Enjoy this round-up featuring breaking news about stem cells, cell and gene therapies, exosomes, cord blood, and beyond.
In recent years, several universities and research hospitals have performed small-scale Phase I clinical trials using exosomes. So far, only a few companies have reached the pivotal point and transitioned into human clinical trials. Testing a new therapeutic in human subjects for the first time is a major step in the translation of any novel treatment from the laboratory bench to clinical use.
As operations for cell and gene therapy (CGT) programs become more demanding and supply chain management becomes more complex, your program may face challenges adapting to these changes. For example, increasing manufacturing and procedure volumes in combination with new and changing protocols will require the use of more automated, flexible and efficient processes. Tightening regulations will require greater documentation and process control. This article utlines five strategies to help your program succeed in the fast-changing industry of cell and gene therapies.
The immunotherapy field is booming, with cell therapies like CAR-T cells taking the lead. The FDA approved the world’s first CAR-T therapy, Kymriah, in 2017, and it is now available at over 130 treatment centers across the U.S. As immunotherapies are approved for more cancers and awareness of this treatment modality increases, the global market will continue to grow. To better understand how these cells function, Axion BioSystems is bringing its expertise in live-cell assay systems to help develop better preclinical models of immune-cell mediated killing of tumor cells.
C-Stem™ outperforms 2D & aggregate cultures in benchmark of cell culture technologies for the mass-production of human induced pluripotent stem cells
Bordeaux, France (May 11, 2021) – One month after announcing unprecedented exponential amplification of human pluripotent stem cells (hiPSCs) in a 10L bioreactor French cell therapy company, TreeFrog Therapeutics, released previously unpublished data at the 24th Annual Meeting of the American Society for Cell & Gene Therapy. The findings compare the continuous expansion over 28 days of hiPSCs in 2D cell culture, aggregate culture in bioreactor, and bioreactor-based C-Stem™ technology. The benchmark demonstrates that C-Stem™ outperforms both the 2D and aggregate cell cultures, with a cumulated amplification of over 151 millionfold over 28 days, achieved in only four passages.
Frederick, MD (May 20, 2021) – RoosterBio, a leading supplier of human mesenchymal stem/stromal cell (hMSC) working cell banks and hMSC bioprocess systems for cell and gene therapy product developers, announced the launch of its product RoosterGEM™, a stand-alone, complete gene transfer medium that is optimized to remove key challenges in primary cell genetic modification. RoosterGEM™, abbreviated for Rooster Genetic Engineering Medium, complements RoosterBio’s hMSC cell engineering system to offer a new and streamlined path for discovery, preclinical, and applied product development research – faster than ever before.
Miami, FL (May 19, 2021) – Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR) has signed an exclusive distribution agreement with Apex Services Pakistan to import and distribute Zofin™ to hospitals and clinics in the country. Apex Services will exclusively market, promote and distribute Zofin™ in Pakistan in connection with treatments for COVID-19 patients, subject to the approval of all necessary licenses for the use of Zofin™ by the Drug Regulatory Authority of Pakistan. Earlier this month, the Drug Regulatory Authority of Pakistan approved the use of Zofin™ for a single patient on compassionate grounds.
LACHUA, FLA. (April 21, 2021) – Cardiovascular disease kills more than 650,000 Americans annually. Those who survive myocardial infarction remain at risk of death because the human heart has a very limited ability to repair itself. No one has yet found a method that leads to cardiovascular regeneration for a damaged human heart. But that may change thanks to a revolutionary medical paper written by Dr. Ian White, President and CSO of Neobiosis, a company that focuses on the science of regenerative medicine. White’s paper explains that neonatal cardiac repair is mediated by the epicardium, which is a single layer of cells that covers the heart.
San Diego, CA (May 5, 2021) — Global Institute of Stem Cell Therapy and Research (GIOSTAR), a worldwide leader in regenerative technologies, is pleased to announce the formation of GIOCOV. The company – launched in tandem with TAG Investment Bankers LTD of London (TAG) – is a wholly owned subsidiary aimed at delivering stem cell-based treatments for the acute phase of COVID-19, along with the chronic phase (known as Long COVID).
DRESDEN, Germany (April 29, 2021) — denovoMATRIX GmbH, a developer of technologies that enable high quality stem cell expansion, will participate in the new European collaborative project funded by the European Union’s Horizon 2020 research and innovation program. Project Carrier4TSC was selected as one of the top 30 proposals. The innovative project deals with the development of novel microcarriers for application in cell therapies. The Eurostar jury selection for funded projects was based on scientific relevance as well as market need for novel products and services.
Today, exosomes are increasingly being investigated as a novel class of therapeutics that could explain the functions of many types of traditional cell therapies. To explain this trend, we contacted an esteemed exosome researcher, Dr. Ramon Coronado Schmilinski. Dr. Coronado Schmilinski was the previous Executive Director at Lester Smith Medical Research Institute and is the current VP Regulatory Affairs and Technology Transfer at Signature Biologics.
Exosomes are secreted by a wide range of cells, with virtually all living cells utilize exosome-mediated communication. Today, exosomes are being explored across a diverse range of therapeutic and diagnostic applications, with particular attention being given by the pharma industry to drug-loaded exosomes. Already, three pharmaceutical companies—Jazz Pharmaceuticals, Roche, and Boehringer Ingelheim—have signed partnership deals with exosome companies with royalties and milestone payments structured to deliver at or around $1B dollars.
Brian C. Handerhan, MBA, Takes on the Newly Created Role of Vice President, Operations for Cell X Technologies, Inc.
CLEVELAND, Ohio – Cell X Technologies’ CEO Peter Johnson MD, announced an important new hire in Brian C. Handerhan, MBA, who will take on the newly created position of Vice President, Operations. In this role, he will oversee sales, marketing, product development, and product management to drive growth in all cell therapy markets served by Cell X. Founded in 2017, Cell X Technologies, Inc. develops and provides equipment, software, and process management tools to support stem cell research and bio-fabrication.
Regenerative medicine is a fascinating topic, because it requires you to ask yourself: Would you want to live to be 200 years old? A scientist from Stanford University thinks that it’s possible – so much so that he has placed a bet that there is someone alive today who will live to be 200 years old. In recent decades, modern scientific developments have led to a dramatic increase in human lifespan. But, how much further can we go?
After choosing a name for your newborn, the next decision you’ll need to make is whether or not you want to save their cord blood. It’s a choice you will need to make swiftly, as the window to successfully store your child’s cord blood and cord tissue is a relatively small one. While there is a lot of information available for expecting parents, sifting through all the options to find the best cord blood bank can be overwhelming, especially if you’re not exactly familiar with all the terms and technology.
April 28, 2021, SUGAR LAND, Texas — Houston-area non-profit Hope Biosciences Stem Cell Research Foundation (HBSCRF) has received FDA authorization for a Phase II double-blind placebo controlled clinical trial to assess the efficacy and safety of multiple intravenous infusions of autologous adipose-derived mesenchymal stem cells to improve activities of daily living and quality of life in subjects with Parkinson’s Disease.
Organicell Receives Approval In Pakistan For The Use Of Zofin™ For Compassionate Grounds For COVID-19
Miami, FL (May 11, 2021) – Organicell Regenerative Medicine, Inc. (OTCMKTS: BPSR), a clinical-stage biopharmaceutical company dedicated to the development of regenerative therapies, announced its Zofin™ therapy has been approved by Pakistani regulators to be used for a COVID-19 patient on compassionate grounds. The approval by regulators is contingent on the strict adherence to the dosing protocols in use by clinical trial investigators conducting Organicell’s ongoing Phase I/II trial for COVID-19.
To date, 53 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 165 requests and issued 62, which means that a handful are not yet public knowledge. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.
Globally, companies have deployed an impressive range of business models for exploiting iPSC technology platforms. To date, iPSCs have found applications within academic research, drug discovery, toxicology testing, cell therapy, personalized medicine, disease modelling, industrial-scale manufacturing, biobanking, clean-meat production, and beyond.
Drug discovery involves high cost and uncertain outcomes. Only a few companies can take the risk of investing enormous amounts of money into research and testing, only to see undesirable side effects emerge during the final human clinical trials. Now, some medicines are being tested for safety on specialized cells developed from human pluripotent cell lines. Cells types which are frequently being using within drug discovery include heart (cardiomyocytes) and liver cells (hepatocytes), which are the organs where 80% of drug failures occur.
Continuously updated, this global list of CAR-T companies keeps expanding as new competitors enter the global marketplace. CAR-T cell therapy is as a type of immunotherapy that teaches T cells to recognize and destroy cancer. Read on to learn more about innovative CAR-T cell therapy companies and the novel technologies they are using to fight cancer.
Since the discovery iPSC technology 15 years ago, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated. Today, methods of commercializing iPSCs include cell therapy, disease modelling, drug development and discovery, toxicology testing, and personalized medicine. Other applications of iPSCs include their use as research products, as well as their integration into 3D bioprinting, tissue engineering, and clean meat production.
To characterize the rapidly expanding exosome market, BioInformant has released a 274-page report that explores growing demand for exosome therapeutics, diagnostics, research tools, and manufacturing technologies. It reveals trend rate data for exosome patents, grants, scientific publications, and trials. It highlights information about exosome industry partnerships, M&A activity, and financing events. It features company profiles for 107 global competitors from across the exosome marketplace. Importantly, it presents market size determinations by market segment with forecasts through 2028.