The Pipeline for iPSC-Derived Cell Therapeutics in 2024

Despite tremendous progress involving the use of induced pluripotent stem cells (iPSCs) within disease modeling and drug discovery applications, it has been a challenging path to achieve the use of iPSC-derived cellular therapeutics in human patients. At present, no iPSC-derived therapeutic has achieved regulatory approval worldwide. However, at least a dozen companies and organizations are conducting human trials with iPSC-derived therapeutics. Additionally, at least 62 companies worldwide are involved with the research development of iPSC-derived cellular therapeutics. This makes it an exhilarating time for the iPSC industry, which is evolving at breakneck pace. 

Optimize Your Upstream and Downstream Cell and Exosome Process Development

Leverage advanced equipment and expertise to develop a custom manufacturing processes that is aligned with regulatory standards, scalable from product development through clinical manufacturing, and designed to rapidly progress from where you are today into tomorrow’s potential advanced therapy. RoosterBio's Process Development Services help you build fit-for-purpose upstream and downstream processes that are economical, ready to scale, and specific to your product needs.

First Patient Treated in Phase 2 GvHD Trial

Melbourne, Australia; 5 March 2024 – Cynata Therapeutics Limited (ASX: “CYP”, “Cynata”, or the “Company”), a clinical-stage biotechnology company specialising in cell therapeutics, confirms that the first patient has been enrolled and treated in its Phase 2 clinical trial of CYP-001 in high-risk acute graft versus host disease (aGvHD). CYP-001 is Cynata’s Cymerus™ off-the-shelf iPSC[1]-derived MSC[2] product candidate for intravenous infusion, which previously generated very encouraging safety and efficacy results in a Phase 1 clinical trial in steroid-resistant aGvHD.[3] The US FDA has cleared an Investigational New Drug (IND) application for CYP-001 and granted the product Orphan Drug Designation[4] for the treatment of aGvHD.

PeptiGrowth Inc. is launching a Novel Synthetic Peptide TPO-alternative peptide (TPOR agonist)

March 26, 2024, Chiyoda-ku, Tokyo – PeptiGrowth Inc. (Headquarters: Chiyoda-ku, Tokyo, President: Jiro Sugimoto) has successfully developed a novel synthetic peptide called “TPO alternative peptide (TPOR agonist)” which has equivalent function to recombinant TPO (Thrombopoietin). PeptiGrowth's peptides are completely chemically synthesized and animal component-free, enabling xeno-free and chemically defined cell culture media.

Defined Bioscience Introduces Ready-CEPT™: A New Cell Viability Enhancer

San Diego, CA, April 1, 2024 – Defined Bioscience has announced the launch of Ready-CEPT™ 1000X, a new product designed to improve stem cell survival, growth, and health during critical processes such as routine passaging, cryopreservation, and freeze-thaw recovery. It introduces a reliable solution for researchers in stem cell research and biotechnology, focusing on enhancing cell viability. Ready-CEPT™ (also known as CEPT) combines key ingredients: chroman-1 (a ROCK inhibitor), emricasan (a pan-caspase inhibitor), polyamines (putrescine, spermine, and spermidine for cell growth), and trans-ISRIB (an ISR inhibitor), into a single formulation. Originating from research conducted at the NIH, Defined Bioscience brings the CEPT formulation to scientists worldwide.

2024 HEALinc Future Health Summit to Convene Experts in Longevity and Regenerative Health

Over April 28-30th, 2024, on Paradise Island in The Bahamas, the 2024 HEALinc Future Health Summit will be convening experts from across the fields of longevity, cellular medicine, and regenerative health. The event will bring together the world’s top scientists, investors, healthcare providers, longevity tech companies, and stem cell companies. By attending, you will be positioned to learn about cutting-edge future health medicines and technologies that can support peak performance at any age.

Aruna Bio Announces FDA Clearance of IND for Lead Program AB126, Enabling the First Exosome to Enter in Human Clinical Trials for a Neurological Indication

BOSTON and ATHENS, Ga., Jan. 16, 2024 — Aruna Bio, Inc., a pioneer in the development of neural exosome-based therapeutics for the treatment of neurodegenerative diseases, announced the U.S. FDA has cleared the Investigational New Drug (IND) application of lead program, AB126. This decision paves the way for the Phase 1b/2a clinical trial in acute ischemic stroke, which is expected to initiate in the first half of 2024. AB126 is an unmodified neural-derived exosome with an innate ability to traverse the blood brain barrier and shows evidence of anti-inflammatory and neuroprotective properties.

Patient Enrolment Completed in CYP-006TK Diabetic Foot Ulcer Clinical Trial

Melbourne, Australia; 8 April 2024 – Cynata Therapeutics Ltd. (ASX: CYP), a clinical-stage biotech company specialising in cell therapeutics, had announced the completion of patient enrolment in its Phase 1 clinical trial of CYP-006TK in diabetic foot ulcers (DFU). CYP-006TK is Cynata’s Cymerus™ iPSC-derived MSC topical wound dressing product candidate, which comprises MSCs seeded onto a novel silicon dressing. Due to reduced blood flow, patients with diabetes are at risk of developing non-healing wounds on the feet/lower limbs, which are also known as DFU. In this trial, CYP-006TK is being investigated as a potential treatment to promote wound healing in patients with DFU.

How Human iPSCs are Revolutionizing Drug Discovery in 2024

Drug discovery involves high cost and uncertain outcomes. Only a few companies can take the risk of investing enormous amounts of money into R&D, only to have undesirable side effects emerge during later stage trials. Thankfully, the discovery of iPSCs and recent achievements in producing iPSC-derived cell types from patients and healthy individuals, along with efficient gene modification techniques, has led to remarkable opportunities to model human disease. While dozens of companies offer human iPSC-derived cell types to support drug discovery and toxicology testing, there are a few clear market leaders, which this article explores.

Organizations Who are Conducting Trials with iPSC-Derived Cellular Therapeutics

iPSCs, also called iPS cells, hold immense potential for regenerative medicine, disease modeling, and drug discovery, because they offer a non-controversial and patient-specific source of stem cells. Unlike embryonic stem cells, which are derived from embryos and raise ethical concerns, iPS cells can be generated from a patient's own cells, reducing the risk of rejection by the immune system when used within regenerative medicine. Despite their enormous promise, no iPSC-derived cellular therapeutic has yet to receive regulatory approval. With this said, there are at least a dozen organizations that are conducting human trials involving iPSC-derived cellular therapeutics. Learn their identities and product candidates here. 

Novo Holdings to Acquire the CDMO Catalent for a Staggering $16.5 Billion Enterprise Value

COPENHAGEN, Denmark and SOMERSET, N.J., February 5, 2024 – Catalent, Inc. (NYSE: CTLT), a leader in enabling the development and supply of better treatments for patients worldwide, and Novo Holdings, a holding and investment company that is responsible for managing the assets and wealth of the Novo Nordisk Foundation,  announced that they  entered into a merger agreement under which Novo Holdings will acquire Catalent in an all-cash transaction that values Catalent at an astounding $16.5 billion on an enterprise value basis.

Kenai Therapeutics Emerges with $82M to Advance Its Allogeneic, iPSC-Derived Therapy for Parkinson’s Disease

SAN DIEGO, CA, Feb 29, 2024 — Kenai Therapeutics, a biotechnology company leveraging induced pluripotent stem cell (iPSC) technology to discover and develop a platform of allogeneic neuron replacement cell therapies for neurological disorders, announced an $82 million Series A financing co-led by Alaska Permanent Fund Corporation, Cure Ventures and The Column Group, with participation from Euclidean Capital and Saisei Ventures. Kenai Therapeutics previously raised seed funding under the name Ryne Bio.

Endonovo Therapeutics Files Patent Application for Cell Therapy Created Using Cord Blood and Tissue Derived Cells

LOS ANGELES, CA– Oct 27, 2015 – Endonovo Therapeutics, Inc. (OTCQB: ENDV), an innovative biotechnology company developing bioelectronics-based products and therapies for regenerative medicine, announced that it has filed a patent application with the United States Patent and Trademark Office (USPTO) on a perivascular cell mixture created using its proprietary Cytotronics™ Platform that would allow for the wide spread use of cord blood in the treatment of malignant and non-malignant hematological disorders.

Revolutionary Elephant iPSC Milestone Reached in Colossal’s Woolly Mammoth Project

DALLAS, Texas, March 06, 2024 – Colossal Biosciences, the world’s first de-extinction company, announced that their Woolly Mammoth team has achieved a global-first iPSC (induced pluripotent stem cells) breakthrough. This milestone advancement was one of the primary early goals of the mammoth project and supports the feasibility of future multiplex ex utero mammoth gestation.

Coeptis Therapeutics to Pursue Phase 2 Program Using Allogeneic Cord-Blood Derived NK Cell Therapy 

WEXFORD, PA, Feb 7, 2024 — Coeptis Therapeutics Holdings, Inc. (Nasdaq: COEP), a biopharmaceutical company developing innovative cell therapy platforms for cancer and infectious diseases, announced plans to pursue DVX201, its proprietary allogeneic stem cell expansion and directed differentiation platform of unmodified natural killer (NK) cell therapy, as an antiviral treatment addressing various respiratory viral infections that lead to hospitalization, including the “tripledemic” of influenza, COVID-19 and respiratory syncytial virus (RSV).

Cellipont Bioservices and Wugen Sign Agreement for the Clinical Manufacture of Wugen’s Off-the-shelf CAR-T Cellular Therapies

The Woodlands, TX, and St. Louis, MO, March 14, 2024 – Cellipont Bioservices, a leading cell therapy Contract Development and Manufacturing Organization (CDMO), and Wugen, Inc., a clinical-stage biotechnology company developing allogeneic, off-the-shelf cell therapies to treat a broad range of hematological and solid tumor malignancies, announced the signing of an agreement for the manufacturing of their CAR-T cell therapies.

New “Placental Tissue Amendment” Bill Clarifies Disclosures Required to Perform Stem Cell Therapies in Utah

The “Placental Tissue Amendment Bill“, part of SB 0199, recently passed by the Utah State Legislature, introduces specific requirements for healthcare providers who plan to adminster treatments involving placental stem cells. The key focus of this bill is to ensure that patients receive adequate disclosures regarding the nature of the treatment, particularly if it involves stem cell therapy that hasn’t been approved by the U.S. FDA. 

Asgard Therapeutics Snags €30M to Develop In Vivo Direct Cell Reprogramming Technologies for Cancer

LUND, Sweden, March 14, 2024 — Asgard Therapeutics, a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy, announced a €30 million Series A financing. The funds will be used to support development of Asgard´s lead program AT-108 to IND-readiness by 2026, expand and reinforce its research and development team, and fuel new reprogramming modalities and delivery platforms to strengthen the pipeline. The round was co-led by RV Invest and Johnson & Johnson Innovation – JJDC, Inc., with participation from existing investors Novo Holdings, Boehringer Ingelheim Venture Fund and Industrifonden.

ExoXpert, a CDMO launched by EXO Biologics to service increasing worldwide demand for exosomes

Liege, Belgium, March 1, 2024 – EXO Biologics SA, a Belgian biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with high unmet medical needs, announced the worldwide launch of ExoXpert(TM), a contract development and manufacturing organization (CDMO) specializing in exosomes. ExoXpert offers a MSC-based exosome manufacturing platform used in European clinical trials. ExoXpert is a wholly owned subsidiary of EXO Biologics(TM).

Induced Pluripotent Stem Cell (iPSC) Industry Trends Dominating in 2024

Induced pluripotent stem cells (iPSCs and iPS cells) are laboratory-made pluripotent stem cells that are produced using non-controversial adult cells. They are derived from somatic cells through reprogramming and turning on the expression of specific pluripotency genes. This reprogramming can be achieved using different techniques with varying efficiencies. Read on to learn about iPSC industry trends that are dominating today in 2024.

Allogeneic iPSCs Gaining Momentum Due to Ease of Access and Economies of Scale

While autologous iPSC-based cell therapies are patient-specific, allogeneic iPSC-based cell therapies use donor-derived cells as a starting material. In recent years, a greater number of allogeneic iPSC-derived cell therapies are being studied than autologous therapies. One of the advantages of allogeneic therapy is that a large number of doses can be generated simultaneously from a single batch of iPSCs and banked.

Pivotal Trends Impacting the Global Cord Blood and Tissue Industry in 2024

The cord blood industry came into existence in the early 1990’s with the formation of several cord blood banks in the U.S. and worldwide. It has been 40 years since it was first proposed that stem and progenitor cells were present in human cord blood. Today, the cord blood and tissue industry is evolving in new and unexpected ways. This article explores six key trends that are shifting and shaping the commercial landscape of this industry, forever. 

Historical Data on the Global Cord Blood and Tissue Banking Services Market

The Global Cord Blood and Tissue Banking Services Market is composed of revenue produced by companies that are engaged with the collection, processing, storage, and release of umbilical cord blood and tissue products. The global cord blood industry came into existence in the early 1990’s with the formation of a handful of cord blood banks within the United States. Shortly thereafter, these companies began being established in other regions as well.

LifeCell International, India’s Largest Cord Blood Bank

LifeCell is India’s largest cord blood bank, offering stem cell banking services and diagnostic testing. Established in 2004, its BabyCord program is one of the largest cord blood banking programs in the region, with 50,000+ parents preserving newborn stem cells each year and 375,000 units cryopreserved.

What Is An RMAT? List of Publicly Announced RMAT Designations (90)

To date, what number of U.S. FDA-approved RMATs have been issued and to whom have they been awarded? The answer is that 90 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced by biotech and pharma companies. However, the FDA states it has received 248 requests and issued 101, which means that a handful are not yet public knowledge. Therefore, a few companies are operating in stealth mode with regard to their RMAT designations and approximately 41% of RMAT applications get approved (101 approvals / 248 applications = 40.7%).

CAR-T Cell Companies Proliferate: List of CAR-T Companies Worldwide

CAR-T cell therapy is a type of immunotherapy that teaches T cells to recognize and destroy cancer.  This article provides a comprehensive list of CAR-T therapy companies worldwide. Read on to learn about CAR-T market competitors and the innovative technologies they are using to fight cancer.

The Emerging Role of Exosome Therapeutics in 2024

Exosomes are extracellular vesicles (EVs) of endosomal origin that range in size between 30 and 150 nanometers. Exosomes are secreted by a wide range of cells, since virtually all living cells utilize exosome-mediated communication. Companies participating in exosome sector are of two types: companies using “naïve” exosomes and companies using “engineered” exosomes. Naïve exosomes are the exosomes naturally released by cells, while “engineered” exosomes are those loaded with additional biomolecules in the laboratory.

What Are iPSCs, Who is Funding Them, and What Trials are Underway?

Induced pluripotent stem cells (iPSCs) can be made by reprogramming mature adult cells back into an embryonic-like state. Derived from skin or blood cells, iPSCs are not controversial, because they are made from adult cells. As pluripotent stem cells, they can give rise to all of the tissues that form the human body. This articles explores key benefits of iPSCs, leading entities who are funding iPSC research and development, and groundbreaking iPSC trials that are being conducted in human patients worldwide. 

Mesenchymal Stem Cells / Medicinal Signaling Cells (MSCs) - Advances & Applications

MSCs are essential raw materials for many regenerative medicine products, including cell-based therapies, engineered tissues, exosome products, and cultured meat production. Growing attention is also being given to technologies to support commercial-scale production of MSCs. The first MSC-based cell therapy product was approved in 2010 in South Korea. To date, ten MSC-based cell therapy products have gained approval, as well as 19 MSC-based bone matrices. This global strategic report presents market size figures with forecasts through 2030, as well as rates of MSC clinical trials, scientific publications, patents, funding events, strategic collaborations, and beyond.

Global Exosome Market – Market Size, Forecast, Trials, and Trends

Exosome technologies have been developing rapidly in recent years and substantial growth is expected for the market as they get integrated into the fields of liquid biopsy, precision medicine, and regenerative medicine. The report reveals trend rate data for exosome patents, grants, scientific publications, and clinical trials. It highlights information about exosome industry partnerships, M&A activity, and financing events. It features company profiles for 118 global competitors from across the exosome marketplace. Importantly, it also presents market size determinations by segment with forecasts through 2030.

Global Induced Pluripotent Stem Cell (iPSC) Industry Report - Market Size, Trends, and Forecasts

In addition to companies who are developing iPSC-derived cellular therapeutics, there are an ever-growing number of market competitors who are commercializing iPSC products for use across a diverse range of applications. These applications include drug development and discovery, disease modeling, toxicology testing, and personalized medicine, as well as tissue engineering, 3D bioprinting, and clean meat production. This report reveals critical industry metrics, including but not limited to rates of scientific publications, clinical trials, patents, funding events, strategic partnerships, and M&A activity. It identifies all major iPSC market competitors worldwide. Importantly, it presents global market size figures further segmented by iPSC Application, Technology, Cell Type, and Geography (North America, Europe, Asia/Pacific, and RoW), accompanied by future market projections through 2030.

Interested to learn about other market segments, such as MSCs, iPSCs, CAR-T cells, exosomes, cord blood and tissue, or the cell therapy industry at large? 

Explore the BioInformant Shop.

*Want to advertise in this newsletter? Contact us at Info@BioInformant.com.