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

ABSTRACT

Latest developments in the field of Advanced Therapy Medicinal Products and regenerative medicine compiled from publicly available information and press releases from non-academic institutions in August 2025.

1. Business development

1.1. Licensing agreements: Apertura, Galibra, Emugen

Apertura Gene Therapy (NY, USA; https://aperturagtx.com), a biotechnology company focused on innovative gene therapy solutions announced several licensing deals with Galibra Neuroscience and Emugen Therapeutics to utilize Apertura’s human transferrin receptor 1 capsid (TfR1 CapX) for their central nervous system (CNS) programs [1,2]. A third venture-backed biotechnology company has entered into an Option Agreement with Apertura for multiple CNS indications.

TfR1 CapX is an intravenously (IV) dosed AAV capsid that binds to the human transferrin receptor, TfR1, to cross the blood-brain barrier and broadly transduce tissues in the brain. In extensive preclinical testing conducted independently by several groups, TfR1 CapX has demonstrated a selectivity to the CNS compared to other tissues with more than 50% neuron transduction and 90% astrocyte transduction across different brain regions.

Galibra Neuroscience (MA, USA; https://galibraneuroscience.com) will utilize TfR1 CapX for GABA-related disorders, which is the major brain inhibitory neurotransmitter keeping neural communication in check and preventing over-excitation. GABA levels in healthy individuals are highly regulated with over 20 genes responsible for regulating GABA signaling. Variants of these genes lead to GABA imbalance, the root cause of several rare pediatric disorders.

Emugen Therapeutics (MA, USA; https://www.emugentx.com) is developing novel gene therapy solutions to address neurodevelopmental and neurodegenerative diseases.

1.2. Manufacturing agreement: Andelyn and Amplo

Andelyn Biosciences (OH, USA; www.andelynbio.com), a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Amplo Biotechnology (CA, USA; https://amplobiotechnology.com/), a pioneer in adeno-associated virus (AAV) regenerative medicines for neuromuscular junction (NMJ) affecting conditions [3]. The partnership will provide scalable manufacturing of clinical-grade AAV material, leveraging Andelyn’s suspension AAV Curator® platform. Andelyn Biosciences suspension AAV Curator® platform is a highly characterized, scalable, suspension AAV platform that leverages a data-driven approach to adapt and optimize customer processes while providing a pathway to cGMP and commercial manufacturing. By adopting this platform, Amplo Biotechnology aims to advance its AAV gene therapy programs toward clinical evaluation, focusing on the safety and efficacy required for upcoming trials.

2. Achievements, launches …

2.1. Kaiwa

Kaiwa Technology (China; http://kaiwatech.com/) have revealed plans to unveil a prototype of the world’s first gestational robot to give birth to a human infant by 2026 [4]. The new “pregnancy robot” uses artificial womb technology, bringing hope to those struggling with infertility and opening a new chapter in reproductive science.

The fetus would grow within the humanoid robot’s belly with a complete artificial womb, bathed in artificial amniotic fluid. The nutrients would be delivered through attached tubes, mirroring natural gestation. The robot would interact with humans during pregnancy with the goal of simulating the entire nine-month cycle from conception to birth.

The technology may also open doors to novel society possibilities, like the single person or same-sex couple easier access to biological children from their own genetic material.

Chinese officials are cooperating closely with Kaiwa Technology to develop legislative and policy blueprints to address new ethical and legal challenges.

2.2. Manhattan Genomics

Manhattan Genomics (USA; https://manhattangenomics.com) has officially launched its Manhattan Project, with the stated mission of preventing and ultimately eradicating inherited diseases through germline genome editing at the zygote stage [5,6]. The company has outlined guiding principles focused on prevention, reducing suffering, reproductive choice, and data-driven regulation. According to the company, preclinical studies will involve animal models and advanced cellular systems to establish safety and efficacy before progressing toward clinical applications.

2.3. Synchron

Synchron (NY, USA; https://synchron.com), a brain-computer interface (BCI) company, has released the first-ever public demonstration of an individual using an iPad controlled entirely by thought, leveraging Apple’s built-in accessibility features and new Brain-Computer Interface Human Interface Device (BCI HID) protocol [7].

The video features a participant in Synchron’s COMMAND clinical study and a person living with ALS, who uses the company’s implantable BCI to navigate the iPad home screen, open apps, and compose text all without using his hands, voice, or eyes.

This moment follows Apple’s announcement in May of a new BCI Human Interface Device (BCI HID) input protocol. With the new protocol, Apple’s operating systems can leverage brain signals as a native input method for the first time.

Synchron has completed two human clinical trials since 2019 and is preparing for a larger-scale study [8,9]. The company’s implantable BCI is now powered by Chiral AI™, a proprietary foundation model of cognition

3. Clinical trials

3.1. Pluripotent stem cells

3.1.1. Lineage Cell Therapeutics

Lineage Cell Therapeutics (CA, USA; https://lineagecell.com), a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf,” cell therapies for serious neurological and ophthalmic conditions, announced that the first-ever chronic spinal cord injury patient has been treated in the Company’s DOSED (Delivery of Oligodendrocyte Progenitor Cells (OPCs) for Spinal Cord Injury: Evaluation of a Novel Device) clinical study at UC San Diego Health [10,11]. The DOSED study is designed to evaluate the safety and utility of a new parenchymal spinal delivery system, a novel delivery device developed to deliver OPC1 directly to the site of injury in patients with spinal cord injury (SCI) and will enroll both subacute (between 21 and 42 days following injury) and chronic (between 1 and 5 years following injury) SCI patients.

OPC1 is an investigational, allogeneic stem cell-derived cell transplant, comprised of oligodendrocyte progenitor cells. OPC1 is designed to replace or support cells in the injured spinal cord that are absent or dysfunctional due to traumatic injury and is intended to help restore or augment functional activity in persons suffering from an SCI. Improved functional activity can lead to greater mobility and enhanced quality of life for patients and significant cost-savings for caregivers. The first patient treated in DOSED was a neurologically complete SCI injury (American Spinal Injury Association Impairment Scale [AIS] grade A), with a single neurological level of injury from T1 to T10, and the novel spinal delivery system successfully administered the intended one-time injection of 10 million OPC1 cells.

3.1.2. Sana Biotechnology

Sana Biotechnology (WA, USA; https://sana.com/) has published a journal article that discusses 12-week results from an investigator-sponsored trial, conducted at Uppsala University Hospital, evaluating the transplantation of UP421, a primary human pancreatic islet cell therapy engineered with Sana’s hypoimmune (HIP) technology, without the use of immunosuppressive medications in a 42-year-old patient living with type 1 diabetes for over three decades [12–14]. The intramuscular transplantation of HIP-modified pancreatic islet cells is safe and well-tolerated and demonstrates that these cells evade autoimmune and allogeneic immune recognition, persist, and secrete insulin in a glucose-dependent manner over the 12-week evaluation period reported in the article. Twelve-week PET-MRI scanning also confirmed islet cells at the transplant site.

3.2. Immune cell therapy

3.2.1. Immunity bio

ImmunityBio (CA, USA; https://immunitybio.com), an immunotherapy company, announced early findings from its QUILT-106 Phase I trial, showing highly promising complete responses in the first two patients treated to date with late-stage Waldenstrom macroglobulinemia (WM) – a type of non-Hodgkins lymphoma (NHL) – using its CD19 CAR-NK (CD19 t-haNK) natural killer cell therapy [15,16].

QUILT-106 is a first-in-human trial evaluating the safety and preliminary efficacy of CD19 CAR-NK cell therapy alone and in combination with rituximab in patients with relapsed or refractory (R/R) CD19⁺ CD20⁺ B-cell NHL.

CD19 CAR-NK is a targeted high-affinity natural killer cell therapy – an off-the-shelf, allogeneic NK cell line engineered to express a CD19-specific chimeric antigen receptor (CAR) and a high-affinity CD16 (FcγRIIIa 158 V) receptor. This design enables dual anti-tumor mechanisms: direct CAR-mediated cytotoxicity and augmented antibody-dependent cellular cytotoxicity when paired with anti-CD20 monoclonal antibody rituximab. Combining CD19 CAR-NK cells with rituximab could thereby target CD19⁺/CD20⁺ lymphoma cells to enhance tumor cell killing.

3.3. Gene therapy

3.3.1. CorrectSequence

CorrectSequence Therapeutics (China; www.correctsequence.com), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the treatment of severe diseases, has announced the successful treatment of the first sickle cell disease (SCD) patient using its high-precision base-editing therapy CS-101 [17,18].

The patient, a 21-year-old woman from Nigeria previously suffered from recurrent vaso-occlusive crises (VOCs), demonstrated a significant and sustained increase in fetal hemoglobin (HbF) levels, accompanied by a marked reduction in sickle hemoglobin (HbS). Six months post-treatment, the HbF-to-HbS ratio stabilized at 65%:35%, with total hemoglobin consistently exceeding 120 g/L. Over the six-month follow-up period, the patient experienced no VOCs and successfully resumed normal daily activities – marking the first reported clinical treatment of sickle cell disease in China using base editing technology.

CS-101 is an autologous CD34⁺ cell product engineered using in vitro base editing to modify the BCL11A binding site within the HBG promoter. This alteration prevents BCL11A from binding, thereby reactivating γ-globin expression and increasing fetal hemoglobin (HbF) levels in the blood. Elevated HbF compensates for the loss of adult hemoglobin (HbA) function, potentially achieving a clinical cure.

3.3.2. Epicrispr

Epicrispr Biotechnologies (CA, USA; https://epicrispr.com), a clinical-stage company pioneering gene-modulating therapies, announced that the first patient has been dosed in its global first-in-human clinical trial of EPI-321, an investigational, one-time epigenetic editing therapy for facioscapulohumeral muscular dystrophy (FSHD) [19,20].

EPI-321 is an investigational, single dose, gene-modulating therapy designed to silence aberrant expression of DUX4 in skeletal muscle, a gene that is incorrectly activated within muscle cells in people living with FSHD, leading to progressive muscle degeneration. Delivered intravenously via a clinically validated AAV vector, EPI-321 has demonstrated robust suppression of DUX4 expression and protection of muscle tissue in preclinical models. EPI-321 has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.

3.3.3. Kelonia

Kelonia Therapeutics (MA, USA; https://keloniatx.com), a biotech company revolutionizing in vivo gene delivery, has announced that the first patient has been dosed in the inMMyCAR study, a Phase 1 clinical trial evaluating KLN-1010, a novel in vivo gene therapy that generates anti-BCMA CAR-T cells, in patients with relapsed and refractory multiple myeloma [21,22].

KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR‑T treatments, KLN‑1010 is administered to patients via direct transfusion and is designed to generate durable CAR‑T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.

KLN‑1010 is an investigational in vivo gene therapy that generates anti-BCMA CAR-T cells, targeting a protein expressed on the surface of multiple myeloma cells. Unlike traditional CAR‑T treatments, KLN‑1010 is administered to patients via direct transfusion and is designed to generate durable CAR‑T cells inside the body after a single dose, potentially eliminating the need for long wait times to receive treatment. This may overcome several limitations faced by current CAR-T approaches, including limited access to treatment and preconditioning chemotherapy.

4. Regulations, approvals, acquisitions …

4.1. Acquisitions

4.1.1. Kite and Interius

Kite (CA, USA; https://www.kitepharma.com), a Gilead Company (CA, USA; www.gilead.com/), has entered into a definitive agreement to acquire Interius BioTherapeutics (PA, USA; https://interiusbio.com), a privately held biotechnology company developing in vivo CAR therapeutics, for U$350 M [23].

This acquisition complements Kite’s expertise in cell therapy by incorporating Interius’s integrating in vivo platform. This approach enables the generation of CAR T-cells directly within the patient’s body and may offer a more durable and long-lasting therapeutic effect by inserting DNA into the patient’s genome. Unlike traditional CAR T therapies that require cell harvesting, engineering and reinfusion, Interius’s innovative, off-the-shelf yet personalized approach is designed to be delivered via a single intravenous infusion, eliminating the need for preconditioning chemotherapy and complex cell processing.

4.1.2. Northwest Therapeutics and Advent BioServices

Northwest Biotherapeutics (MD, USA; https://nwbio.com), a biotechnology company developing DCVax® personalized immune therapies for solid tumor cancers, announced that it has entered into an agreement to acquire Advent BioServices (UK; www.adventbio.uk) from its owner, Toucan Holdings [24]. The transaction is expected to close as soon as certain legal conditions are fulfilled. At closing, Advent will become a wholly owned subsidiary of the Company, and this is expected to enable the Company’s operations to become fully integrated.

Northwest Biotherapeutics is a biotechnology company focused on developing personalized immunotherapy products that are designed to treat cancers more effectively than current treatments, without toxicities of the kind associated with chemotherapies, and on a cost-effective basis. The Company has a broad platform technology for DCVax® dendritic cell-based vaccines. The Company’s lead program involves DCVax®-L treatment for glioblastoma (GBM). The Company has completed a 331-patient Phase III trial of DCVax-L for GBM and submitted a marketing authorization application (MAA) for commercial approval in the UK. The MAA is currently undergoing review [25].

4.1.3. Terumo and OrganOx

Terumo Corporation (Japan; www.terumo.com) has entered into a definitive agreement with OrganOx (UK; www.organox.com) to acquire all of its outstanding shares. Upon completion of the transaction, OrganOx will become a wholly owned subsidiary of Terumo [26]. The total transaction value is approximately US$1.5 billion.

OrganOx is a medical technology company specializing in advanced organ preservation devices for Normothermic Machine Perfusion (NMP). NMP and other ex-vivo machine perfusion (EVMP) technologies can preserve organs longer by circulating oxygen and nutrient rich perfusate through the organ at near-body temperature. In addition, NMP devices enable real-time monitoring of organ condition during storage and transport. This aims to help prevent the transplantation of organs with impaired function – with the goal of improving transplant success rates – as well as leading to a more effective use of organs from marginal donors.

4.1.4. US WorldMeds and Adaptimmune

US WorldMeds (USWM; KY, USA; https://usworldmeds.com/) reported the successful closing of the previously announced acquisition of Adaptimmune Therapeutics (UK; www.adaptimmune.com) cell – therapy assets – including TECELRA (afamitresgene autoleucel) [27].

Under the terms of the Asset Purchase Agreement, USWM paid US$55 million in cash at closing and will fund up to an additional US$30 million in performance-based milestone payments tied to commercial and regulatory outcomes. Adaptimmune has retained rights to its pre – clinical programs, including PRAME – and CD70–directed T – cell therapies, and its allogeneic pipeline.

5. Green light

5.1. AAVantgarde

AAVantgarde Bio (Italy; www.aavantgarde.com/en/), a clinical-stage biotechnology company developing generation gene therapies for inherited retinal diseases, has announced that the U.S. FDA has granted Fast Track Designation for AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4 [28]. The Investigational New Drug (IND) application for AAVB-039 was cleared to proceed by the FDA.

Stargardt disease is the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults. AAVB-039 addresses the underlying genetic cause of the disease by providing the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations.

5.2. Beam Therapeutics

Beam Therapeutics (MA, USA; https://beamtx.com), a biotechnology company developing precision genetic medicines through base editing, has announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD) [29]. The one-time therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promotor regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the predominant hemoglobin variant during development and early life. The safety and efficacy of BEAM-101 is being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in adult patients with SCD with severe VOCs.

5.3. Curacell

CuraCell (Sweden; https://curacell.se) has announced that the Paul Ehrlich Institute (PEI), the German federal authority for vaccines and biomedicines, has approved the company’s Clinical Trial Application (CTA) to initiate a Phase I/IIa clinical trial evaluating its novel autologous Tumor-Infiltrating Lymphocyte (TIL) therapy, CC-38, in patients with metastatic colorectal cancer and metastatic prostate cancer [30].

The open-label Phase I/IIa trial will enroll up to 16 patients with advanced solid tumors, including colorectal and prostate cancer (EU-CT-Number: 2025-521227-70–00). The study will be conducted at Krankenhaus Nordwest in Frankfurt. The trial will assess the feasibility of repeated administration, with safety and tolerability as the primary endpoints. Secondary objectives include evaluating preliminary anti-tumor activity and immune responses.

5.4. ExCellthera and CordEx

ExCellThera (QC, Canada; https://excellthera.com) and its wholly owned subsidiary Cordex Biologics (QC, Canada; https://cordexbio.com), announced the conditional marketing authorization of Zemcelpro® by the European Commission (EC) for the treatment of adults with hematological malignancies requiring an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available [31]. This EC decision authorizes the marketing of Zemcelpro in all European Union member states, as well as Iceland, Norway, and Liechtenstein.

Zemcelpro, also known as UM171 Cell Therapy, is a novel personalized cryopreserved hematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.

5.5. Galapagos

Galapagos Cell Therapeutics (Begium; www.glpg.com) has announced that the US FDA has granted RMAT designation to GLPG5101, a second generation anti-CD19/4-1BB CAR-T product candidate for the treatment of relapsed/refractory mantle cell lymphoma (R/R MCL), administered as a single fixed intravenous dose [32]. The safety, efficacy, and feasibility of decentralized manufactured GLPG5101 are currently being evaluated in the ATALANTA-1 Phase 1/2 study in eight hematological malignancies with high unmet need [33].

Galapagos’ innovative decentralized cell therapy manufacturing platform is designed to enable the administration of fresh, fit, stem-like early memory cells with a median vein-to-vein time of seven days, greater physician visibility, and improved patient experience. The platform consists of an end-to-end xCellit® workflow management and monitoring software system, a decentralized, functionally closed, automated manufacturing platform for cell therapies (using Lonza’s Cocoon®) and a proprietary quality control testing and release strategy.

5.6. Opus

Opus Genetics (NC, USA; https://opusgtx.com), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, announced that the U.S. FDA has accepted its IND application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD [34].

Best disease, or vitelliform macular dystrophy, is a rare, inherited retinal condition causing macular degeneration by mutations in the BEST1 gene, leading to progressive vision loss and, in some cases, blindness.

OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium cells where the defective gene resides.

With this IND clearance, Opus Genetics plans to initiate a Phase 1/2 clinical trial in the second half of 2025 [35]. The multi-center, open-label study will evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-BEST1 in patients with genetically confirmed BEST1-related IRD. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

5.7. PureString

Purespring Therapeutics (UK; https://purespringtx.com), a precision nephrology company focused on transforming the treatment of kidney diseases, has announced that its UK CTA for a planned Phase I/II study of PS-002, Purespring’s lead program, in patients with immunoglobin A (IgA) nephropathy (IgAN) has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA), the NHS Health Research Authority (HRA) and Research Ethics Committee (REC) [36,37]. PS-002 uses an AAV vector carrying a DNA transgene designed to express a complement-modulating protein in kidney podocytes.

PS-002 was developed to target the underlying cause of many kidney diseases by modulating complement activation in the kidney via precision targeting of podocytes. The program is initially focused on the treatment of IgA nephropathy, a rare and chronic autoimmune kidney disease that primarily affects young adults. In this type of nephropathy, aberrant IgA protein becomes trapped in the glomeruli, causing complement activation, inflammation, damage, and scarring. A significant proportion of affected patients will go on to develop kidney failure despite currently available therapies.

5.8. Qihan

Hangzhou Qihan Biotech (China; www.qihanbio.com), a company applying multiplexable genome editing technology to cell therapies and organ transplantation, has announced that the U.S. FDA has cleared its IND application for QT-019B [38]. This universal, dual-target CAR-T cell therapy is designed to treat refractory systemic lupus erythematosus (rSLE), and the clearance enables the initiation of a Phase 1/2 clinical trial in the United States, marking a significant milestone in the development of novel cell-based therapies for autoimmune diseases.

The therapy is derived from healthy donor leukapheresis products and engineered through gene editing to stably express two distinct chimeric antigen receptors (CARs) targeting CD19 and BCMA, enabling simultaneous recognition and elimination of cells expressing either antigen. To reduce the risk of graft-versus-host disease (GvHD), the T cell receptor (TCR) is knocked out. Additionally, multiplexable gene editing is employed to induce hypoimmunity, meaning the CAR-T cells are less likely to be recognized and attacked by the patient’s own NK and T cells, thereby reducing immune rejection and cytotoxicity while enhancing persistence.

5.9. Trailhead bio

Trailhead Biosystems (OH, USA; https://trailbio.com), a biotechnology company advancing scalable human cell models derived from induced pluripotent stem cells (iPSCs), announces the commercial release of TrailBio® Hematopoietic Progenitor Cells [39]. These progenitor cells are derived from a single, well-characterized iPSC line using Trailhead’s proprietary High-Dimensional Design-of-Experiments (HD-DoE®) platform, providing researchers with a consistent, scalable, and ready-to-use tool for hematopoietic research.

TrailBio Hematopoietic Progenitor Cells express key hematopoietic markers including CD34, CD43, and CD90. Functional characterization confirms their ability to form myeloid and erythroid colonies in colony-forming unit (CFU) assays, differentiate into multiple hematopoietic lineages and demonstrate hematopoietic commitment through transcriptomic profiling.

Designed for applications such as hematopoietic disease modeling, drug screening, and hematotoxicity studies, TrailBio Hematopoietic Progenitor Cells enable a reliable human model system that eliminates donor variability and supports reproducible results.

5.10. VeonGen

VeonGen Therapeutics (Germany; https://veongen.com/), a clinical-stage genetic medicine company developing next-generation gene therapies, has announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation – associated retinal dystrophies [40]. By delivering a full-length, functional ABCA4 gene, VG801 has the potential to address the underlying genetic root cause of Stargardt disease across all ABCA4 mutations.

VG801 is a dual-AAV gene therapy in development for Stargardt disease and other ABCA4-associated retinal dystrophies. It is designed to restore full-length, functional ABCA4 expression through VeonGen’s proprietary vgRNA REVeRT platform, combined with engineered vgAAV capsids that enable efficient delivery to photoreceptor cells. By delivering a functional ABCA4 gene, VG801 has the potential to address the underlying genetic cause of all forms of ABCA4-mutated Stargardt disease. VG801 is currently being evaluated in a Phase I/II clinical trial, with patient dosing underway [41].

6. Capital market & finances

6.1. Anocca

Anocca (Sweden; www.anocca.com), a leading clinical-stage T-cell immunotherapy company, has successfully raised US$46 M (SEK440M) in financing [42]. The additional capital will be used to drive the continued progress of VIDAR-1, Anocca’s gene-edited TCR-T cell therapies targeting mutant KRAS in pancreatic cancer, through early-stage clinical development, as well as progress Anocca’s preclinical pipeline [43]. The company has built a unique discovery engine that uses programmable human cells to recreate and manipulate T cell immunity. These proprietary technologies enable scaling of TCR-T cell therapy development, allowing the systematic generation of libraries of products that represent personalized treatments for the broad patient populations.

VIDAR-1 is designed as a multi-product umbrella trial targeting oncogenic driver mutations in KRAS within pancreatic ductal adenocarcinoma (PDAC). It will investigate up to 20 patients per product in a set of phase I/II studies. Phase I is currently conducted at eight sites in four countries with additional countries and sites in phase II. Patients will be eligible to enroll if they have an HLA, and KRAS mutation, matching an available product.

6.2. Gameto

Gameto, a clinical-stage biotechnology company developing stem cell-derived therapies for reproductive health, has completed a US$44 M Series C financing round, bringing total capital raised to US$127 M [44]. The Series C funding enables Gameto to complete the ongoing Phase 3 trial for its lead program, Fertilo, and file for regulatory approvals globally [45]. Fertilo is already cleared to commercialize many ex-U.S. jurisdictions and is currently used in clinics in Peru, Mexico, and Australia, with additional clearances in Japan, India, Singapore, Guatemala, Argentina, Dominican Republic, and Paraguay. Five babies have been born with Fertilo so far and over 20 pregnancies have been recorded.

6.3. Strand Therapeutics

Strand Therapeutics (MA, USA; www.strandtx.com), a next-generation mRNA-based therapeutics company, has announced that it raised US$153 M Series B funding [46]. The funding will advance Strand’s pipeline, led by STX-001, a programmable mRNA therapy that expresses the cytokine IL12 directly from the tumor microenvironment. STX-001 encodes IL-12 which Strand has designed so that it can reprogram the tumor microenvironment and stimulate a systemic anti-tumor immune response. Unlike traditional mRNA therapies, Strand’s approach uses self-replicating mRNA, ensuring localized and durable therapeutic activity. STX-001 is currently in a clinical trial [47].

6.4. Tahoe

Tahoe Therapeutics (CA, USA; www.tahoebio.ai) announced US$30 million in new funding to build the definitive foundational dataset for training Virtual Cell Models [48]. With this, the team will generate one billion single-cell datapoints, mapping one million drug-patient interactions, a scale previously impossible. The dataset will support the discovery of new precision medicines for cancer and beyond. Tahoe will also select a single partner to share the data and accelerate translation to clinical outcomes.

The raise follows the release of Tahoe‑100 M, the world’s first gigascale single-cell dataset, which has become foundational for teams building virtual cell models, ranging from major AI labs to focused research institutions. Open-sourced just a few months ago, Tahoe-100 M has been downloaded nearly 100,000 times. The dataset and the models trained on it have already led to the discovery of promising new therapeutic candidates for major cancer subtypes as well as novel targets across multiple modalities.

Tahoe is now expanding on that foundation: the company plans to generate one billion single-cell datapoints, mapping how tens of thousands of drug molecules interact with human biology. This new dataset will expand the boundaries of biological foundation models, aiming to reduce clinical trial failure rates and accelerate the development of precision medicines.

6.5. Wugen

Wugen (MO, USA; https://wugen.com), a clinical-stage biotechnology company pioneering the next generation of allogeneic, off-the-shelf CAR-T cell therapies, has closed US$115 M equity financing [49]. The proceeds will advance the ongoing pivotal T-RRex study of WU-CART-007 in relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma [50].

WU-CART-007, also known as soficabtagene geleucel, is a CD7-targeted, CRISPR-edited allogeneic CAR-T cell therapy with potential to be the first approved “off-the-shelf” CAR-T for T-cell malignancies. In a completed global Phase 1/2 study, WU-CART-007 achieved an overall response rate of 91% and a composite complete remission rate of 73% at the recommended Phase 2 dose. The median duration of response exceeded six months with manageable safety.

Funding Statement

The author(s) reported there is no funding associated with the work featured in this article.

Disclosure statement

Dusko Ilic is a member of the Regenerative Medicine Editorial Board. He was 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.