Industry updates from the field of stem cell research and regenerative medicine in January 2025

ABSTRACT

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in January 2025.

1. Business development

1.1. Collaboration agreement: Aspen Neuroscience and Mytos

Aspen Neuroscience (CA, USA; www.aspenneuroscience.com) has advanced its plans for automating production of ANPD001, including a capacity expansion for autologous manufacturing at its San Diego-based GMP facility [1]. ANPD001, an investigational cell therapy program for treating people with Parkinson’s disease (PD), is currently being studied in the company’s ASPIRO Phase 1/2a clinical trial [2].

Designed and built for the manufacture and testing of ANPD001, this 2000 m² (22,000 sq. ft.) facility contains an additional 750 m² (8,000 sq. ft.) to accommodate future automation.

As part of this expansion, Aspen has entered into a collaboration agreement with Mytos (UK; www.mytos.bio), an automated cell manufacturing company, to focus on advancing plans to automate production of dopaminergic neuronal precursor cells (DANPCs) for ANPD001.

Designed to replace lost dopamine-producing neurons in the brain, Aspen differentiates these DANPCs from patient-specific induced pluripotent stem cells (iPSCs), derived from the patient’s own skin cells. This autologous approach has the potential of restoring lost function without the need for immunosuppressive drugs.

The collaboration with Mytos addresses an important step for automating the manufacturing process for differentiation of DANPCs from iPSCs. The Mytos platform, combined with Aspens’ bioinformatics and other enabling technologies, will allow Aspen to further automate production of ANPD001 and treat greater numbers of patients with their own cells.

1.2. Collaboration agreement: Orna and Vertex

Orna Therapeutics (MA, USA; www.ornatx.xom), through its wholly owned subsidiary ReNAgade Therapeutics, has announced a three-year strategic research collaboration with Vertex Pharmaceuticals (MA, USA; www.vrtx.com) to utilize Orna’s novel and proprietary lipid nanoparticle (LNP) delivery solutions to enhance Vertex’s efforts in developing next generation gene editing therapies for patients with Sickle cell disease (SCD) and Transfusion-dependent beta-thalassemia (TDT) [3].

Under the terms of the agreement, Orna will receive upfront payments of US$65 million, including an investment in the form of a convertible note, and is eligible to receive up to US$635 million based upon the achievement of specified pre-clinical, research, development, regulatory and commercial milestones related to SCD/TDT products. Additionally, Orna is further eligible to receive up to US$365 million in additional option fees and milestones per product for up to ten additional products if Vertex options rights in additional indications. Orna will be eligible to receive tiered royalties on future net sales of any products that may result from this collaboration. Vertex is funding the three-year research collaboration and holds an option to extend the research collaboration term.

Orna Therapeutics is dedicated to designing and delivering a new class of fully engineered circular RNA (oRNA®) therapeutics to unlock the potential of RNA medicine to treat diseases anywhere in the body. Orna’s circular RNA transcripts have advantages over traditional mRNA approaches, including simplified production, improved formulation into lipid nanoparticles, and superior protein expression

1.3. Licensing agreement: Synthego and Astra Zeneca

Synthego (CA, USA; www.synthego.com), a supplier of CRISPR solutions, has entered a strategic licensing agreement with global biopharmaceutical company AstraZeneca (UK; www.astrazeneca.com) [4]. This agreement grants Synthego a global license to manufacture and distribute AstraZeneca’s novel CRISPR gene editing enzyme, eSpOT-ON, recently published as engineered ePsCas9, to the worldwide research community [5]. This state-of-the-art nuclease, developed by AstraZeneca, will be paired with Synthego’s best-in-class gRNA to enable improved health outcomes.

Under the terms of the agreement, Synthego also receives exclusive commercialization rights including the right to sublicense this nuclease for therapeutic and commercial use.

1.4. Partnership agreement: Cellipont and Secretome

Cellipont Bioservices (TX, USA; www.cellipont.com), a cell therapy Contract Development and Manufacturing Organization (CDMO), and Secretome Therapeutics (TX, USA; www.secretometherapeutics.com), a biotechnology company advancing therapies derived from neonatal cardiac progenitor cells (nCPCs), have announced a partnership for cGMP manufacturing of Secretome Therapeutic’s lead asset STM-01 [6]. Under this partnership, Cellipont will provide technology transfer, analytical method transfer, and cGMP manufacturing for the STM-01 Master Cell Bank.

1.5. Partnership agreement: Gingko and Universal Cells

Ginkgo Bioworks (MA, USA; www.ginkgo.bio), has announced a research collaboration with Universal Cells (WA, USA; https://universalcells.com), an Astellas company (Japan; www.astellas.com), to optimize next-generation iPSC-derived cancer cell therapies [7]. By combining Universal Cells’ proprietary iPSC-derived cell technologies with Ginkgo’s expertise in design and screening large CAR libraries and its high throughput, multimodal immune cell engineering platform, the companies aim to accelerate the development of more potent and durable cell therapies while maintaining manufacturability at scale.

1.6. Partnership agreement: Orna and Simnova

Orna Therapeutics (USA, MA; www.ornatx.xom), and Simnova Biotech (China; www.simnovabio.com) have announced the expansion of their strategic collaboration to include BCMA as a designated biological target for RNA-based therapeutics development [8]. This partnership leverages Orna’s circular RNA (oRNA®) technology and Simnova’s expertise in cell therapy to deliver transformative treatments for patients worldwide.

Under the agreement, Simnova will pursue the research and development and commercialization of in vivo panCar cell therapies targeting BCMA in greater China and Orna will retain all rights for development and commercialization in the rest of the world. Each party will have the right to receive an upfront payment within their respective licensed territories and is eligible for clinical development, regulatory, and commercialization milestones as well as royalties on any approved products derived from the collaboration.

1.7. Partnership agreement: Viralgen and Axovia

Viralgen (Spain; https://viralgen.com) and Axovia Therapeutics (UK; https://axoviatherapeutics.com) are joining forces to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS) [9]. BBS is a genetic condition that causes multi-system disorders, with obesity and progressive retinal dystrophy being among the most debilitating symptoms. This disease appears in individuals carrying biallelic mutations in the BBS1 gene, leading to progressive vision loss and complete blindness, among other manifestations. This partnership will combine Axovia’s expertise in gene therapy development with Viralgen’s AAV manufacturing capabilities.

1.8. Strategic partnership agreement: Verismo and IFLI

Verismo Therapeutics (PA, USA; www.verisomtherapeutics.com), a clinical-stage biotechnology company developing the novel KIR-CAR platform for solid tumors and blood cancers, has announced a strategic investment from the Institute for Follicular Lymphoma Innovation (IFLI; CA, USA; www.i-fli.org), a global nonprofit foundation dedicated to advancing treatment options for follicular lymphoma (FL) [10]. This partnership aims to enhance Verismo’s SynKIR™-310 pipeline and provide catalytic investment for the pipeline’s FL arm, accelerating the development of next-generation cell therapies that address high unmet medical needs.

The investment from IFLI will help advance Verismo’s SynKIR™-310 pipeline, which aims to treat FL along with other non-Hodgkin lymphoma (NHL) subtypes. IFLI’s deep expertise in FL will be crucial in guiding Verismo through FL-specific clinical considerations and aiding in patient recruitment.

The KIR-CAR platform is a multi-chain CAR T cell therapy and has been shown in preclinical animal models to be capable of maintaining antitumor T cell activity even in challenging tumor microenvironments. Using NK cell derived KIR and DAP12 split signaling provides a novel combined activation and co-stimulation separate from the usual T cell stimulation pathways. It also enables sustained chimeric receptor expression and improves KIR-CAR T cell long-term function. This results in prolonged T cell functional persistence and leads to regression of solid tumors in preclinical models that are resistant to traditional CAR T cell therapies.

Therapeutics is currently running a multicenter, open-label study of a single infusion of SynKIR™-310 in participants with post-CAR T and CAR-naive relapsed/refractory B-NHL [11], including FL. This clinical study is a basket trial in B-NHL subtypes. The design includes two dose levels and an expansion cohort at the Recommended Phase 2 Dose (RP2D), with a total enrollment of up to 18 patients. With IFLI’s investment totaling up to US$4 M over 3 years, Verismo aims to expand the number of FL-focused clinical sites to enroll more FL patients.

2. Achievements, launches …

2.1. Astraveus

Astraveus SAS (France; www.astraveus.com), the Benchtop Cell Factory company, has announced the first ever successful end-to-end production of CAR-T cells within a microfluidic benchtop system [12]. Using its fully automated Lakhesys Benchtop Cell Factory^TM, Astraveus has been able to demonstrate the potential of its unique microfluidic cell therapy processing approach, which has the ability to decrease manufacturing costs and increase throughput by an order of magnitude.

The fully automated Lakhesys Benchtop Cell Factory integrates the entire cell therapy manufacturing process and onboard analytics into an all-in-one system, harnessing advanced microfluidic technology for optimal cell quality and efficient processing. Through scalable parallelization from pre-clinical to commercial stages, the Lakhesys Benchtop Cell Factory drastically reduces cost of goods, minimizes required lab space, and eliminates cell stress throughout the manufacturing process.

2.2. Bit.Bio

bit.bio (UK; www.bit.bio), a cell programming company, has launched its ioTracker Cells range, starting with GFP ioMicroglia, a tool designed to accelerate complex neurodegenerative disease research and drug discovery [13]. This innovative new range represents a strategic expansion of bit.bio’s ioCells Toolkit, introducing advanced live-cell imaging assay capabilities to further enhance the functionality of its flagship products.

2.3. CellFE

CellFE® (CA, USA; www.cellfebiotech.com), a non-viral gene editing company, has launched Resting T Cell Kit CellFE T-Rest™, a first-in-class cell manufacturing transfection media product designed specifically for quiescent T cell workflows [14]. The novel kit supports a fully optimized gene editing workflow that utilizes resting T cells as a starting material, offering a new paradigm for cell therapy manufacturers to develop safer and more potent therapies compared to traditional workflows.

2.4. EditCo

EditCo Bio (CA, USA; www.editco.bio), a leader in cutting-edge genetic engineering and cellular model solutions, has launched XDel Knockout Cells, an innovative product designed to revolutionize the field of CRISPR gene editing [15].

Traditional CRISPR knockout methods often depend on a single guide RNA that, in tandem with SpCas9, generates assorted insertions or deletions (indels) at the target cut site. This approach can be unpredictable, leading to variable edits and incomplete knockouts.

EditCo’s smart informatics generates a multi-guide design which is composed of up to 3 sgRNAs targeting a single gene of interest. The guides are spatially coordinated and work cooperatively to induce a guided repair that results in fragment deletion in an early exon, making it the most reliable knockout strategy compared to other pooled strategies.

2.5. L’oreal

L’Oréal Groupe (France; www.loreal.com) unveiled Cell BioPrint, a tabletop hardware device that provides personalized skin analysis in just five minutes, using advanced proteomics [16]. The L’Oréal Cell BioPrint device also features NanoEntek’s (South Korea; www.nanoentek.com) exclusive microfluidic lab-on-a-chip technology, which leverages some of NanoEnTek’s 100+ patents to measure the presence of L’Oréal’s groundbreaking protein biomarkers in five minutes. It works through a simple, noninvasive process. Cell BioPrint is scheduled to pilot with a L’Oréal brand in Asia later in 2025.

2.6. Ncardia

Ncardia (Belgium; www.ncardia.com), an iPSC technology company, has launched Ncyte® Heart in a Box and Ncyte® Microglia, designed to drive innovation in research and drug discovery [17,18].

The Ncyte® Heart in a Box system is a 3D cardiac microtissue model derived from hiPSC and integrates three essential cardiac cell types: ventricular cardiomyocytes, endothelial cells, and cardiac fibroblasts. Together, these high-purity components form a physiologically relevant microtissue that replicates the complexity and functionality of the human heart.

This innovative model offers a robust platform for studying heart development, disease progression, and cellular interactions within the cardiac environment. Researchers can leverage the enhanced predictive power of Ncyte® Heart in a Box for therapeutic development, safety screening, and precision medicine applications.

Ncyte® Microglia are hiPSC-derived models that closely mimic primary human microglia, exhibiting key functional characteristics such as phagocytosis, cytokine signaling response, and the expression of microglial markers including IBA1, TREM2, and TMEM119. These features make Ncyte® Microglia highly suitable for studying microglial behavior in disease-relevant contexts, assessing immune responses, and enabling high-throughput drug screening applications. Microglia is increasingly recognized as a key factor in the pathology of several neurodegenerative diseases, including Alzheimer’s disease (AD), Amyotrophic Lateral Sclerosis (ALS), and Parkinson’s disease (PD).

2.7. Nucleus biologics

Nucleus Biologics (CA, USA; https://nucleusbiologics.com), a cell culture media company, has received EXCiPACT certification (Belgium; www.excipact.org) for Pharmaceutical Excipients and Pharmaceutical Auxiliary Materials (PAMs) and ISO 9001 certification from Société Générale de Surveillance (SGS; Switzerland; www.sgs.com) [19]. This Excipient GMP certification will better support the cell culture media, buffers, reagents, and supplements needs for our cell and gene therapy customers. This certification, in addition to our ISO 13,485 certification, underscores Nucleus Biologics’ commitment to delivering the highest quality products that meet the most stringent safety, consistency, and compliance requirements of the biopharmaceutical industry.

EXCiPACT is an internationally recognized certification standard specifically designed for excipient manufacturers. It ensures that facilities adhere to the highest standards of GMP and Good Distribution Practice (GDP). Nucleus Biologics is only the second cell culture media manufacturer and the only West Coast USA and custom supplier to be certified to EXCiPACT for PAMs.

2.8. ONI

Oxford Nanoimaging (ONI; UK; https://oni.bio), a life science tools company focused on developing super-resolution microscopy, has launched the Aplo Scope, a single molecule super-resolution microscope that enables scientists to observe and measure molecular interactions within cells and tissues with unparalleled precision [20].

2.9. Parse

Parse Biosciences (WA, USA; www.parsebiosciences.com), a single cell sequencing company, has announced a significant expansion of its Evercode™ WT Mega Kit functionality [21]. Researchers can now analyze up to 384 samples and 1 million cells in a single run, unlocking new possibilities for high-throughput studies.

2.10. Pluristyx

Pluristyx (WA, USA; https://pluristyx.com), a provider of GMP iPSC technologies, has announced the immediate availability of the PSXi013 iPSC line made under GMP conditions [22]. PSXi013 has been reprogrammed using mRNA technology. As the lowest passage iPSC bank available on the market (delivered at Passage 10), PSXi013 effectively minimizes the risk of genetic drift, allows the customer to select a clone on their schedule, and provides a more stable and clinically relevant starting material versus higher passage cell banks. PLSXi013 is available for immediate licensing with a simplified structure directly through Pluristyx.

3. Clinical trials

3.1. Pluripotent stem cells

3.1.1. Aspen Neuroscience

Aspen Neuroscience (USA, CA; www.aspenneuroscience.com) has successfully completed dose escalation and the first two cohorts of patients in the ASPIRO study, an ANPD001 Phase 1/2a clinical trial in Parkinson’s Disease (PD) [2,23]. The Autologous-derived Study of a Parkinson’s Investigational Regenerative therapy in an Open-label trial (ASPIRO) was launched in 2024 to assess ANPD001 in patients with moderate-to-severe PD. The study is examining escalating doses, with the primary endpoint being safety and tolerability of ANPD001. Secondary endpoints include improvement in “on” time, when patients experience periods of symptom control, and improvements in motor symptoms and quality of life based on standard PD rating scales.

Aspen’s manufacturing process starts from a small sample of the patient’s own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) and then differentiation of the iPSCs into DANPCs. These DANPCs are transplanted into the putamen, replacing cells that were lost or damaged due to disease. The quality of each person’s cells is assessed at every manufacturing stage using Aspen’s proprietary machine learning-based genomics tests. ANPD001 has received Fast Track designation by the US FDA.

3.1.2. Gameto

Gameto (NY, USA; www.gametogen.com), a clinical-stage biotech company developing iPSC-based therapies for women’s health, has announced that the US FDA has cleared its Investigational New Drug (IND) application for Fertilo, to enable the launch of the first US-based Phase 3 clinical trial for an iPSC-based therapy [24]. Fertilo is Gameto’s ovarian support cell (OSC) technology, designed to mature eggs outside the body using iPSC-derived cells. Unlike traditional methods, which rely on 10–14 days of high-dose hormonal stimulation to mature eggs, Fertilo uses engineered, young ovarian support cells to recreate the natural egg maturation process in a dish. This process replaces 80% of hormone injections required with traditional in vitro fertilization (IVF), and reduces the duration of treatment cycles to just 2–3 days, offering patients a more comfortable and less invasive experience. This also significantly reduces risks such as ovarian hyperstimulation syndrome and alleviates side effects caused by high-dose hormone treatments.

Gameto will launch its Phase 3 clinical trial across 15 sites in the United States. The study will assess key outcomes, including embryo development, pregnancy rates (as a measure of efficacy), and maternal health and live birth rates (as safety endpoints). The protocol involves up to two days of hormonal injections stimulation, with up to two embryo transfers, and is open to couples struggling with infertility for at least one year. Participants who do not achieve pregnancy during the trial will be offered a voucher for a second attempt. The Phase 3 study will be a double-blind, randomized controlled trial to ensure robust data collection and analysis.

4. Gene therapy

4.1. Genprex

Genprex (TX, USA; www.genprex.com), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, has announced that the first patient has been enrolled and dosed in the Phase 2 expansion portion of the Company’s Acclaim-3 clinical study of Reqorsa® Gene Therapy (quaratusugene ozeplasmid) in combination with Tecentriq® (atezolizumab) as maintenance therapy for patients with extensive stage small cell lung cancer (ES-SCLC) [25,26].

Tumor suppressor genes are often deleted or inactivated early in the process of cancer development. REQORSA contains a plasmid that expresses TUSC2, a tumor suppressor gene protein. Nearly 100% of SCLCs have reduced or no TUSC2 protein expression, and 41% completely lack TUSC2 protein expression. Preclinical studies support the hypothesis that re-expressing the TUSC2 protein may lead to improved clinical efficacy in combination with Tecentriq, a monoclonal antibody against PD-L1.

5. Other

5.1. Aethlon

Aethlon Medical (CA, USA; www.aethlonmedical.com), a medical therapeutic company focused on developing products to treat cancer and life-threatening infectious diseases, has announced a significant milestone: the treatment of the first patient with the Hemopurifier in its Australian safety, feasibility and dose-finding clinical trial of the Hemopurifier [27]. This trial is designed for patients with solid tumors who have stable or progressive disease during anti-PD-1 monotherapy treatment, such as Keytruda® (pembrolizumab) or Opdivo® (nivolumab).

The Aethlon Hemopurifier is an investigational medical device designed to remove enveloped viruses and tumor-derived extracellular vesicles from circulation. The Hemopurifier is an extracorporeal device that is used in concert with a blood pump. The device incorporates plasma separation, size exclusion, and affinity binding to an affinity resin containing a plant lectin. Mannose on the surface of enveloped viruses and extracellular vesicles binds to the plant lectin within the device. Extracellular vesicles released from solid tumors have been implicated in the spread of cancers known as metastasis as well as in the resistance to immunotherapy and chemotherapeutic agents. Removal of enveloped viruses and extracellular vesicles has been observed in in vitro studies and in human subjects. The Hemopurifier holds a US FDA Breakthrough Device for the treatment of individuals with advanced or metastatic cancer who are either unresponsive to or intolerant of standard-of-care therapy. The Hemopurifier also holds an FDA Breakthrough Device designation and an open Investigational Device Exemption (IDE) application related to the treatment of life-threatening viruses that are not addressed with approved therapies.

6. Regulations, approvals, acquisitions …

6.1. Acquisitions

6.1.1. Takara Bio and Curio

Takara Bio USA (Japan; www.takarabio.com) has announced the acquisition of Curio Bioscience (CA, USA; https://curiobioscience.com), a company in the field of spatial genomics. This strategic acquisition combines two innovative spatial biology platforms with Takara Bio’s industry-leading portfolio of single-cell genomics tools [28].

Curio’s advanced Trekker and Seeker technologies integrate spatial information with molecular data, enabling researchers to transform single-cell sequencing data into spatially resolved maps. This transformation offers high-resolution insights into the organization and function of cells within their native tissue environments.

7. Green light

7.1. JW Therapeutics

JW Therapeutics (China; www.jwtherapeutics.com), a biotechnology company focused on developing, manufacturing and commercializing cell immunotherapy products, announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China (NMPA) granted Breakthrough Therapy Designation for Carteyva® (relmacabtagene autoleucel) as second-line treatment in adults with relapsed or refractory large B-cell lymphoma [29]. Carteyva is an anti-CD19 autologous chimeric antigen receptor T (CAR-T) cell immunotherapy product independently developed by JW Therapeutics based on a CAR-T cell process platform of Juno Therapeutics (a Bristol Myers Squibb company).

7.2. Ventris Medical

Ventris Medical (CA, USA; www.ventrismedical.com), a privately held orthobiologics and tissue regeneration company, has announced that the US FDA has granted 510(k) clearance for Backpack® (Porous Biologic Scaffold, K240765) [30]. Backpack represents a new class of biomaterials designed for the optimization of cell proliferation and bone formation and is used in orthopedic and spinal fusion procedures. Backpack is available in two versions. One version consists of a collagen mesh pouch prefilled with our osteoinductive Allocell® AF fibers and in the second version, the collagen mesh pouch is prefilled with our surface activated Amplify® granules.

8. Capital market & finances

8.1. Colossal

Colossal Biosciences (TX, USA; https://colossal.com), the world’s first de-extinction company, has announced US$200 M in a Series C financing [31]. Since launching in September 2021, Colossal has raised US$435 M in total funding. This latest round of capital places the company at a US$10.2 billion valuation. Colossal will leverage this latest infusion of capital to continue to advance its genetic engineering technologies while pioneering new revolutionary software, wetware and hardware solutions, which have applications beyond de-extinction including species preservation and human healthcare.

8.2. Mesoblast

Mesoblast (Australia; www.mesoblast.com) has completed a global private placement primarily to Mesoblast’s existing major US, UK, and Australian shareholders raising US$160 (A$260) million at A$1.50 per share [32].

Proceeds will be used to fund:

• US commercial launch of Ryoncil® (remestemcel-L) for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients;

• Clinical development including acceleration of the second Phase 3 study in inflammatory chronic low back pain (CLBP) which is actively recruiting;

• Expansion of commercial manufacturing activities in preparation for product uptake and demand; and

• Working capital and general corporate purposes.

Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of SR-aGvHD in children 2 months and older is the first US FDA-approved mesenchymal stromal cell (MSC) therapy.

8.3. Vertex

Vertex Pharmaceuticals (MA, USA; www.vrtx.com) has announced a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel) [33].

The reimbursement agreement comes as the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending CASGEVY’s use in the NHS. It means that eligible SCD patients in England now have access to the therapy following the prior agreement for transfusion-dependent beta thalassemia (TDT) patients announced last August.

Funding Statement

This paper was not funded.

Disclosure statement

Dusko Ilic is a member of the Regenerative Medicine Editorial Board. They were not involved in any editorial decisions related to the publication of this article. Dusko Ilic has received an honorarium from Taylor & Francis for the contribution of this work. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.