Industry updates from the field of stem cell research and regenerative medicine in May 2024

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 May 2024.

1. Business Development

1.1. Collaboration agreement: Cellular Origins & Catapult

Cellular Origins (UK; https://cellularorigins.com), a company focused on enabling scalable, cost-effective and efficient manufacture of cell and gene therapies (CGTs), and the Cell and Gene Therapy Catapult (CGT Catapult; UK; https://ct.catapult.org.uk), an independent innovation and technology organization specializing in the advancement of the cell and gene therapy industry, have announced a collaboration to demonstrate universal automation of CGT manufacturing [1].

The partnership will undertake the physical and digital integration of existing bioprocessing equipment at the CGT Catapult's Stevenage Manufacturing Innovation Centre (MIC) using Cellular Origins' robotic platform, Constellation™. By bringing together CGT Catapult's expertise in GMP operations and optimization with Cellular Origins' Constellation, the goal will be to evidence the benefits of automation in real world applications and advance the fields' understanding of how automation can impact the barriers to scale that have held progress back.

1.2. Collaboration agreement: Astellas & YASKAWA

Astellas Pharma (Japan; www.astellas.com) has signed a memorandum of understanding with YASKAWA Electric Corporation (Japan; www.yaskawa-global.com) to begin discussions on the creation of an innovative cell therapy ecosystem through the integration of pharmaceutical and robotics technologies [2]. This memorandum is legally non-binding and will lead to further specific discussions between the two companies in the future.

The complexity of product manufacturing is a significant challenge to commercializing cell therapy. Substantial investments are required to develop complex manufacturing processes, transfer technology among manufacturers and establish large-scale manufacturing facilities for commercialization. This makes it difficult for startups and academia to commercialize cell therapy independently.

Since the end of 2017, Astellas has been advancing drug discovery and manufacturing technology research in cell therapy through the introduction of the humanoid robot ‘Maholo’, developed by the Robotic Biology Institute, a subsidiary of YASKAWA.

Under the terms of the memorandum, the companies will begin discussions to potentially develop a platform that may seamlessly link early-stage research to commercialization, utilizing the state-of-the-art Maholo robot to manufacture high-quality products and shorten the R&D period of cell therapy.

Furthermore, the companies would consider the possibility of potentially offering the use of the platform to startups and academia, aiming to develop an advanced cell therapy ecosystem that may enable the discovery and nurturing of innovation while potentially reducing investment in investigational drug manufacturing based on the Pharmaceutical and Medical Device Act.

Astellas would provide cell manufacturing technology, clinical development and regulatory insights related to cell therapy. YASKAWA would provide and develop state-of-the-art robotics and factory automation technologies.

1.3. Collaboration agreement: Cellipoint & Adva

Cellipont Bioservices (TX, USA; https://cellipont.com), a cell therapy Contract Development and Manufacturing Organization (CDMO), and Adva Biotechnology (Israel; www.advabio.com), a private biotechnology company providing a decentralized, automated and efficient manufacturing platform for advanced cell therapies, have announced its partnership with Adva Biotechnology marking the launch of the groundbreaking ADVA X3^® platform in North America [3]. The ADVA X3 is an advanced, fully automated platform for manufacturing cell therapies, streamlining processes and reducing costs. This alliance signifies a leap forward in personalized treatment, bringing cutting-edge therapies to patients worldwide.

The ADVA X3 is an all-in-one automated system that simplifies and accelerates the manufacturing of CAR-T therapies from manual to fully automated and cGMP in as little as 3 months. It represents a major advancement in manufacturing efficiency and quality control, ensuring the delivery of life-saving therapies with minimal user involvement. Moreover, the ADVA X3 significantly reduces costs, making it accessible to a broader patient population. The unique combination of Cellipont's best-in-class process and manufacturing expertise and purpose-built facility coupled with the ADVA X3 metabolic sensing-based AI-driven automated manufacturing platform allows therapy developers to quickly go from manual processing to fully automated and GMP. Additionally, the ADVA X3^® system supports cell therapies such as NK, TCR, TIL and exosomes from activation to final cell product.

1.4. Collaboration agreement: Galapagos & Adaptimmune

Galapagos NV (Belgium; www.glpg.com) and Adaptimmune Therapeutics (UK; www.adaptimmune.com) have entered into a clinical collaboration agreement with an option to exclusively license Adaptimmune's next-generation T cell receptor (TCR) T-cell therapy (uza-cel) targeting MAGE-A4 for head & neck cancer and potential future solid tumor indications, using Galapagos' decentralized cell manufacturing platform [4].

Uza-cel is a next-generation clinical-stage engineered TCR T-cell therapy developed by Adaptimmune, targeting the MAGE-A4 cancer antigen expressed in various solid tumors. Uza-cel is engineered to express the CD8α co-receptor alongside the engineered TCR that targets MAGE-A4. Data indicate that co-expression of CD8α may broaden and increase the immune response against solid tumors.

The Adaptimmune sponsored Phase I SURPASS trial with centrally manufactured uza-cel has shown encouraging results in head & neck cancer with an overall response rate of 80% [5]. Initial in vitro results suggest that uza-cel, produced on Galapagos' decentralized manufacturing platform, yields early phenotype T-cells that could improve efficacy and durability compared with uza-cel centrally manufactured on Adaptimmune's platform. In addition, Galapagos' decentralized manufacturing platform offers the potential for the delivery of fresh, fit cells with a vein-to-vein time of 7 days in a patient population in which rapid access to treatment is vital.

1.5. Collaboration agreement: Galapagos & Blood Centers of America

Galapagos NV (Belgium; www.glpg.com) and Blood Centers of America (BCA; RI, USA; https://bca.coop) have announced that they have entered into a strategic collaboration for the decentralized manufacturing of Galapagos' chimeric antigen receptor (CAR) T cell therapies through BCA's network in the US [6].

Under the terms of the collaboration agreement, BCA will provide access to its extensive network of over 50 community blood centers in 43 states for Good Manufacturing Practice compliant manufacturing for Galapagos' CAR-T hemato-oncology clinical program.

Galapagos will leverage BCA's network to initiate technology transfer to multiple sites in parallel for the decentralized manufacturing of its CAR-T product candidates, close to cancer treatment centers, while also accessing apheresis capacity at BCA sites when required. In addition, BCA will play a crucial coordinating role by supporting site initiation and onboarding to accelerate Galapagos' efforts and ensure consistent quality.

Galapagos' decentralized manufacturing platform could address many of the limitations that currently available CAR-T production is facing. It has the potential to offer greater speed and scalability, with the delivery of fresh, fit cells with a vein-to-vein time of 7 days and the possibility for greater physician control and improved patient experience.

1.6. Collaboration & licensing agreement: Astellas & Poseida

Astellas Pharma (Japan; www.astellas.com) and Poseida Therapeutics (CA, USA; https://poseida.com) have announced that Xyphos Biosciences (CA, USA; www.xyphosinc.com), a wholly owned subsidiary of Astellas, and Poseida have entered into a research collaboration and license agreement to develop novel convertibleCAR^® programs by combining the innovative cell therapy platforms from each of the companies [7].

Poseida is advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. In oncology, its pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors that address patient populations with high unmet medical need. Xyphos utilizes a novel and proprietary ACCEL™ technology platform that uses its convertibleCAR in combination with proprietary MicAbodies to target tumor cells. ACCEL technology is based on a synthetic biology approach that utilizes the binding of an engineered protein ligand to an orthogonal engineered receptor which forms the extracellular domain of a convertibleCAR. The convertibleCAR is targeted to tumor cells with a tumor-associated antigen-specific engineered antibody-like molecule (MicAbody) containing the engineered ligand.

Under the terms of the agreement, the companies plan to combine Poseida's proprietary allogeneic CAR-T platform with Xyphos' ACCEL technology to create one Poseida-developed CAR-T construct to form the basis of two convertibleCAR product candidates targeting solid tumors. Xyphos will reimburse Poseida for costs incurred as part of the research agreement and will be responsible for the development and future commercialization of products generated from the collaboration. Poseida will receive US US$ 50 M upfront plus potential development and sales milestones and contingency payments of up to US$ 550 M in total. Additionally, Poseida is eligible for up to low double digit tiered royalties as a percentage of net sales.

1.7. Licensing agreement: HuidaGene & Synthegc

HuidaGene Therapeutics (China; www.huidagene.com), a global clinical-stage biotechnology company focused on advancing CRISPR-based genomic medicines, and Synthego Corporation (CA, USA; www.synthego.com), a provider of innovative CRISPR solutions for developing cell and gene therapies, have announced a partnered licensing agreement for the high-fidelity Cas12 CRISPR nuclease (hfCas12Max) [8]. This partnership underscores the significant clinical utility of hfCas12Max, naturally complementing Synthego's focus on advanced GMP manufacturing capabilities. Under this licensing agreement and pursuant to undisclosed financial terms between HuidaGene and Synthego, HuidaGene grants Synthego manufacturing and commercialization rights for the hfCas12Max nuclease and optimized gRNA for research; HuidaGene also grants Synthego the right to sublicense the nuclease for therapeutic use.

hfCas12Max, a novel CRISPR gene-editing system developed through HuidaGene's HG-PRECISE platform, stands out for its superior on-target editing efficiency and reduced off-target editing activity in mammalian cells. This novel CRISPR gene-editing system also offers the advantage of packaging into a single viral vector, an important requirement for many cell and gene therapies. The commercialization of hfCas12Max will significantly enhance the accessibility of this CRISPR gene-editing system, providing increased freedom-to-operate and facilitating the development of CRISPR-based cell and gene therapies.

1.8. Partnership agreement: AmplifyBio & Xcell

Xcell Biosciences (CA, USA; www.xcellbio.com), a company focused on cell and gene therapy manufacturing applications, and AmplifyBio (CA, USA; www.amplify-bio.com), a contract development and manufacturing organization (CDMO), have announced the launch of a partnership designed to streamline and improve the manufacturing process for engineered TCR therapies targeting solid tumors [9].

1.9. Partnership agreement: Theragent & Pluristyx

Theragent (CA, USA; https://theragent.com), a CDMO focused on advancing next-generation cell- and tissue-based therapies, has announced a partnership with Pluristyx (WA, USA; https://pluristyx.com), an early stage, privately held biotechnology company specializing in induced Pluripotent Stem Cell (iPSC) products [10]. Combined, this partnership will allow the companies to provide a seamless, end-to-end pathway to therapeutics developers from iPSC derivation to commercial production of iPSC-derived drug products.

Under the terms of the collaboration, Pluristyx's iPSC platform, including licenses for iPSC derivation, consent for use, and novel mechanisms such as the FailSafe^® functional suicide switch and iACT Stealth Cells^® immune evasion technology, will be utilized for early-stage programs and then synchronously transferred to Theragent for GMP processing including scale-up and clinical- to commercial-scale production at their state-of-the-art manufacturing facility in Arcadia, CA.

2. Achievements, launches…

2.1. Final Spark

FinalSpark (https://finalspark.com), a Swiss biocomputing startup, has launched the first-ever online platform enabling global researchers to conduct experiments remotely on biological neurons in vitro [11]. The platform provides around-the-clock access to 16 human brain organoids aimed at developing the world's first living processor. These bioprocessors, composed of living neurons capable of learning and processing information, consume a million times less power than traditional digital processors, potentially reducing the environmental impact associated with the rising use of computers [12].

2.2. PBS Biotech

PBS Biotech (CA, USA; www.pbsbiotech.com), a provider of single-use bioreactor systems and process development services, announced the successful attainment of the ISO 9001:2015 certification of its Quality Management System (QMS) [13]. This certification reflects PBS Biotech's commitment to meet the needs of customers by maintaining effective quality standards across its operations. The ISO 9001:2015 certification will ensure that PBS Biotech's single-use bioreactor systems are manufactured and delivered under the highest standards of quality management, significantly enhancing their reliability, robustness and efficacy for commercial manufacturing of cell therapy products.

2.3. Reprocell

REPROCELL (Japan; www.reprocell.com) has announced the offering of Master Cell Bank (MCB) manufacturing service for generation of human iPSC (hiPSC) and human Mesenchymal Stem Cells (hMSC) for therapeutic applications in the USA [14]. In contrast to conventional clean rooms that utilize cell culturing and processing in open incubators and open hoods, the Xvivo System consists of modular sets of closed incubators and closed hoods, integrated together as co-chambers and sub-chambers. The enclosed nature of the system allows each chamber to undergo individual environmental control and monitoring. These controls allow greater consistency of the culture environment and reduced risk of contamination, resulting in a superior product and a faster turn-around time.

2.4. TC BioPharm

TC BioPharm (UK; www.tcbiopharm.com) a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer and other indications, has announced that the company's lead therapeutic TCB008 will be available to patients via a newly launched compassionate use program in the UK [15].

Compassionate use refers to the prescription of unlicensed medicinal products to treat a patient in instances where their individual clinical needs cannot be met with the available licensed treatments. Patients enrolling in these clinical trials must meet specific entry criteria, both to protect vulnerable patient populations and ensure the validity of study data. These data must be reviewed and approved by regulatory agencies before products can be marketed as licensed medicinal products for patient treatment.

Access to TCB's cell therapy products through the compassionate use program will provide medical practitioners with treatment options for patients who are ineligible to access experimental treatments through ongoing clinical trials.

2.5. X-Therma

X-Therma (CA, USA; https://x-therma.com), a biotechnology company developing a breakthrough platform for regenerative medicine and organ preservation, has achieved GMP commercial readiness with its flagship product, XT-Thrive^® [16].

XT-Thrive^® is a dimethyl sulfoxide (DMSO)-free, serum-free, protein-free and chemically defined cryopreservation media enabling the preservation of mammalian cells at ultra-low temperatures (-70°C to -196°C). It incorporates a fundamentally new class of cryoprotectant, named peptoids, fully synthetic defined molecules are inspired by natural antifreeze proteins (hyper-effective and non-toxic) to prevent aberrant ice formation and risk of shock-induced ice damage. This product can be used for the cryopreservation of cells used for manufacture of biological products including monoclonal antibodies, recombinant proteins, fusion proteins and vaccines. XT-Thrive does not require a manual wash-out step, thus elevating its safety profile in comparison to other cryopreservation media.

3. Clinical trials

3.1. Pluripotent stem cells

3.1.1. Cynata

Cynata Therapeutics (Australia; www.cynata.com), a clinical-stage biotechnology company specializing in cell therapeutics, has announce the publication of two-year follow-up data of CYP-001 in patients with steroid-resistant acute graft versus host disease (SR-aGvHD) [17,18]. CYP-001 is Cynata's Cymerus™ off-the-shelf iPSC1-derived MSC2 product for intravenous infusion, which is being investigated as a potential immune modulating treatment. The paper reports sustained outcomes at the two-year follow-up in the Phase I clinical trial of CYP-001 in patients with SR-aGvHD. Key results include a two-year overall survival rate of 60% (9/15 patients), with no treatment-related serious adverse events or safety concerns identified. Historically the prognosis in patients with SR-aGvHD has been very poor, with two-year overall survival rates below 20%.

3.2. Immune cells

3.2.1. Carisma

Carisma Therapeutics (PA, USA; https://carismatx.com), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, has announced that the first patient was dosed in its Phase I clinical trial evaluating CT-0525, an ex vivo gene-modified autologous CAR-Monocyte cellular therapy, for the treatment of patients with solid tumors that overexpress HER2 [19,20].

3.2.2. iCell

iCell Gene Therapeutics (NY, USA; www.icellgene.com), a clinical stage biopharmaceutical company focused on immunotherapies for autoimmune disorders and cancer, has announced the publication of positive results of an investigator initiated clinical trial (IIT) for its BCMA-CD19 compound CAR (cCAR) T cell immunotherapy [21–23]. The clinical trial evaluated the safety and efficacy of a complete humoral reset of both long-lived plasma cells and B cells in 13 systemic lupus erythematosus (SLE) patients treated with iCell's cCAR, including 11 patients with SLE and lupus nephritis (LN). All patients (12/13) who received an initial dose of 3 × 10⁶ cCAR cells/kg were negative for all autoantibodies, including those derived from long-lived plasma cells, 3 months post-cCAR and the complement returned to normal levels. These patients achieved symptom-free and medication-free remission, with post-cCAR follow-up to 46 months. cCAR therapy was well tolerated with mild cytokine-release syndrome. iCell considers this IIT to be a successful proof of concept; the Company plans to file an investigational new drug application (IND) for its BCMA-CD19 cCAR in the United States and China.

iCell's cCAR, with two independently functioning CARs in a single construct targeting the B cell CD19 and the plasma cell BCMA surface antigens, is uniquely designed to completely reset humoral immunity and address the underlying cause of SLE/LN through the elimination of all elevated autoantibodies including those produced by long-lived plasma cells.

3.3. Other

3.3.1. RION

RION (MN, USA; https://riontx.com), a clinical-stage regenerative medicine company at the forefront of exosome therapeutics, announced today the first subject was dosed in the Phase II clinical trial with its novel product, known as Purified Exosome Product™ (PEP™) [24,25]. This event marks a pivotal milestone in evaluating PEP™ as an innovative treatment for chronic conditions such as Diabetic Foot Ulcers (DFUs). PEP was administered to the first subject as part of a 40-subject Phase IIA study. The Phase IIA study is a multi-center, prospective, open-label and randomized study to evaluate the safety and efficacy of topically applied PEP. The objective of the study is to compare subjects treated with PEP versus Standard of Care for up to 12 weekly applications.

3.3.2. Strand

Strand Therapeutics, the world's first programmable mRNA company developing curative therapies for cancer, autoimmune diseases and beyond, has dosed the first patient in their Phase I, first-in-human trial of STX-001, an investigational multi-mechanistic, synthetic self-replicating mRNA technology that expresses an IL-12 cytokine for an extended duration, directly into the tumor microenvironment [26,27]. The Phase I trial is an open-label, multi-center first-in-human dose-escalation trial, evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary antitumor activity of STX-001 alone, or in combination with pembrolizumab in patients with treatment refractory advanced solid tumors.

4. Regulations, approvals, acquisitions…

4.1. Green light

4.1.1. Be Biopharma

Be Biopharma (MA, USA: https://be.bio), a company pioneering the discovery and development of Engineered B Cell Medicines (BCMs), has announced the clearance of its IND from the US FDA for BE-101, a first-in-class BCM in development for the potential treatment of hemophilia B [28]. The Phase I/2 clinical trial, BeCoMe-9, is a multi-center, first-in-human dose escalation study designed to assess the safety and preliminary efficacy of BE-101 in adult participants with moderately severe to severe hemophilia B. Be Bio expects to begin dosing participants in the second half of 2024.

BE-101 is an autologous first-in-class B Cell Medicine (BCM) that is engineered to insert the human FIX gene into primary human B cells, allowing for expression of active FIX for the treatment of hemophilia B. BE-101 has the potential to express sustained therapeutic FIX activity levels with a single infusion with the flexibility to be re-dosed, if needed. The potential to maintain therapeutic FIX activity levels while reducing dosing frequency associated with current FIX replacement regimens would address the considerable infusion burden associated with current therapies and potentially drive significant reductions in the annualized bleeding rates and FIX usage.

4.1.2. Bristol Myers Squibb

Bristol Myers Squibb (NJ, USA; www.bms.com) has announced that the US FDA has granted accelerated approval for Breyanzi^® (lisocabtagene maraleucel; liso-cel), a CD19-directed CAR T cell therapy, for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy [29]. This indication is approved under accelerated approval based on response rate and duration of response.

A couple weeks later, the US FDA has granted an approval for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor [30]. This FDA approval marks the fourth distinct subtype of non-Hodgkin lymphoma for which Breyanzi is approved, making it the CAR T cell therapy available to treat the broadest array of B-cell malignancies.

In both cases, Breyanzi is delivered as a one-time infusion with a single dose containing 90 to 110 × 10⁶ CAR-positive viable T cells. Breyanzi has exhibited a consistent safety profile and across clinical trials. The safety profile of Breyanzi allows for the option of outpatient treatment and management of patients.

4.1.3. CorTec

CorTec (Germany; www.cortec-neuro.com) has announced that the US FDA has approved an Investigational Device Exemption (IDE) application involving the closed-loop Brain Interchange Implant System [31]. This clinical study will investigate a novel stroke rehabilitation treatment using cortical stimulation to enhance plasticity within the brain. With the clearance of the Brain Interchange System for human use CorTec is prepared to serve clinicians and research groups with its advanced implant technology to investigate novel treatment options for neurological diseases.

5. Capital market & finances

5.1. Aspen Neurosciences

Aspen Neuroscience (CA, USA; https://aspenneuroscience.com), a private biotechnology company developing personalized regenerative therapies, has received a CLIN2 grant award of US$ 8 M from the California Institute for Regenerative Medicine (CIRM; CA, USA; www.cirm.ca.gov), the world's largest institution dedicated to regenerative medicine, to support clinical research aimed at treating Parkinson's disease (PD) [32].

The grant, a first for an autologous therapeutic for degenerative neurological conditions, will help advance the development of ANPD001, an investigational iPSC-derived dopaminergic neuron replacement therapy. ANPD001 is being studied in a First in Human Phase I/IIa clinical trial for patients with moderate to advanced PD, to assess safety and tolerability [33]. This is the first use of the autologous approach in a multi-patient and multi-center clinical trial.

5.2. BioLineRX

BioLineRx (Israel; https://biolinerx.com), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, has received a notification letter from the Nasdaq Stock Market (NY, USA; www.nasdaq.com) [34]. The letter notifies the Company that it is not in compliance with the minimum bid price requirement set forth in Nasdaq Listing Rules for continued listing on the Nasdaq Capital Market, since the closing bid price for the Company's American Depositary Shares (ADS) listed on the Nasdaq was below USD $ 1.00 for 30 consecutive trading days. Nasdaq Listing Rule 5550(a)(2) requires listed securities to maintain a minimum bid price of USD $ 1.00 per share, and Nasdaq Listing Rule 5810(c)(3)(A) provides that a failure to meet the minimum bid price requirement exists if the deficiency continues for a period of 30 consecutive business days.

The Notice had no immediate effect on the Company's Nasdaq listing or the trading of its ADSs, and during the grace period, as may be extended, the Company's ADSs continued to trade on the Nasdaq under the symbol “BLRX”.

5.3. CIRM

The California Institute for Regenerative Medicine (CIRM; CA, USA; www.cirm.ca.gov), one of the world's largest institutions dedicated to regenerative medicine, has awarded US$ 53 M to fund various projects from its Clinical and Translation programs [35]. Among awardees are four companies: Neurona Therapeutics (CA, USA; www.neuronatherapeutics.com) for development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug-resistant focal epilepsy (US$ 3.8 M), Reply Holdings (CA, USA; https://replay.bio) for hypoimmunogenic iPSC-derived TCR-NK cells for oncology (US$ 4.1 M), Axent Biosciences (CA, USA; no website available) for a high-quality, accessible cell therapy for Parkinson's Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation (US$ 4.0 M), and AcuraStem (CA, USA; https://acurastem.com) for development of an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies (US$ 4.0 M).

5.4. Gameto

Gameto (NY, USA; www.gametogen.com), a biotechnology company dedicated to advancing treatment options in women's health, has closed an oversubscribed US$ 33 M Series B financing round [36]. This latest funding brings Gameto's total capital raised to US$ 73 M. In the US, the financing will support the clinical development of Fertilo, their novel investigational in vitro maturation (IVM) solution containing engineered ovarian support cells (OSCs) to mature eggs outside of the body. Following productive discussions with the US FDA, Gameto received tentative approval to proceed to Phase 3 trials, subject to the completion of certain assay and manufacturing requirements. The funding will continue to support the commercial launches of Fertilo in Australia and Latin America where it is already being used in the clinic.

Gameto's novel approach uses cellular engineering to create a pure population of highly potent OSCs from a female clinical-grade hiPSC line that recreates the dynamic, bidirectional follicular environment in a dish when co-cultured with immature eggs. This technology is designed to replace hormonal injections and shorten the in vitro fertilization (IVF) and egg freezing cycle from 10–14 days to 2–3 days. The manufacturing process using clinical grade iPSCs provided by Reprocell (Japan; www.reprocell.com) has been published on BioRxiv [37,38].

5.5. Luxa

Luxa Biotechnology (NJ, USA; www.luxabiotech.com) has been awarded a US$ 4 M grant from the California Institute for Regeneration Medicine, supporting its ongoing Phase I/2a clinical study of the RPESC-RPE-4W cell transplantation for the treatment of dry age-related macular degeneration (AMD) [39,40].

The RPESC-RPE-4W, a cutting-edge therapy developed from adult retinal pigment epithelial stem cells, aims to replace deteriorated retinal cells and offers the potential to significantly restore vision in patients affected by dry AMD.

5.6. OverT

OverT Bio (NY, USA; www.overt.bio), a data-driven company working to unlock the curative potential of cell therapies in solid tumors, has raised US$ 16 M in seed funding [41]. OverT is the first to use patients' immune response to cancer to find new receptors and targets that can be applied across the human population. The team searches the entire human genome to find new ways to make cell therapies more durable and capable of destroying cancers [42,43].

Financial disclosure

The author D 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.

Competing interests disclosure

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Writing disclosure

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