Category Archives: Regenerative Medicine

A new approach is paving the way for improved stem cell therapies and regenerative applications using cells from pigs. Led by Wan-Ju Li, a SCRMC researcher and associate professor in the Department of Orthopedics and Rehabilitation and the Department of Biomedical Engineering, this new study published in Scientific Reports offers a viable strategy to enhance the generation of induced pluripotent stem cellsfrom large animal cells and provides researchers with insight into the underlying mechanism controlling the reprogramming efficiency of cells. In turn, this approach will allow researchers to reprogram cells more efficiently into iPSCs which can be used to study regenerative therapies aimed at treating everything from osteoarthritis to heart disease.

While this approach can be applied to regenerative therapies targeting any organ or tissue, Li and his Musculoskeletal Biology and Regenerative Medicine Laboratory study cartilage, so he developed the model by deriving iPSCs from the fibroblast cells of three different breeds of miniature pigs including Wisconsin miniature swine, Yucatan miniature swineand Gttingen minipigs. Fibroblast cells are easily obtained for cellular reprogramming and Li is interested in using these cells to efficiently develop cartilage cells that can be used to help patients experiencing osteoarthritis. But, while his goal for the study was specific, the model has wide-reaching implications.

"This model we created can be used for many applications," says Li. "In successfully developing iPSCs from three different breeds of minipigs, we learned we can take somatic skin cells from these pigs that we programmed ourselves into iPSCs and then inject them back into the same animal to treat the disease. Or we can take the cell that carried the disease gene and put that into the culture dish and use that as a disease model to study disease formation."

Li explained that iPSCs can be created from nearly any type of somatic cell, such as skin or blood cells, that are reprogrammed back into an embryonic-like pluripotent cells. These pluripotent stem cells are the bodys master cells and are, therefore, able to become nearly any cell in the body. Harnessing the power of such a cell and being able to grow these versatile cells in the lab is invaluable to medicine as these cells can be used for the regeneration or repair of damaged tissue and in drug testing to see how medication will impact heart, liver, or other cells within the body.

Through this research, Li and his lab have provided researchers with insight into the underlying mechanism controlling the reprogramming efficiency of iPSCs, allowing researchers to harness to power of iPSCs and develop them more efficiently. Specifically, he discovered that the expression level of the switch/sucrose nonfermentable component BAF60A, which is essentially a protein that can remodel the way DNA is packaged, helps to determine the efficiency of iPSC generation. He also noted that the BAF60A is regulated by STAT3, a transcription factor protein that plays a role in cell growth and death. Through this, Li discovered that the efficiency of iPSC generation is based on the expression level of these proteins and that the expression levels vary among pig breeds.

"While we successfully developed iPSCs and programmed iPSCs from the three different strains of pig, we noticed that some pigs had a higher reprogramming efficiency,"says Li. "So, the second part of our findings, which is significant in biology, is understanding how these differences occur and why."

Li shared that understanding why different pig breeds have varying levels of reprogramming efficiency will directly translate to understanding differences in the effectiveness of iPSC generation between individual humans. In fact, a previous study by Mackey et al., has shown that a person's ethnicity may impact their cell's reprogramming efficiency. So, understanding what mechanisms control cellular reprogramming will be crucial to developing effective protocols of iPSC generation for individualized therapies.

"With this model, we can study musculoskeletal regeneration particularly cartilage regeneration for osteoarthritis patient,"says Li. "But we think the impact is way beyond the application of orthopedics because from now on, anybody on campus who is interested in using the technology we have developed for a minipig model, can reprogram their cells into iPSCs and then these cells and the animal can be used to investigate heart disease, kidney disease, neuronal diseaseor any type of a disease."

Translating this research to improve human health, is deeply important to Li. He has spent much of his career studying novel approaches to regenerate cartilage and bone for orthopedic applications and developing a translational model like this means that science is one step closer to healing more patients using stem cells.

"I feel really touched by the stories people share. You cannot imagine how many emails come in asking me if they can become the first human patient in our future clinical trial,"Li says. "People are in desperate need for something, especially when those people feel the current surgical procedure or intervention is not suitable for them. I have to keep saying, 'wait for another two, three years, maybe we'll be ready for a clinical trial,'but for me, it's time to move on and really do our larger animal studies to fulfill our promise. At least that way, I can fill the gap between the lab and clinical trials as the larger animals must be studied before you go into a clinical trial."

Li's development of a reliable and translational model for the generation of iPSCs in a large animal is critical as it has been a challenge to generate pig iPSCs with efficiency. The reprogramming efficiency of pig cells is relatively low compared to that of human or mouse cells, but large animal studies remain a crucial step in bringing treatments to clinical trials.

Interest in moving these treatments forward has grown and while this study was funded in part by NIH, Li also received support from the Plunkett Family Foundation in Milwaukee through their donation to the UW Stem Cell and Regenerative Medicine Center. After hearing of Li's research, Gwen Plunkett and her daughter Karen visited Li and his lab in 2019 to learn more and were inspired to support research into stem cells for cartilage regeneration.

"Innovation in medicine sparks critical change, for the world and the survival of our species and the Plunkett Family mission is to be a catalyst in stem cell and regenerative medicine research,"says Karen Plunkett. "We supported Jamie Thomson's lab in the early days when federal funding for human stem cell research was restricted. More recently, we continued our commitment to this research by supporting Dr. Wan-Ju Li's stem-cell based approaches for regenerating skeletal tissues, cartilageand bone for orthopedic applications. Additionally, it is personally gratifying to be able to support the SCRMC while my son completes his senior year studying neurobiology at UWMadison.We are happy to be part of the University of Wisconsin family."

Li shared that the donation was profoundly impactful and allowed him to further his goal of using stem cells to help patients struggling with osteoarthritis as well as other joint diseases.

"I want to make sure that our findings in stem cell research can be used to help people,"says Li. "I just feel this internal drive to study this area and I feel good knowing this model carries significant weight in terms of its potential for translational stem cell research and the development of therapeutic treatments."

This research was supported by grants from the National Institutes of Health (R01 AR064803) and the Plunkett Family Foundation. The UW Department of Pathology and Laboratory Medicine and UWCCC (P30 CA014520) and the Small Animal Imaging andRadiotherapy Facility and Flow Cytometry Laboratory, supported by UWCCC (P30 CA014520) also provided facilities and services.

Source: University of Wisconsin-Madison, whichis solely responsible for the information provided, and wholly owns the information. Informa Business Media and all its subsidiaries are not responsiblefor any of the contentcontained in this information asset.

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Reprogramming pig cells leads way for new regenerative therapies - National Hog Farmer

This week, researchers delivered insights and breakthroughs in regenerative medicine, Long COVID, immuno-oncology and inflammatory diseases.

Epigenetic Treatment Shows Potential in Spinal Cord Regeneration

Researchers reported that activation of the CBP/p300 protein family led to increased axon growth, regenerative signaling and synaptic plasticity in the spinal cord after injury in mice.

The study, led by Imperial College London,used a molecule called TTK21 to activate the genetic program to induce axon regeneration in neurons. During this process, TTK21 changes the epigenetic state by activating the CBP/p300 family of co-activator proteins, effectively leading to increased axon regeneration.

In the study, researchers began treating the mice 12 weeks after severe spinal cord injury and continued for 10 weeks. They found axon sprouting in the spinal cord and retraction of motor neurons above the injury. These changes are believed to have been spurred by increased gene expression related to regeneration, which the researchers attributed to the TTK21 treatment.

The results, while early and limited, are a step toward potential treatments for spinal cord injury.

The next steps will be to enhance the effects of the treatments and trigger the regenerated axons to connect to the rest of the nervous system. The ultimate goal is to enable animals, and eventually people, to regain movement lost from their injuries.

Neurological Consequences Evident in Long-Term COVID Study

In a recent attempt to decode the long-term impacts of COVID-19, researchers built a dataset fromthe national healthcare databases of the US Department of Veterans Affairs.

The study,led by the Clinical Epidemiology Center, Research and Development Service at VA St. Louis Health Care System,included154,068 individuals with COVID-19, 5,638,795 contemporary controls and 5,859,621 historical controls.

Results were published Thursday in Nature Medicine.

Upon examination of the data sets, the researchers noted an increased risk in a series of neurological consequences. These included: ischemic and hemorrhagic strokes, episodic disorders, extrapyramidal and movement disorders, mental health disorders, musculoskeletal disorders, sensory disorders, Guillain-Barr syndrome and encephalitis or encephalopathy.

They estimated a hazard ratio of 1.42 neurological sequelae per thousand COVID-19 cases, and 70.69 burdens per 1,000 cases.

These numbers were not impacted by the severity of the infection or the need for hospitalization. The researchers note the sample set comprised a majority of white men, so further exploration into other demographic groups may be necessary for future studies.

Novo Nordisk and Octagon Unite on Inflammatory R&D

Octagon Therapeutics, a pre-clinical biopharmaceutical focused on autoimmune disease, announced the initiation of a multi-year research collaboration with Novo Nordiskto studypotential treatments for inflammatory diseases.

Octagon will bring its functional target discovery approach and novel chemistry approach, while Novo will contribute its specific disease expertise.

Uli Stilz, vice president of the Bio-Innovation Hub at Novo Nordisk, commented on the synergies.

Combined with our disease understanding in the cardiometabolic space and Octagons approach in targeting specific lymphocyte populations that drive disease progression, it will be exciting to see what therapeutic discoveries the collaboration can lead to, he said.

Regen's RNA/DNA-Based Cancer Immunotherapy

Regen BioPharmaannounced the filing of a provisional patent application for its RNA/DNA-based approach to cancer immunotherapy.

The patent pertains to what Regen believes is the first combination of an immunotherapy and a gene silencing agent in a single drug.

The novel composition acts as a checkpoint inhibitor drug that also silences the genes that regulate T-cells and cancer cells such as NR2F6 and Survivin.

The new drug takes advantage of aptamers - a short RNA or DNA sequence that can also function as an antibody, recognizing specific proteins and binding to them. The proprietary sequence codes for inhibitory RNA, while keeping the aptamer intact on the other side. When it binds to a checkpoint such as PD-1, that RNA is converted within the cell, thus killing it.

Used to target cancer cells, this could lead to better disease control and treatment. There is also hope that this process could be used to activate T cells, improving their immunotherapeutic potential.

Toronto-Based Researchers Uncover Potential Solve for CAR T Toxicities

Allogeneic double-negative (DN) chimeric antigen receptor (CAR)-T cells inhibit tumor growth without off-tumor toxicities, a new study found.

Current CAR-T therapies approved by the FDA in treating blood cancers are limited by their level of toxicity and cost of production. Researchers from the Toronto General Hospital Research Institute, University Health Network, reported a new version of these therapies that did not have these drawbacks.

The researchers found healthy donor-derived allogeneic DNTs as a CAR-T cell therapy platform had high levels of efficacy in both the human and mouse models.

The researchers found the DN CAR-T cells were as effective as previously studied CAR-T cells but did not have the associated toxicity levels. They also come with the advantage of being made from mixed donors and remained effective even after being frozen for long periods of time. This feature could potentially overcome manufacturing challenges in the space.

The authors noted these features make DN CAR-T cellsan attractive off-the-shelf CAR-T cell therapy option.

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Research Roundup: Regenerative Potential, Long COVID Insights, Immuno-Oncology and More - BioSpace

Wellness and Esthetics Expert, Dr. Giselle M. Batcheller, In #1 Selling Medical Book Worldwide

Park City, Utah local and owner of Wellness and Esthetics and CARE Esthetics Utah, Dr. Giselle M. Batcheller, is featured in Quintessences 2022 #1 Selling Book Worldwide, CARE Esthetics.

Although the book was just published earlier this year, the new book written about the latest technologies in dermatology, orthopedics, regenerative medicine, and facial esthetics quickly catapulted to the number one selling spot for Quintessence.

Dr. Batcheller explains, CARE Esthetics outlines the latest technologies in regenerative medicine and facial esthetics. Quintessence historically publishes texts for doctors, yet because of the vast interest, CARE Esthetics was published for the clients of the esthetics community. That shows how highly sought after this information is.

Dr. Batcheller believes this trend will continue. Natural solutions and regenerative medicine and esthetics is a hot topic. Everyonenot just the clinicianwants to know about it. This is the future of medicine and aesthetics, and it is a very exciting future!

Dr. Batcheller provides a variety of services at Wellness and Esthetics in Park City with the most cutting edge technologies and world class equipment.

Wellness and Esthetics menu includes a variety of treatments for both health and aesthetics including PRF injections, full body (and localized) light therapy called photobiomodulation, strength-based body contouring that reduces both visceral and subcutaneous fat naturally. In addition, there are wellness enhancing laser treatments of the airway for athletic performance and Obstructive Sleep Apnea, non-surgical cavitation treatments, scar revisions, regenerative joint injections, natural treatments for non-invasive pelvic floor strengthening, and more.

Dr. Giselle M. Batcheller (DDS, NMD) and her clients are putting Park Citys Wellness and Esthetics community on the global map for advanced regenerative medicine in 2022. Dr. Batchellers passion is helping her clients look and feel their best in the most healthy and natural ways possible.

Dr. Batcheller was the first doctor in Utah to use these advanced protocols discussed in CARE Esthetics. Dr. Batcheller has been working in natural and holistic medicine since 2011. She started performing regenerative medicine and all-natural esthetics procedures with lasers and smart blood concentrate technologies in 2018. Since then, Dr. Batcheller has taught these sought after methods to dozens of prestigious doctors and providers from across the United States as well as locally throughout Utah as theyve been advancing. Dr. Batcheller has received accolades from world experts in her industry. She has been widely recognized and sought after to teach and to perform her services. She has called Park City home since 2016, and after providing these services throughout Utah and on the East Coast, shes delighted to bring her gifts to her Wellness and Esthetics office in Park City.

CARE is the acronym for the Center for Advanced Rejuvenation and Esthetics, and the book is written about a the technologies offered in CARE Esthetics centers.

Quintessence International Publishing Group is a scientific and clinical publishing company with publishing houses in 14 countries. Theyve produced more than 1,500 books from more than 3,000 authors.

Media ContactCompany Name: Wellness and Esthetics + CARE EstheticsContact Person: Dr. Giselle M. BatchellerEmail: Send EmailPhone: 833.411.4PRF (833.411.4773)Country: United StatesWebsite: https://wellnessandesthetics.com/

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Dr. Batcheller's Wellness and Esthetics, Mecca for Regenerative Medicine and Esthetics - Digital Journal

MIAMI, FL / ACCESSWIRE / September 20, 2022 / Organicell Regenerative Medicine, Inc. (OTCQB:OCEL), a clinical-stage biopharmaceutical company focused on the development of innovative biological therapeutics and regenerative medicine, announced that the Company has recruited a new Chief Operating Officer, Chief Information Officer, and Director of Finance.

Organicell's new Chief Operating Officer, Ryan Likes, has previously served as Co-Chief Operating Officer of Super Deluxe, a division of Turner Broadcasting, and Chief of Operations and Business Affairs of Televisa USA, the English language division of Grupo Televisa. Ryan has a juris doctorate from the Yale Law School and started his career as a corporate attorney in the Century City office of O'Melveny and Myers specializing in Mergers & Acquisitions and Finance.

Organicell's new Chief Information Officer, David Aciego, formerly served as Chief Information Officer of HTCS Corp, a managed source provider for technical services. Throughout his career,

Mr. Aciego has designed multiple systems including proprietary Enterprise Resource Planning (ERP) and Customer Relationship Management (CRP) solutions which enabled the streamlining of ordering deficiencies between different organizations and third-party distributors. Mr. Aciego has been programming since the age of seventeen and has worked with Cisco, IBM, Dell, Microsoft, Fortinet, and Meraki. Mr. Aciego served in the United States Marine Corps where he worked as an engineer on the implementation of network technologies.

Jacqueline (Jackie) Domenech, Director of Finance for Organicell, has over 10 years of Controllership experience in various healthcare and technology companies - including a recent Controller role at MD LIVE. She has successfully led transitions from outsourced accounting providers and implemented ERP systems.

Matt Sinnreich, who joined Organicell in August of 2022 as Chief Operating Officer and Acting Chief Executive Officer, will continue as President and Acting Chief Executive Officer.

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Mr. Sinnreich said, "At this stage of growth it is necessary to invest in people that can handle specific tasks and responsibilities. I am a believer in creating systems for every facet of a business that can be taught, duplicated, and scaled. I made these strategic hires to surround myself with talent with specific core-competencies in the areas of the company that needed enhancement.

Over the past few months, we have consulted with our department heads and long-term employees to identify where we as a company can improve efficiency, cut costs, and manage risk."

Sinnreich continued, "The first thing we needed was someone to help centrally manage operations and interface with our legal team. Hence, our COO hire had to be not only an experienced COO in large companies, but also a lawyer by trade. Ryan Likes has the skill sets to assist in creating processes throughout the organization and provide a completely different perspective on deal making. The deals we are working on in business development and recruiting other key additions to our team are similar in structure and milestones to talent and entertainment deals, so Ryan's experience and education will be a tremendous asset.

Next, we needed a robust Enterprise Resource Planning (ERP) system and Customer Relationship Management (CRM) system that can handle our current needs and scale with the Company's growth plan. David Aciego is running point on the development of a fully integrated, HIPAA compliant, ERP + CRM system that is currently being built on the Microsoft Dynamics platform. Data collection and management, coupled with the ability to automate Organicell's forecasting, inventory management, and staff needs, is essential in helping our company scale."

Sinnreich concluded, "Finally, our CFO needed support to better service our staff's budgetary needs to focus on the high-level compliance work and financial reporting that comes along with a publicly traded company. The Company needed systems and regulations put in place to manage and track expenses. Companies at this growth-stage rely on active and accurate cash management to expand and thrive. Jackie is highly experienced in this department with a proven track record and we believe that she will be an incredible asset to this Company."

Future Press Releases and Updates

Investors interested in receiving periodic press releases and updates are encouraged to

send an e-mail to ir@organicell.com

About Organicell Regenerative Medicines, Inc.

Organicell Regenerative Medicine, Inc. (OTCQB:OCEL) is a clinical-stage biopharmaceutical company principally focusing on the development of innovative biological therapeutics for the treatment of degenerative diseases and the provision of other related services. The Company's proprietary products are derived from perinatal sources and manufactured to retain the naturally occurring exosomes, hyaluronic acid, and proteins without the addition or combination of any other substance or diluent. To learn more, please visit https://organicell.com/.

Forward-Looking Statements

Certain of the statements contained in this press release should be considered forward-looking statements within the meaning of the Securities Act of 1933, as amended (the "Securities Act"), the Securities Exchange Act of 1934, as amended (the "Exchange Act"), and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are often identified by the use of forward-looking terminology such as "will", "believes", "expects", "potential", or similar expressions, involving known and unknown risks and uncertainties. Although the Company believes that the expectations reflected in these forward-looking statements are reasonable, they do involve assumptions, risks and uncertainties, and these expectations may prove to be incorrect. We remind you that actual results could vary dramatically as a result of known and unknown risks and uncertainties, including but not limited to: potential issues related to our financial condition, competition, the ability to retain key personnel, product safety, efficacy and acceptance, the commercial success of any new products or technologies, success of clinical programs, ability to retain key customers, our inability to expand sales and distribution channels, legislation or regulations affecting our operations including product pricing, reimbursement or access, the ability to protect our patents and other intellectual property both domestically and internationally, and other known and unknown risks and uncertainties, including the risk factors discussed in the Company's periodic reports that are filed with the SEC and available on the SEC's website (http://www.sec.gov). You are cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements attributable to the Company or persons acting on its behalf are expressly qualified in their entirety by these risk factors. Specific information included in this press release may change over time and may or may not be accurate after the date of the release. Organicell has no intention and specifically disclaims any duty to update the information in this press release.

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Organicell Investor Relations1-888-963-7881 Ext.701IR@organicell.com

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Organicell Regenerative Medicine, Inc. Announces Expansion of Management Team - Yahoo Finance

Clinical assessments three-months post-treatment report no serious adverse events and reduced seizure frequency to date in first trial participant

Data Safety Monitoring Board clears advancement of trial to continue enrollment

Pioneering cell therapy approach could provide a disease-modifying treatment for drug-resistant focal epilepsy

Data presented at ISSCR-ASGCT Conference: Emerging Therapies at the Intersection of Genetic and Cellular Technologies

SAN FRANCISCO, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced the presentation of a clinical case study from the initial dose cohort of the Phase 1/2 first-in-human epilepsy clinical trial of its lead program, NRTX-1001. The data from three months post-cell therapy administration to the first patient in this study show that there were no serious adverse events associated with NRTX-1001 treatment and that the patient has thus far experienced reduced seizure frequency from an average prior history of 30+ seizures per month to four seizures in total during three months of follow-up to date. The data are being presented by Catherine Priest, Ph.D., Neuronas chief development officer at the ISSCR-ASGCT Conference: Emerging Therapies at the Intersection of Genetic and Cellular Technologies being held in Madison, WI, September 21 -23, 2022.

The reduced number of seizures reported by the first person to receive NRTX-1001 is very encouraging, and we remain cautiously optimistic that this reduction in seizure frequency will continue and extend to others entering this cell therapy trial. NRTX-1001 administration has been well tolerated thus far in the clinic, which is in line with the extensive preclinical safety data collected by the Neurona team, said Cory R. Nicholas, Ph.D. Neuronas president and chief executive officer. With recent clearance from the Data Safety Monitoring Board we are excited to continue patient enrollment. We are very grateful to these first participants, and thank the clinical teams for the careful execution of this pioneering study.

In addition to the preclinical data supporting the clinical program, the presentation highlights initial data from the first patient treated in Neuronas clinical trial who received a starting dose of NRTX-1001. The patient is a young adult male with a 9-year history of seizures and was diagnosed with unilateral mesial temporal lobe epilepsy (MTLE). In the six months prior to the administration of NRTX-1001, the patient experienced an average of 32 seizures per month, despite being on several antiepileptic medications. The patient received NRTX-1001, and the treatment was well tolerated; there have been no serious adverse events to date. The patient has reported having four seizures during the first three months since receiving NRTX-1001.

The clinical trial is funded in part by the California Institute for Regenerative Medicine (DISC2-10525; TRAN1-11611; CLIN2-13355).

About Neuronas Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)Neuronas multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE, with five patients to be treated at a starting dose and five at a higher dose. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit http://www.clinicaltrials.gov (NCT05135091). The first part of the clinical trial is supported by a recently announced $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

About NRTX-1001NRTX-1001 is a regenerative neural cell therapy candidate derived from human pluripotent stem cells. The fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE)An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite treatment with approved drugs, which means that there is a huge unmet medical need in this community. MTLE is the most common type of focal epilepsy in adults and primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. For people with seizures that are resistant to anti-seizure drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or ablated by laser, can be an option. However, the current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

About NeuronaNeuronas regenerative cell therapy candidates have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit http://www.neuronatherapeutics.com

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Neurona Therapeutics Presents Clinical Case Study from First Human Trial of Regenerative Cell Therapy, NRTX-1001, in Adults with Drug-Resistant Focal...

New York, NY, and Brisbane, California, September 22, 2022 - Pfizer and Sangamo Therapeutics announced that the Phase 3 AFFINE study evaluating giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe hemophilia A, has re-opened recruitment. Trial sites will begin to resume enrollment this month, with dosing expected to resume in October. All trial sites are anticipated to be active by the end of 2022 and a pivotal readout is expected in the first half of 2024.

About the AFFINE studyThe Phase 3 AFFINE (NCT04370054) study is an open-label, multicenter, single arm study to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male participants with moderately severe to severe hemophilia A. Eligible study participants will have completed at least six months of routine FVIII prophylaxis therapy during the lead-in Phase 3 study (NCT03587116) in order to collect pretreatment data for efficacy and selected safety parameters.

The primary endpoint is impact on annualized bleeding rate (ABR) through 15 months following treatment with giroctocogene fitelparvovec. This will be compared to ABR on prior FVIII prophylaxis replacement therapy. The secondary endpoints include FVIII activity level after the onset of steady state and through 15 months following infusion of giroctocogene fitelparvovec.

About giroctocogene fitelparvovecThe U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product designation from the European Medicines Agency. Giroctocogene fitelparvovec is being developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug (IND) application to Pfizer. Giroctocogene fitelparvovec is currently being studied in the Phase 3 AFFINE study.

About Pfizer: Breakthroughs That Change Patients LivesAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

About Sangamo TherapeuticsSangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently dont exist. To learn more, visit http://www.sangamo.com and connect with us on LinkedIn and Twitter.

Pfizer Disclosure NoticeThe information contained in this release is as of September 22, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about an investigational hemophilia A therapy, giroctocogene fitelparvovec, including its potential benefits and the phase 3 AFFINE study, including anticipated timing of active trial sites, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications for any potential indications for giroctocogene fitelparvovec may be filed in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether giroctocogene fitelparvovec will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of giroctocogene fitelparvovec; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Sangamo Therapeutics Disclosure NoticeThis release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding plans and timing regarding active trial sites in the Phase 3 AFFINE clinical trial, including the resumption of patient enrollment, expectations regarding the anticipated timing of dose resumption and data readouts for the Phase 3 AFFINE trial, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Sangamos actual results may differ materially and adversely from those expressed in these forward-looking statements. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the evolving COVID-19 pandemic and its impact on the global business environment, healthcare systems and the business and operations of Sangamo and Pfizer, including the enrollment of patients and operation of clinical trials; the research and development process; the uncertain timing and unpredictable nature of clinical trial results, including the risk that therapeutic effects in the Phase 3 AFFINE trial will not be durable in patients; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products and product candidates; the commercialization of approved products; the potential for technological developments that obviate technologies used by Sangamo and Pfizer in giroctocogene fitelparvovec; the potential for Pfizer to terminate the giroctocogene fitelparvovec program or to breach or terminate its collaboration agreement with Sangamo; the potential for Sangamo to fail to realize its expected benefits of its collaboration with Pfizer; Sangamos lack of resources to fully develop, obtain regulatory approval for and commercialize its product candidate, giroctocogene fitelparvovec; and other risks and uncertainties described in Sangamo's filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2021, as supplemented by Sangamos Quarterly Report on Form 10-Q for the quarter ended June 30, 2022. The information contained in this release is as of September 22, 2022, and Sangamo undertakes no duty to update forward-looking statements contained in this release except as required by applicable laws.

Pfizer Media Contact:+1 (212) 733-1226[emailprotected]

Pfizer Investor Contact:+1 (212) 733-4848[emailprotected]

Sangamo Investor Relations & Media ContactLouise Wilkie[emailprotected][emailprotected]

More:
Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment - Pfizer

The University of Kentucky (UK) Gluck Equine Research Center has announced the 2022 inductees to the Equine Research Hall of Fame. This prestigious award is an international forum to honor outstanding achievements in equine research and those who have made a lasting tribute benefiting equine health. To celebrate this legacy the UK Gluck Equine Research Foundation will induct four scientists into the UK Equine Research Hall of Fame Oct. 26 at Kroger Field, in Lexington.

Peers of the four individuals and past awardees nominated them for their outstanding achievements in equine research. The inductees are Lisa Fortier, DVM, PhD, Dipl. ACVS, Katrin Hinrichs, DVM, PhD, Dipl. ACT, Jennifer Anne Mumford, PhD, and Stephen M. Reed, DVM, Dipl. ACVIM.

In research, we always stand on the shoulders of those who go before us with great discoveries, said Nancy Cox, UK vice president for land-grant engagement and College of Agriculture, Food and Environment dean. This years recipients have made substantial contributions that will ensure an excellent future for equine research..

The success of Kentuckys horse industry is inseparable from the decades of hard work by outstanding equine researchers, said Stuart Brown, DVM, chair of the Gluck Equine Research Foundation. Though impossible to measure, it is a unique privilege to recognize the impact made by these four scientists in advancing the health and well-being of the horse and, on behalf of the entire equine community, show our appreciation.

Over the past 30 years Fortier has garnered an international reputation for significant contributions in equine joint disease, cartilage biology, and regenerative medicine. She has focused her research on early diagnosis and treatment of equine orthopedic injuries to prevent permanent damage to joints and tendons. She is perhaps best known for her work in regenerative medicine, pioneering the use of biologics such as platelet rich plasma, bone marrow concentrate, and stem cells for use in horses and humans. Fortiers lab has also been instrumental in breakthroughs related to cartilage damage diagnosis and clinical orthopedic work. A testament to her impact is that 87% of U.S. equine veterinarians now use biologics for regenerative medicine in their equine patients.

Fortier earned her bachelors and DVM degrees from Colorado State University. She completed her residency at Cornell University, where she also earned a PhD and was a postdoctoral fellow in pharmacology. She now holds the James Law professor of surgery position at Cornells College of Veterinary Medicine. She is the editor-in-chief of the Journal of the American Veterinary Medical Association and serves on the Horseracing Integrity and Safety Authority Racetrack Safety Standing Committee.

Hinrichs devotes her career to research primarily in equine reproductive physiology and assisted reproduction techniques. Specifically, her focus has included equine endocrinology, oocyte maturation, fertilization, sperm capacitation and their application to assisted reproduction techniques.

Hinrichs 40 years of research have led to several significant basic and applied research achievements. The applied accomplishments include producing the first cloned horse in North America and developing the medical standard for effective intracytoplasmic sperm injection and in vitro culture for embryo production in horses. She has mentored more than 85 veterinary students, residents, graduate students, and postdoctoral fellows in basic and applied veterinary research. Her laboratories have hosted approximately 50 visiting scholars from throughout the world.

Hinrichs earned her bachelors and DVM degrees from the University of California, Davis. She completed residency training in large animal reproduction at the University of Pennsylvanias New Bolton Center and earned a PhD at the University of Pennsylvania.

A posthumous inductee, Mumford earned international respect as one of the most prominent researchers of equine infectious diseases, in particular equine viral diseases. Her distinguished career at the Animal Health Trust, in Newmarket, U.K., began when she became the first head of the newly established equine virology unit. Her work focused on the leading causes of acute infectious respiratory disease in the horse, primarily equine herpesvirus and equine influenza virus, and, to a lesser extent, Streptococcus equi, the causative agent of equine strangles.

Mumford made numerous significant contributions in these areas, including developing improved vaccines, diagnostics, and international surveillance. She also helped establish research groups in the related fields of equine genetics and immunology.

During Mumfords more-than-30-year career, she established the Animal Health Trust as one of the worlds leading centers for the study of the biology, epidemiology, immunology, and pathology of diseases, including equine herpesvirus rhinopneumonitis and equine influenza, as well as bacterial diseases, including Streptococcus and Clostridium.

Reeds nominators credited him as the last word in equine neurology. Reed is widely recognized as one of the most prominent equine neurologists worldwide. His list of 180 peer-reviewed publications includes significant contributions to equine medicine, neurology, physiology, and pathophysiology, and has earned him worldwide recognition throughout the equine community. He has shared in his achievements as a mentor and role model for hundreds of aspiring equine practitioners.

One of the most unique and refreshing things about Dr. Reed is he absolutely embodies the need and overlap of discovery science with clinical assessments to further our understanding of equine neurologic disease, wrote Jennifer Janes, DVM, PhD, associate professor of veterinary pathology at the UK Veterinary Diagnostic Laboratory, in her letter of support for the nomination. This mission has served as the foundation and pillars of his long career in equine veterinary medicine.

Reed earned his bachelors and DVM degrees from The Ohio State University. He completed internship and residency training in large animal medicine at Michigan State University.

Established in 1990, the UK Equine Research Hall of Fame honors international scientific community members who have made equine research a key part of their careers, recognizing their work, dedication, and achievements. Nominees can be living or deceased, active in or retired from the field of equine research.

Read more:
2022 UK Equine Research Hall of Fame Inductees Announced The Horse - TheHorse.com

Multi-Year Deal to Focus on Phase 3 Clinical Trial Supplies and Commercial Manufacturing

KING OF PRUSSIA, Pa., Sept. 20, 2022 /PRNewswire/ -- The Center for Breakthrough Medicines (CBM), a contract development and manufacturing organization (CDMO) dedicated to addressing the unique requirements associated with commercializing cell and gene therapies, and jCyte, a clinical-stage biotech company focused on preserving and restoring vision in patients with retinitis pigmentosa (RP) and other retinal degenerative diseases, today announced a multi-year manufacturing agreement wherein CBM will be the primary manufacturer of jCyte's U.S. Food & Drug Administration's Regenerative Medicine Advanced Therapy (RMAT) designated cell therapy, jCell. Under the terms of the agreement, CBM will provide Phase 3 clinical trial supplies and commercial drug product following Biologics License Application (BLA) approval of jCyte's proprietary jCell platform technology. CBM will also provide all BLA-enabling Chemistry, Manufacturing and Controls (CMC) work to support the program through regulatory approval and commercialization.

"jCyte is conducting truly advanced, groundbreaking research into treating patients with RP and other retinal degenerative diseases, and we are honored to partner with them during this critical phase of their clinical trial program as they prepare for BLA submission and commercialization," said Joerg Ahlgrimm, Chief Executive Officer of Center for Breakthrough Medicines. "We have built world-class technical teams combined with industry-leading product and process development, manufacturing, and testing capabilities, all located within a single manufacturing facility. Our unique and focused approach simplifies and expedites the completion of critical product and process development work and subsequent manufacturing campaigns, thereby shortening the time to market entry for cell therapies such as jCell. We look forward to working closely with the jCyte management team to fulfill our joint mission of safely getting their innovative cell therapy (jCell) to the many patients suffering from RP and other retinal degenerative diseases who have significant unmet medical needs and no available treatment options."

"We have made tremendous progress in advancing our innovative cell therapy program to this key stage of clinical development, and CBM's broad manufacturing capabilities, dedicated and experienced team, flexibility to fit our needs, and ability to provide fully dedicated manufacturing suites and analytical testing services tailored to our final drug manufacturing process made them an ideal partner to support our program," said Dr. Shannon Blalock, Chief Executive Officer of jCyte. "RP affects approximately one in 3,500 people worldwide. Our goals are to make jCell the first cell therapy approved to address this devastating condition and the first-line agent of choice. Partnering with a patient-focused, fully integrated, mission-centric CDMO like CBM will help ensure we achieve our goals."

RP is a rare genetic disease that is typically diagnosed in children and adolescents. It causes progressive vision loss leading to legal blindness by early adulthood. Nearly 2 million people suffer from the disease worldwide, including approximately 100,000 people in the U.S., which makes RP the leading cause of inherited blindness.To date, there is no approved treatment for 99% of RP patients.

jCell is a first-in-class investigational treatment for RP involving a simple, minimally invasive intravitreal injection, which can be easily administered within an ophthalmologist's office. The principal mechanism of action is the sustained release of established neurotrophic factors which have been shown to reduce photoreceptor cell death and promote the function of surviving photoreceptors. jCell therapy aims to preserve vision by intervening in the disease process at a time when host photoreceptors' function can be protected and potentially restored.

About The Center for Breakthrough Medicines (CBM)

CBM is a purposed built cell and gene therapy contract development and manufacturing organization (CDMO) based in the heart of Philadelphia's Cellicon Valley. CBM offers pre-clinical through commercial manufacturing capabilities including process development for cell therapy, plasmid DNA, and viral vector manufacturing, as well as cell banking, cell processing, and a full suite of complimentary testing and analytical capabilities. Through a fully integrated, end-to-end solution, CBM accelerates time to market without compromising quality.

About jCyte, Inc.

jCyte, Inc. is a clinical-stage biotech company focused on developing its first-in-class regenerative cell therapy, jCell, for retinitis pigmentosa (RP) and other retinal degenerative diseases. The Company is pioneering a new era of regenerative therapies to address the significant unmet medical needs of patients suffering from a broad set of retinal degenerative conditions. For more information, visit http://www.jcyte.com.

SOURCE Center for Breakthrough Medicines

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Center for Breakthrough Medicines (CBM) and jCyte Announce Strategic Partnership to Manufacture jCyte's Innovative Cell Therapy for Treatment of...

Are you someone who likes to educate or entertain yourself with podcasts while going about your day? If so, be sure to check out the AVMA journals new podcast series.

Veterinary Vertex is a weekly podcast that takes you behind the scenes of the clinical and research discoveries published in JAVMA and AJVR. Tune in to learn about cutting-edge veterinary research and gain in-depth insights you wont find anywhere else. Come away with knowledge you can put to use in your own practice along with a healthy dose of inspiration to remind you what you love about veterinary medicine.

Each week, the journals editors talk with the author or authors of a topical manuscript published recently in JAVMA or AJVR. Join them as they learn more about the authors clinical and research discoveries, as well as personal successes that have helped bring each author to their current career prowess.

For example: The first Veterinary Vertex episode, released Tuesday, offers a wide-ranging conversation with veterinary oncologist Dr. Nicole P. Ehrhart, one of the authors of Regenerative medicine 2.0: Extracellular vesiclebased therapeutics for musculoskeletal tissue regeneration.

Dr. Ehrhart talks about translational and regenerative medicine, including how she discovered her passion for this field of inquiry through an unplanned volunteer experience early in her career. She also shares details of how she came to drive the process to implement surveillance testing for COVID-19 in her state of Colorado.

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Science on the go: Catch the Veterinary Vertex podcast - American Veterinary Medical Association

The library will be named the Elaine Boyd Library, and the pledge will support library improvements and student services.

RMU alum Russ Olsen (82) has made a multi-million dollar pledge to Robert Morris University designated for the Robert Morris University Library.

Olsens pledge, made in honor of his stepmother Elaine Boyd, will support improvements to the library along with ongoing student support and services. The library will now be named the Elaine Boyd Library.

This is a great day for Robert Morris University. Our library is the intellectual heart of the university, and a key driver in the academic success of our students, said RMU President Michelle Patrick. Through his incredible generosity, Russ has ensured that the Elaine Boyd Library will continue to meet the needs of our students and the entire campus community far into the future.

Russ Olsen most recently served as Chief Executive Officer of Triad Life Sciences, Inc., an emerging regenerative medicine company with a groundbreaking FDA-cleared technology platform engineered to address unmet clinical needs in complex surgical wounds, chronic stalled wounds, and burns. Russ graduated from Robert Morris University in 1982 with a degree in Management and has over 35 years of experience in executive management, sales, and marketing. He gained much of his executive management experience while holding senior positions in marketing, business development, and sales for Fortune 500 companies.

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Russ Olsen Makes Multi-Million Dollar Pledge in support of the Robert Morris University Library - Robert Morris University