products. In fact, it is estimated that close to 30% of drug substance and drug product related operations of advanced therapy medicinal products are already outsourced to capable CMOs / CDMOs. Further, over the last few years, several service providers have also forged strategic alliances among themselves, in order to further expand existing capabilities and augment their respective service offerings. Given the rising trend of outsourcing in the healthcare industry, and the ongoing efforts of service providers to further improve their portfolios, we believe that the cell therapy packaging products and services market is likely to evolve at a steady pace in the next few years.

SCOPE OF THE REPORT The Cell Therapy Packaging Products and Services Market by Type of Therapy (T-cell Therapies, Dendritic Cell Vaccines, Stem Cell Therapies, NK Cell Therapies and Other ATMPs), Package Engineering Design (Primary and Secondary Packaging), Scale of Operation (Clinical and Commercial) and Geography (North America, Europe, Asia Pacific and Rest of the World) , 2021-2030 report features an elaborate study of the current scenario and future opportunity within the cell therapy packaging products and services market. In addition, the report features an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. Amongst other elements, the report includes: An overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis based on their year of establishment, company size, location of headquarters, package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen), type of passive system used, temperature ranges supported, type of cells handled and additional services offered. A detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players. An in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies). An analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership. A list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate). A case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies. Detailed profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

One of the key objectives of the report was to estimate the existing market size and future growth potential of the cell therapy packaging providers market. We have provided informed estimates on the likely evolution of the market in the short to mid-term and long term, for the period 2021-2030. Our year-wise projections of the current and future opportunity have further been segmented across [A] type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), [B] package engineering design (primary and secondary packaging), [C] scale of operation (clinical and commercial) and [D] geography (North America, Europe, Asia Pacific and Rest of the World)

All actual figures have been sourced and analyzed from publicly available information forums. Financial figures mentioned in this report are in USD, unless otherwise specified.

KEY QUESTIONS ANSWERED Who are the leading players offering cell therapy packaging services and products? What are the key considerations and challenges associated with cell therapy packaging? Which partnership models are commonly adopted by stakeholders engaged in this industry? Which players are likely to partner with cell therapy packaging service and product providers? What factors are likely to influence the evolution of the cell therapy packaging service providers market? What are the likely future trends in cell therapy packaging services market? How is the current and future opportunity likely to be distributed across key market segments?

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary research. For all our projects, we conduct interviews with experts in the area (academia, industry and other associations) to solicit their opinions on emerging trends in the market. This information is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been validated from multiple sources of information.

The secondary sources of information include: Annual reports Investor presentations SEC filings Industry databases News releases from company websites Government policy documents Industry analysts views

While the focus has been on forecasting the market till 2030, the report also provides our independent views on various non-commercial trends emerging in this industry. The opinions are solely based on our knowledge, research and understanding of the relevant market trends gathered from various secondary sources of information.

CHAPTER OUTLINES Chapter 2 is an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the cell-based therapy manufacturing market and its likely evolution in the short to mid-term, and long term.

Chapter 3 provides a general introduction to the pharmaceutical filling and packaging, including information on types of pharmaceutical packaging. The chapter lays emphasis on cell therapy packaging, primary and secondary packaging of cell therapies. In addition, it discusses the key considerations and challenges associated with cell therapy packaging.

Chapter 4 features an overview of the current market landscape of cell therapy packaging services providers, including a detailed analysis on the year of establishment, company size and location of headquarters. In addition, the chapter highlights the analysis on the package engineering design (primary and secondary), type of packaging (active and passive), packaging material (dry ice and liquid nitrogen) and type of passive system used. Further, the chapter also presents analysis on temperature ranges supported, type of cells handled and additional services offered.

Chapter 5 fpresents a detailed assessment of the current market landscape of cell therapy packaging products, providing information on type of packaging container (bags, vials, shipping container and others), container fabrication material (plastic and others), storage temperature conditions, type of cells packed and usability of containers. In addition, the chapter highlights analysis of cell therapy packaging product provider(s), based on various parameters such as year of establishment, company size and location of headquarters and key players.

Chapter 6 includes profiles of the key players offering cell therapy packaging services and products. Each profile includes a brief overview of the company, details on cell therapy service portfolio, information on cell therapy packaging product portfolio, recent developments and an informed future outlook.

Chapter 7 presents in-depth competitiveness analysis of cell therapy packaging service providers based in different geographies, by taking into consideration the service strength of a company (based on its experience), packaging portfolio (package engineering design, type of packaging, type of packaging material and temperature ranges supported) service portfolio (type of additional services offered) and company size (small, mid-sized and large companies).

Chapter 8 provides an analysis of the various partnerships established between cell therapy packaging providers and cell therapy developers, during the period, 2016-2021. It includes a brief description of various types of partnership models (namely service alliance, technology / platform integration agreement, merger and acquisition) adopted by stakeholders engaged in this domain. It is worth mentioning that the data captured during our research was analyzed based on several parameters, such as year of partnership, type of partnership, purpose of partnership, type of service covered and geographical location of players involved in a partnership.

Chapter 9 presents a list of more than 250 cell therapy developers anticipated to partner with cell therapy packaging providers, which have been shortlisted on the basis of developer strength (based on company size), type of therapy and pipeline maturity (based on stage of development of drug candidate).

Chapter 10 features a case study highlighting the companies that claim to have the required expertise and capabilities for development and manufacturing of cell therapies, along with the information on their year of establishment, company size, location of headquarters and location of manufacturing facility. In addition, the chapter presents analysis on type of cells manufactured, source of cells, scale of operation and manufacturing capabilities / services of the aforementioned companies.

Chapter 11 presents a detailed market forecast analysis, providing insights on the likely growth of continuous manufacturing equipment providers market for the period 2021-2030. In addition, the chapter presents the likely distribution of the projected future opportunity based on type of therapy (T-cell therapies, dendritic cell vaccines, stem cell therapies, NK cell therapies and other advanced therapy medicinal products), package engineering design (primary and secondary packaging), scale of operation (clinical and commercial) and geography (North America, Europe, Asia Pacific and Rest of the World).

Chapter 12 is a summary of the overall report, which presents the insights on the contemporary market trends and the likely evolution of cell therapy products and services market.

Chapter 13 is an appendix that contains tabulated data and numbers for all the figures provided in the report.

Chapter 14 is an appendix that provides the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06130493/?utm_source=GNW

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Cell Therapy Packaging Products and Services Market by Type - GlobeNewswire

Shoreline Biosciences, an immunotherapy company formed in 2020 during the early days of the COVID-19 pandemic, was propelled into the proverbial catbirds seat this summer with two mega-deals, and now plans to double its employee base from 50 to 100 in the coming six to nine months.

Shoreline Biosciences is developing an off-the-shelf, targeted, allogeneic approach to natural killer (NK) and macrophage cell therapies, which Kleanthis G. Xanthopoulos, Ph.D., chairman and CEO, believes is one-of-a-kind.

This summers deals with Kite Pharmaceuticals, a Gilead Company, and BeiGene totaling $4 billion and boasting combined upfront payments of over $120 million to further develop its induced pluripotent stem cell (iPSC) programs are potent validation of Shorelines technology as well as its management. They go a long way toward ushering in what Xanthopoulos called the era of cell therapy.

Shorelines growing success is based on two pillars: solid science and experienced management.

You have to have the high science, but also enormous institutional know how, Xanthopoulos told BioSpace. These cells are finicky. There are significant challenges in manipulating them. Our founders have 20 years experience in this field.

BeiGene and Kite share our vision, and have extensive experience themselves BeiGene is incredible in protein engineering and Kite pioneered CAR T therapies, he said. So, when they chose to partner with Shoreline, they had performed competitive analysis and were saying, in essence, we believe in you and your capabilities.

These partnerships allow Shoreline to fast-forward its platforms.

For a company to bring so much capital and synergy to work is incredibly powerful, unique, and differentiated, Xanthopoulos said. It is difficult to find another early-stage preclinical company that has this kind of recognition and validation.

The new clinical data from Fate Therapeutics regarding its NK and T cell therapies help the field, too.

The science is very comprehensive, and we believe it will bring those programs forward, renewing interest in pluripotent stem cells and NK cells, Xanthopoulos surmised.

Shoreline Biosciences is developing off-the-shelf allogeneic therapies, bringing the benefit of stem cell therapies to many more patients than is possible with autologous transplants.

For autologous therapies, Xanthopoulos explains, It is costly to take patient cells, manipulate them in the lab, add guided chimeric antigen receptors, and return them to the patients There also are issues with rejection, as well as the more significant issues of cytokine release syndrome. Patients with aggressive tumors dont have the four to six weeks that method requires.

Shorelines allogeneic approach, therefore, creates targeted, off-the-shelf stem cell therapies.

The only effective way to do this is to start with pluripotent stem cells that can differentiate into more than 200 cell types, Xanthopoulos said. At Shoreline, we differentiate into hemopoietic cells. We are focused on NK cells and macrophages.

We have, basically, released the brakes for proliferating and activating the NK cells, he said, by editing out a negative regulator of activation and proliferation to create what essentially is a supercharged cell that is more metabolically fitted.

Preclinical studies in animals show the cells resist exhaustion, conferring a better pharmacokinetic profile and the need for fewer cytokines. Importantly, those efforts have been validated by independent researchers, in many published papers.

Results indicate the combination of pluripotent stem cells and NK cells results in a greater ability to kill various tumor cells using 5- to 10-fold less IL2 and IL15. Consequently, Xanthopoulos said, The overall therapy is less expensive.

Shoreline Biosciences plans to take this program into early human trials in the second half of 2022.

In 2023, we expect to file one or two Investigational New Drug (IND) applications, he said.

The benefit of an allogeneic iPSC approach to therapeutics development is clear.

You can perform a lot of genomic edits at the pluripotent state without a great many technical problems, he noted.

For example, transducing macrophages with chimeric antigen receptors (CAR) requires specialized vectors and results in low yields. But, if you start with pluripotent stem cells, you can make the modifications, isolate a single clone with the characteristics you want, and generate trillions of cells. With one patient dose requiring approximately 100 million cells, this method can lower the approximate $400,000 cost of therapy substantially.

To do this for NK cells, Shoreline Biosciences created a unique, powerful engine and then determined how to decorate the resulting cells. After considering such issues as cell signaling, targeting, and toxicity, Xanthopoulos said, We engineered what you can think of as hooks where antibodies recognize and interact tightly, for higher killing activity.

As Xanthopoulos, a serial entrepreneur who has founded and operated four previous biotech companies, told BioSpace, Ive never been more excited. Stem cell therapies are proven. They are the next frontier of medicine.

They arent in the distant future, either. I think the era of cell therapies is here and now, and will dominate the landscape going forward. If we learn to make less immunogenic therapies, stem cell therapeutics will continue to increase momentum.

As Shoreline looks to the future, it is building an expansive team and is continuing to hire in all categories. We plan to double to about 100 employees in the next six to nine months, and have several outstanding investors onboard.

Excerpt from:
Shoreline Launches into "Era of Cell Therapy" with $4 Billion in Partnerships - BioSpace

This article was originally published here

Ann Med. 2021 Dec;53(1):1547-1559. doi: 10.1080/07853890.2021.1970218.

ABSTRACT

BACKGROUND: Chimeric antigen receptor (CAR) T-cell therapy shows impressive results in clinical trials. We conducted a meta-analysis based on the most recent data to systematically describe the efficacy and safety of anti-BCMA CAR T therapy for patients with relapsed or refractory multiple myeloma (R/R MM).

METHODS: PubMed, Embase, Web of Science, Cochrane library, ClinicalTrials.gov, China Biology Medicine disc (CBM disc) and Wanfang Data were searched on 8 November 2020. Registration number of PROSPERO was CRD42020219127.

RESULTS: From 763 articles, we identified 22 appropriate studies with 681 patients. The pooled overall response rate (ORR) was 85.2% (95%CI 0.797-0.910), complete response rate (CRR) was 47.0% (95%CI 0.378-0.583), and minimal residual disease (MRD) negativity rate was 97.8% (95%CI 0.935-1.022). The pooled incidence of grade 3-4 cytokine release syndrome was 6.6% (95%CI 0.036-0.096) and neurotoxicity was 2.2% (95%CI 0.006-0.038). The median progression-free survival (PFS) was 14.0 months and median overall survival (OS) was 24.0 months. Subgroup analysis showed dual epitope-binding CAR T cells achieved the best therapy outcomes and humanized CAR T cells had the best safety profile. Patients who were older, heavily pre-treated or received lower dose of CAR T cells had worse ORR. There was no significant difference in ORR, CRR and PFS between patients with and without high-risk cytogenetic features. The PFS and CRR of non-extramedullary disease (EMD) group was superior to those of EMD group.

CONCLUSION: Anti-BCMA CAR T therapy is effective and safe for patients with R/R MM. It can improve the prognosis of patients with high-risk cytogenetic features while the prognosis of patients with EMD remains poor. Moreover, patients are likely to benefit from an earlier use of CAR T therapy and human-derived CAR T cells have obvious advantages based on the existing data.

PMID:34459681 | DOI:10.1080/07853890.2021.1970218

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Comprehensive meta-analysis of anti-BCMA chimeric antigen receptor T-cell therapy in relapsed or refractory multiple myeloma - DocWire News

SAN JOSE, Calif., Aug. 30, 2021 /PRNewswire/ -- Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer and infectious diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for its CAR-T (Chimeric Antigen Receptor-T cell) therapy, which is being developed in partnership with Moffitt Cancer Center (Moffitt).

Anixa Biosciences, Inc. (PRNewsfoto/Anixa Biosciences, Inc.)

Dr. Robert Wenham, Chair of Gynecological Oncology at Moffitt will lead the clinical trial.

This CAR-T therapy was developed by Dr. Jose Conejo-Garcia and his research team, when he was at the Wistar Institute. Dr. Conejo-Garcia is currently the Chairman of Immunology at Moffitt.

Anixa Biosciences has an exclusive worldwide license to the technology.

This technology is an autologous cell therapy. The therapeutic product is comprised of engineered T-cells that target the follicle stimulating hormone receptor (FSHR). FSHR is found at immunologically relevant levels exclusively on the granulosa cells of the ovaries. Since the target is a hormone receptor, and the target-binding domain is derived from its natural ligand, this technology is also known as CER-T (Chimeric Endocrine Receptor T-cell) therapy, a new type of CAR-T.

Dr. Amit Kumar, President and CEO of Anixa stated, "We are pleased that the FDA has authorized us to commence human clinical trials of our potentially paradigm-shifting therapy. Outcomes for women with late-stage ovarian cancer are poor, so if this approach demonstrates efficacy, it would have a dramatic impact on patients for whom there are no other options."

"This is a major milestone for our program. CAR-T therapy has had major successes in B-cell malignancies, but no clinical success in other types of tumors. We feel that our technology could be the first CAR-T approach that will demonstrate efficacy in a solid tumor indication, and we are looking forward to testing and verifying that assertion in human trials," said Dr. Wenham.

Story continues

In addition to Drs. Wenham and Conejo-Garcia, other key members of the team at Moffitt include:

Dr. Marco Davila, Clinician and Director of the Cell Therapy Facility at Moffitt

Dr. Daniel Abate Daga, Assistant Director of the Cell Therapy Facility at Moffitt

Dr. Ana Marie Landin, Experimental Cell Therapy Manager at Moffitt

Dr. Theresa Boyle, Department of Pathology at Moffitt

Carrie Thomas, FDA Regulatory Liaison at Moffitt

Tam Jackson, Project Management Consultant

Scientists and Members of the Moffitt Cell Therapy Facility

Members of the Moffitt Alliance Relations Team

About Anixa Biosciences, Inc.Anixa is a publicly-traded biotechnology company developing a number of programs addressing cancer and infectious disease. Anixa's therapeutics portfolio includes a cancer immunotherapy program which uses a novel type of CAR-T, known as chimeric endocrine receptor T-cell (CER-T) technology, and a Covid-19 therapeutics program focused on inhibiting certain viral protein function. The company's vaccine portfolio includes a vaccine to prevent breast cancer, and specifically triple negative breast cancer (TNBC), the most deadly form of the disease, and a vaccine to prevent ovarian cancer. These vaccine technologies focus on immunizing against specific proteins that have been found to be expressed in certain forms of cancer. Anixa continually examines emerging technologies in complementary fields for further development and commercialization. Additional information is available at http://www.anixa.com.

Forward-Looking Statements: Statements that are not historical fact may be considered forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not statements of historical facts, but rather reflect Anixa's current expectations concerning future events and results. We generally use the words "believes," "expects," "intends," "plans," "anticipates," "likely," "will" and similar expressions to identify forward-looking statements. Such forward-looking statements, including those concerning our expectations, involve risks, uncertainties and other factors, some of which are beyond our control, which may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements. These risks, uncertainties and factors include, but are not limited to, those factors set forth in "Item 1A - Risk Factors" and other sections of our most recent Annual Report on Form 10-K as well as in our Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. You are cautioned not to unduly rely on such forward-looking statements when evaluating the information presented in this press release.

Contact:Mike Catelanimcatelani@anixa.com408-708-9808

Cision

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SOURCE Anixa Biosciences, Inc.

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Anixa Biosciences and Moffitt Cancer Center Announce FDA Clearance to Initiate Clinical Trial of Ovarian Cancer CAR-T Immunotherapy - Yahoo Finance

The National Comprehensive Cancer Network (NCCN) has announced a significant update to the guidelines for COVID-19 vaccine administration, including a third dose, in patients with cancer, according to a press release issued by the organization.1

The recommendation indicated that several groups of individuals should be eligible for a third dose of the COVID-19 vaccine, including those with new or recurring solid tumors within 1 year of their initial vaccine dose regardless of therapy, as well as those with active hematologic malignancies. Patients who have received a stem cell transplant (SCT) or engineered cellular therapy such as CAR T-cell therapy within the past 2 years should also eligible, as well as those who are recipients of SCT on immunosuppressive therapy or with a history of graft-versus-host disease regardless of when the transplant took place.

COVID-19 can be very dangerous, especially for people living with cancer, which is why were so grateful for safe and effective vaccines that are saving lives, Robert W. Carlson, MD, chief executive officer of the NCCN, said in a press release. Our organization exists to improve the lives of people with cancer; we have a long track record for making recommendations that improve quality and length of life. We want our patients to live the longest and best lives possible, which means following the science on vaccination and mask-wearing.

The NCCN COVID-19 Vaccine Advisory Committee consists of multidisciplinary physicians across the NCCNs Member Institutions. In particular, the committee includes experts in infectious diseases, vaccine development and delivery, cancer management, and medical ethics. The recommendations, which are based on available evidence and expert consensus, have been utilized globally to aid in making management decisions over the course of the COVID-19 pandemic.

When it comes to peoples safety, we have to take every precaution, Steve Pergam, MD,

MPH, associate professor of the Vaccine and Infectious Disease Division at Fred Hutchinson

Cancer Research Center and infection prevention director at Seattle Cancer Care Alliance, as well as the co-leader of the NCCN COVID-19 Vaccination Advisory Committee, said in a press release. That means even after a third dose of vaccine, we still recommend immunocompromised peoplesuch as those undergoing cancer treatmentcontinue to be cautious, wear masks, and avoid large group gatherings, particularly around those who are unvaccinated. All of us should do our part to reduce the spread of COVID-19 and get vaccinated to protect those around us from preventable suffering.

Additional recommendations from the updated guidelines suggest that all eligible caregivers and close contacts of those with cancer should be immunized whenever possible.2 The use of all vaccines with FDA approval or emergency use authorizationincluding the Pfizer/BioNTech BNT162b2 mRNA vaccine (Comirnaty), the Moderna mRNA-1273 SARS-CoV-2 vaccine, and the Janssen Ad26.COV2.S Adenovirus vector vaccineis recommended in eligible patients by the committee.

Those who are receiving allogeneic or autologous SCT or CAR T-cell therapy will need to wait at least 3 months post-therapy before receiving the vaccine. Patients with hematologic malignancies, including those receiving cytotoxic chemotherapy such as cytarabine and anthracycline-based induction regimens for acute myeloid leukemia, need to delay inoculation until absolute neutrophil count recovery. Those who are experiencing marrow failure from their disease and/or are expected to have limited or no recovery from their therapy as well as those who are receiving long-term maintenance are able to receive the vaccine once it is available.

In solid tumors, the vaccine may be received once available for those who are undergoing treatment with cytotoxic chemotherapy, targeted therapy, checkpoint inhibitors, and other immunotherapy or radiation therapy. Those who are undergoing major surgery need to wait a few days following their surgical procedure before getting the vaccines.

The Center for Disease Control (CDC) currently recommends a third dose of the mRNA COVID-19 vaccines for individuals who are moderately to severely immunocompromised. This includes:

The CDC recommends that a third dose of the mRNA COVID-19 vaccine should be given at a minimum of 4 weeks following the second dose of either the Moderna or Pfizer vaccines. Notably, although preliminary data indicate that a third dose could augment antibody titers in an immunocompromised population of patients, those who have been diagnosed with cancer continue to be at a higher risk for infection with COVID-19 and COVID-19related complications. Even with the additional dose, infection is still possible meaning that precautions such as avoiding crowds and wearing a mask are recommended.

Read the rest here:
NCCN Issues Updated Guidelines Regarding Third COVID-19 Vaccine Booster for Those With Cancer - Cancer Network

Stem cells are divided into two major classes; pluripotent and multipotent. Pluripotent stem cells are replicating cells, which are derived from the embryo or fetal tissues. The pluripotent stem cells facilitate the development of cells and tissues in three primary germ layers such as mesoderm, ectoderm, and endoderm.

Increasing expansion of facilities by market players for stem cell therapies is expected to propel growth of the stem cell therapy market over the forecast period. For instance, in January 2018, the University of Florida, U.S. launched the Center for Regenerative Medicine that is focused on development of stem cell therapies for the treatment of damaged tissue and organ. The Centre for Regenerative Medicines is divided into two segments such as focus groups and shared services. Focus groups such as research and development activities for stem cell therapies; and the shared services segment offers technical resources related to stem cell therapies.

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Furthermore, rising collaboration activities by key players are expected to drive growth of the global stem cell therapy market. For instance, in May 2018, Procella Therapeutics and Smartwise, a medtech company entered into a collaboration with AstraZeneca Pharmaceuticals. Under this collaboration, AstraZeneca utilized Procella Therapeutics stem cell technology for the development of stem cell therapies in cardiovascular diseases. Moreover, in April, 2019, CelluGen Biotech and FamiCord Group collaborated to develop new stem cell-based drugs and advanced medical therapies (ATMP)

Major Company Profiles Covered in This Report:Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc., Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., Anterogen Co. Ltd., Pharmicell Co., Inc., and Stemedica Cell Technologies, Inc.

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Increasing research and development activities by the market players are one of the major factors that are expected to drive growth of the stem cell therapy market over the forecast period. For instance, in July 2018, the Emory Orthopaedics & Spine Center, in collaboration withSanford Health, Duke University, Andrews Institute, and Georgia Institute of Technology, received US$ 13 million grant from the Marcus Foundationfor a multicenter clinical trial studying stem cell options for treating osteoarthritis.The Phase 3 trial was initiated in March 2019, and is expected to complete by December 2021. Moreover, in December 2017, Mayo Clinic initiated the clinical phase I study on safety and feasibility of mesenchymal stem cell therapy in patients with intracerebral hemorrhage. This therapy is used for treating acute spontaneous hemorrhagic stroke. The study is expected to complete by December 2020.

Moreover, increasing product launches by key players is expected to boost demand for stem cell therapy over the forecast period. For instance, in August 2017, LifeCell, a healthcare service provider, launched an advanced umbilical cord-stem cell collection kit which offers enhanced protection to neonatal stem cells from environmental damage and temperature fluctuations.

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[PDF] Stem Cell Therapy Market Size Strong Revenue and Generated Opportunities UNLV The Rebel Yell - UNLV The Rebel Yell

Each week onDocWire News, editors bring you the latest news and research in hematology and oncology.In case you missed it, here are this weeks top headlines.

Editors spoke with Spiro Rombotis, CEO of Cyclacel Pharmaceuticals, Inc., a company developing innovative therapies that target various phases of cell control for treating advanced cancers. Mr. Rombotis discussed the strides his company is making in developing and testing targeted therapies that may actually drive cancer cells to self-destruct.

Essentially, when a cancer cell has progressed to the point that if the generated its code contains mistakes that can be communicated to daughter cells, many of the own circulatory mechanisms of the body are dysfunctional or absent, he explained. By inhibiting both CDK2 and CDK9, we can reinstitute these mechanisms, or programed suicide, and engage those anti-cancer mechanisms you want to stop working and induce the cancer cell to commit suicide.

Watch the full interview to learn more.

Spiro Rombotis, CEO of Cyclacel Pharmaceuticals, on Innovative Targeted Therapies for Advanced Cancer

Primary care providers (PCP) play a critical role in cancer diagnosis, according to a new study from researchers at the University of Michigan Rogel Cancer Center. The findings showed that the older women who reported feeling included in healthcare decisions with their PCP were more likely to be up to date on both colorectal and cervical cancer screenings.

When the researchers compared women who completed both screenings compared to those who had only one screening, they found that communication about health decisions with a PCP was still associated with being up to date on both screenings compared to one.

This creates a hypothesis that comprehensive cancer screening may best be accomplished through a PCP-patient relationship and not through targeted single cancer site screenings, said coauthor Diane M. Harper, MD, MPH.

Primary Care Providers Play Important Role in Cancer Screening Uptake

Patients with schizophrenia can and should participate in cancer screening to the same extent as the general population, with assistance, according to a study published in Acta Psychiatrica Scandinavica. In this first-of-its-kind study, researchers conducted a randomized trial to assess the benefits of interventions for early cancer screening in patients with schizophrenia.

The dissemination of this intervention into routine clinical practice may help bridge the gap between early detection of cancer and mortality across people with schizophrenia and the general population, said the study authors.

Intervention is Needed to Increase Screening for Early Cancer Screening in Patients with Mental Illness

Finally, for men with low-risk to favorable intermediate-risk prostate cancer undergoing active surveillance, high-intensity interval training (HIIT) was found to be beneficial. A new study found that patients who underwent HIIT exercises three times per week experienced decreased PSA level and velocity and prostate cancer cell line LNCaP growth compared to standard care patients.

The HIIT group experienced decreased PSA level (1.1 g/L), PSA velocity (1.3 g/L/year), and prostate cancer cell line LNCaP growth (0.13 optical density unit) compared with the usual care group.

Demonstration that HIIT alone, without dietary changes, resulted in improved cardiorespiratory fitness and biochemical parameters in men with localized prostate cancer on active surveillance and growth inhibition at the cellular level is novel and noteworthy, write the authors of an accompanying editorial.

High-Intensity Interval Training Beneficial in Prostate Cancer

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Hem/Onc Roundup: Pharma CEO Talks Cellular Therapy, and More - DocWire News

REGULATED INFORMATION

No statistically significant difference in knee pain reduction between JTA-004, placebo and active comparator, 3months after treatment; favorable JTA-004 safety profile similar to placebo and comparator

Prime focus on the continued development and expansion of its mesenchymal stromal cell based allogeneic cell and gene therapy platform

Management to host conference call today at 4pm CEST / 10am EST - details provided below

Gosselies, Belgium, 30August 2021, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces that the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 did not meet the primary and consequently the key secondary endpoints.

The primary objective of the JTA-004 Phase III study was to demonstrate the efficacy of JTA-004 in reducing osteoarthritic knee pain compared to placebo as measured by the WOMAC pain subscale three months after treatment. A key secondary objective was the comparison between JTA-004 and comparator Hylan G-F 20 in knee pain relief at month 3. Despite JTA-004s favorable safety profile, the study did not achieve its main objectives as no statistically significant difference in pain reduction could be observed between any of the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.

A statistically significant difference in favor of JTA-004 and the active comparator versus placebo was seen in a post-hoc analysis in a subset of patients with higher pain scores at entry.

The Company, in collaboration with existing and potential partners, will consider the options for the future of JTA-004 development.

The execution of the study was flawless and a good safety profile was observed in line with previous results. These JTA-004 efficacy results are disappointing. Knee osteoarthritis studies are recognized across the industry to be challenging to evaluate. They are also frequently complicated by a high placebo effect. We will continue to analyze the data and will consider potential next steps, said Miguel Forte, Chief Executive Officer of Bone Therapeutics. We are now fully committed to the clinical development of our advanced MSC allogeneic cell and gene therapy platform. Bone Therapeutics is concentrating on the development of this platform for the large market of orthopedic indications, with ALLOB. The progress with this platform has enabled us to expand it to other indications, including immunomodulation.

Bone Therapeutics is focused on the development of its core assets, the allogeneic cell therapy platform, including ALLOB. ALLOB is currently being evaluated in a randomized, double-blind, placebo-controlled Phase IIb study in 178patients with fresh tibial fractures at risk of delayed or non-union. 5% to 10% of complicated long bone fractures evolve to delayed union and non-union. This study will assess the potential for a single percutaneous injection of ALLOB to accelerate fracture healing and prevent late-stage complications in these patients. Recruitment is expected to be completed in the first half of 2022 and topline results by the end of 2022. Should the pandemic continue, Bone Therapeutics may have to re-evaluate these timelines and, in that eventuality, will communicate again to the market.

Bone Therapeutics is intensifying its efforts to expand its preclinical and clinical pipeline with additional indications by enhancing and professionalizing the therapeutic capacity of its cell and gene therapy platform. This includes the development of a next generation of genetically engineered mesenchymal stromal cells (MSC) and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells (iPSC).

Conference call

The management of Bone Therapeutics will host a conference call today at 4:00 pm CEST / 10:00 am EST. To participate in the conference call, please select your dial-in number from the list below quoting the conference ID 825 1002 3115#:

Belgium: +32 2 290 9360France: +33 1 7095 0103United Kingdom: +44 208 080 6592United States: +1 646 876 9923

About JTA-004 and Phase III knee osteoarthritis study

JTA-004 is Bone Therapeutics next generation of intra-articular injectable for the treatment of osteoarthritic pain in the knee. It consists of a unique mix of hyaluronic acid - a natural component of knee synovial fluid, plasma proteins, and a fast-acting analgesic. JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain.

The JTA-004 Phase III study is a controlled, randomized, double-blind trial. It evaluates the potential of a single, intra-articular injection of JTA-004 to reduce osteoarthritic pain in the knee, compared to placebo or Hylan G-F 20, the leading osteoarthritis treatment on the market. The study is being conducted in 22 centers across six European countries as well as Hong Kong. More than 700 patients with mild to moderate symptomatic knee osteoarthritis were treated in this study.

About Knee Osteoarthritis

Osteoarthritis (OA), also known as degenerative joint disease, is the most common chronic joint condition in which the protective cartilage in the joints progressively break down resulting in joint pain, swelling, stiffness and limited range of motion. The knee is one of the joints that are mostly affected by osteoarthritis, with an estimated 250 million cases worldwide.

The prevalence of knee osteoarthritis (KOA) is expected to increase in the coming years due to increasingly aging and obese population. Currently, there is no cure for KOA and treatments focus on relieving and controlling pain and symptoms, preventing disease progression, minimizing disability, and improving quality of life. Most drugs prescribed to KOA patients are topical or oral analgesics and anti-inflammatory drugs. Ultimately, severe KOA leads to highly invasive surgical interventions such as total knee replacement.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a diversified portfolio of cell therapies at different stages ranging from pre-clinical programs in immunomodulation to mid stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

Read more from the original source:
Bone Therapeutics announces topline results from Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 - GlobeNewswire

CAMBRIDGE, Mass., Aug. 24, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Arbor Biotechnologies (Arbor) today announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbors proprietary CRISPR gene-editing technology for select diseases.

The new agreement between Arbor and Vertex builds upon the companies first partnership established in 2018. Under this new partnership, Vertex will receive rights to use Arbors technology to research and develop ex vivo engineered cell therapies towards Vertexs goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases.

This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are excited to bring Arbors technology together with Vertexs ongoing programs and capabilities in diabetes, hemoglobinopathies and other diseases to create improved cell replacement therapies for broad populations of patients.

Arbor and Vertex share a common goal to improve the lives of people with life-threatening diseases through the discovery and development of innovative therapies, and Vertex has proven to be an ideal partner in that effort, said Devyn Smith, Ph.D., CEO of Arbor. This agreement demonstrates the versatility and strength of Arbors platform and reinforces our strategic vision of expanding our engineered cell therapy capabilities through partnerships with leading organizations in our industry, while we focus our internal portfolio efforts on genetic medicines.

About the CollaborationUnder the terms of the agreement, Arbor will receive an upfront cash payment and is eligible to receive up to $1.2 billion in potential payments based upon the successful achievement of specified research, development, regulatory and commercial milestones across up to seven potential programs. In addition,Vertexwill pay tiered royalties on future net sales on any products that may result from this collaboration.Vertex will also make an investment in Arbor in the form of a convertible note.

About VertexVertexis a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF,Vertexhas a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition,Vertexhas a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 inCambridge, Mass.,Vertex's global headquarters is now located inBoston'sInnovation Districtand its international headquarters is inLondon. Additionally, the company has research and development sites and commercial offices inNorth America,Europe,AustraliaandLatin America.Vertexis consistently recognized as one of the industry's top places to work, including 11 consecutive years onScience magazine'sTop Employers list and a best place to work for LGBTQ equality by theHuman Rights Campaign. For company updates and to learn more aboutVertex's history of innovation, visitwww.vrtx.comor follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

About ArborArbor Biotechnologies is an early-stage life sciences company discovering and developing the next generation of genetic medicines. Co-founded by Feng Zhang and David Walt, Arbor uses its proprietary discovery engine to discover unique and wholly owned CRISPR-based genetic modifiers with differentiated genetic editing capabilities. This portfolio of gene editors can be custom tailored to address the underlying pathology of genetic diseases. Arbors pipeline of genetic medicines is focused on bringing curative therapies to all patients with genetic disease. Following its strategic partnership with Vertex Pharmaceuticals to accelerate the path to the clinic for Arbors technologies, Arbor recently announced an agreement with Lonza. These partnerships further validate the breadth of applications of Arbors gene editing platform. For more information about Arbors technologies and innovations, visit arbor.bio.

Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation,statements by Bastiano Sanna, Ph.D., and Devyn Smith, Ph.D., in this press release, statements about the potential benefits and results that may be achieved through Vertexs collaboration with Arbor, statements regarding the future activities of the parties pursuant to the collaboration, statements regarding upfront and milestone payments, potential royalties on future sales and Vertexs investment in Arbor in the form of a convertible note. WhileVertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the companys beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of Vertexs collaboration with Arbor may not be achieved, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors inVertex's annual report and subsequent quarterly reports filed with theSecurities and Exchange Commission(SEC) and available through Vertexs website atwww.vrtx.comand on the SECs website atwww.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Vertex Contact:

Investors:Michael Partridge, 617-341-6108orBrenda Eustace, 617-341-6187orManisha Pai, 617-429-6891

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

Arbor Media Contact:Amy Bonanno, +1 914-450-0349abonanno@soleburytrout.comorpress@arbor.bio

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Vertex and Arbor Biotechnologies Establish New Partnership to Develop Novel ex vivo Engineered Cell Therapies - GlobeNewswire

Clinical Trials Arena speaks to Lineage Cell Therapeutics CEO Brian Culley about the firms lead candidate OpRegen for dry AMD and the unexpected cases of retinal restoration in an ongoing Phase I/IIa trial.

Age-related macular degeneration (AMD) is one of the worlds leading causes of blindness in people over the age of 50. There are two types of the disease wet AMD and dry AMD.

The less common wet AMD is well understood as being caused by leaky blood vessels and has numerous effective treatments available. Dry AMD causes layers of the macula (including the photoreceptors and the retinal pigment epithelium) to get progressively thinner and function less and less well. This is called atrophy.

Advanced cases of dry AMD are known as geographic atrophy (GA) because large sections of the retina stop functioning.

There are around two million people in the US suffering with this severe form of the condition, and there are currently no treatments available, representing a huge unmet need. Historically, dry AMD has been viewed as an inevitably progressive disease.

California-headquartered Lineage Cell Therapeutics is taking a very different approach to the disease compared to traditional pharmaceutical approaches, which often involve targeting inflammation with small molecules and antibodies and treating patients either orally or locally in the eye.

With its lead candidate OpRegen, which is in an ongoing Phase I/IIa open-label clinical study, Lineage is manufacturing brand new retina cells and taking a transplant approach to the condition.

Its been very exciting what we have seen in the clinic so far, the companys CEO Brian Culley tells Clinical Trials Arena.

The one-time therapy consists of allogeneic retinal pigment epithelium (RPE) cells and is administered subretinally in patients with dry AMD and GA. The patient is locally anaesthetised and the procedure only takes about 30 minutes.

The firm uses pluripotent stem cells as a starting material to manufacture the therapy. Just as flour can become bread or a cookie, [pluripotent] stem cells have within them the capability or the capacity to become any of the 200 cell types in your body, says Culley.

We instruct the cells to become a specific and exclusive type of cell and then we transplant those into the body. In the case of dry AMD we manufacture enormous numbers of retina cells and only retina cells, and then we transplant those cells to treat disease of the eye.

The manufacture of cells requires exquisite control over your process as you are manufacturing a dynamic living entity at scale, Culley says. If you cannot manufacture at large scale, you will never have an economically affordable solution.

The allogeneic rather than autologous nature of Lineages manufactured retinal cells provides advantages in scale, he argues.

There are approaches that people take even in dry AMD where they take cells from a persons eye, and they manipulate them and then they replace them, but here youre talking about personalised medicine, which sounds great until you consider the cost.

Weve invested significantly in our manufacturing skills and created a huge number of patents from our manufacturing techniques. We are already at the point where, in a three-litre bioreactor, we can manufacture the equivalent of 2500 clinical courses so many thousands of treatments can come from basically a milk jug and scaling up is straightforward because we grow the cells on little microcarriers. We grow the cells in three-dimensional space and that allows us to increase the volume because the cell doesnt really know the difference between growing in a tiny thimble or growing in a swimming pool.

Cell therapy brings with it the complexity of dealing with a whole cell rather than just a single molecule, but that complexity is also the key to this approach.

By the time retina cells are dying off, there are so many things going wrong in the eye that we dont think that a single molecule is going to have enough horsepower to be able to drive a clinical outcome, Culley says. So we think you have to replace the entire cell.

The ongoing Phase I/IIa clinical trial evaluating OpRegen enrolled 24 people. Twelve of the participants treated with the therapy had very advanced AMD and were legally blind.

Culley says this cohort was used to assess the treatments safety and not much was seen in terms of efficacy but some encouraging anatomical changes were observed.

In the eye, the vision cycles metabolic activity leads to a waste material called drusen, which is cleared by healthy retina cells. In some of these patients, a reduction in drusen was observed.

The next group of 12 patients disease was far less advanced and they had far better baseline vision than the first; some even still had their drivers licenses. At their earlier stage of disease, they had more retinal tissue with potential to be rescuable. When the trial moved into the group of patients with better starting vision, Culley and his team started to see very exciting effects in terms of visual acuity and anatomical changes.

We were really happy to see that in that group of individuals, weve had better results, Culley says. We have seen an increase in vision, so people are seeing more letters on an eye chart, compared to their untreated eye. The majority of untreated eyes have gotten worse, which is what you would expect over time. In the treated eye it is reversed; weve actually seen people gain vision.

No patients in the trial, which started treating patients with OpRegen five years ago, have rejected the cells, meaning all have stably integrated them. Most exciting of all are three cases of retinal restoration, which Culley says is really unprecedented stuff due to the progressive nature of the disease.

This is a new phenomenon, says Culley. Human beings cannot regenerate retinal tissue; when you lose retina cells, theyre gone forever. And thats what this disease is all about losing retina cells.

Over a year ago, while looking at images of a patients eyes after OpRegen treatment, doctors noticed retina cells growing back around the spot of GA.

We were absolutely blown away, Culley says. We actually sat on this initial finding for quite some time, because even though it was incredible, and exciting and maybe even predictable in some ways youre transplanting cells and if theyre taken up, survive and are functional why wouldnt this happen but nobody thought it was possible.

The question the firm focused on while analysing the data and sending it out to third-party independent retinal imaging experts was simple why did this patient experience retinal tissue regeneration while no one else did?

We think the answer was that patient got a very complete delivery of our cells all across that atrophic area, explains Culley.

Lineage repeated the procedure and got similarly extensive coverage across another two patients, and they also exhibited retinal restoration. So we now have three cases that have exhibited retinal restoration. Now its not a one-time thing we have some understanding, we could reproduce it, its quite clear.

One of the trial participants atrophy originally got smaller but now has grown back to the size it was three years ago meaning that essentially her AMD had been halted.

In the three years since I had the operation, the eye that was operated on has not deteriorated which I reckon is almost miraculous, said the patient, Sonia Cohen. Its really amazing. I cant imagine what my life would have been like if I had continued to deteriorate.

Culley says that with OpRegens clinical research thus far, his team has learned that being more aggressive with where they place the cells leads to better outcomes.

Now we know that so we can repeat that. I think over time, it will get better and better, and Id be really delighted if we get up to the point where it becomes like LASIK surgery, which is close to 100% success rate.

Later this year, Lineage is planning to discuss the design of its next trial with the US Food and Drug Administration. For now, the three cases of retinal restoration or reversal of AMD have provided a vital proof of concept.

We want to go as quickly as possible into the next study, and having the backing of this reversal is very exciting because, from a statistical perspective, we probably dont need 2,000 patients were talking about hundreds of patients.

On the future of cell therapy, Culley says it is a field that is quickly maturing. It used to be like the wild west but now companies like ours are doing these rigorous clinical trials to find out ways to control it and dry AMD is a really cool place to start, he says. But, there are 199 other cell types that we could investigate so there is a long-term medical story here that is really about ushering in a new branch of medicine manufacturing cells, transplanting them that Im really excited to be part of.

Dry AMD is not the firms only focus; it has also embarked on clinical trials for VAC2, an allogeneic dendritic cell therapy currently enrolling a Phase I trial for the treatment of non-small cell lung cancer and has treated 25 people with spinal cord injuries with its candidate OPC1 in a Phase I/II trial, which Culley says has been very emotional.

I know if I live long enough my retina cells are going to die off; if I smoke four packs of cigarettes [a day] I will get lung cancer, but nobody expects to get a spinal cord injury. All of these young people who fall off mountain bikes or get in car crashes and their lives are turned upside down. Their ability to just get some hand control to be able to move their wheelchair thats mobility and independence so if we can help regain mobility, thats freedom so I think the spinal cord program is really powerful.

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OpRegen: tackling the leading cause of blindness with cell replacement - Clinical Trials Arena