EMBODY Teaming Profiles 

Thank you for showing an interest in ARPA-H’s Engineering of Immune Cells Inside the Body (EMBODY) program. This page is designed to help facilitate connections between prospective proposers. If either you or your organization are interested in teaming, please submit your information via the form below. Your details will then be added to the list below, which is publicly available. 

EMBODY anticipates that teaming will be necessary to achieve the goals of the program. Prospective performers are encouraged (but not required) to form teams with varied technical expertise to submit a proposal to the EMBODY solicitation. For questions, please visit the EMBODY portal. 

EMBODY Teaming Profile Form

Please note that by publishing the teaming profiles list, ARPA-H is not endorsing, sponsoring, or otherwise evaluating the qualifications of the individuals or organizations included here. Submissions to the teaming profiles list are reviewed and updated periodically. 

Interested in learning more about the EMBODY program? 

Teaming Profiles List

To narrow the results in the Teaming Profiles List, please use the input below to filter results based on your search term. The list will filter as you type.

Organization namePoint of contact namePoint of contact emailProvide an additional point of contact for your organization's representative (email only)Location In 200 words or less, describe your organization's current research focus areasIn 200 words or less, tell us what your organization is looking for in potential teaming partnersWhich technical areas within EMBODY does your organization have the capacity to address?
BioCytics, IncBrent Dixonbdixon@biocytics.comrwarin@biocytics.comHuntersville, NCBioCytics, Inc., founded in 2005, is a health technology company with a focused mission to directly apply living cells in clinical trials as affordable individualized immune cellular therapies manufactured at the PoC. It is the only independent, privately held, warm-chain PoC cellular manufacturing facility in the US. Its affiliate, Carolina BioOncology Institute, PLLC (CBOI) is co-located adjacent to BioCytics Good Manufacturing Practices (GMP) facility. BioCytics primary research is focused on its proprietary Warm-Chain Point-of-Care (PoC) Manufacturing and Individualized Autologous Adaptive Cellular Therapy (AACT) Platform. This platform is focused on identifying the most effective patient specific/individualized immune cell therapy, by leveraging circulating tumor cells/cell clusters. BioCytics is fully vertically integrated Human Applications Laboratory (HAL), capable of producing maximally fit immune cells for optimized solid tumor agnostic (pan-cancer) therapeutics. BioCytics-CBOI infrastructure forms a fully integrated warm-chain PoC manufacturing platform where a patient’s immune cells can be collected, by blood draw or leukapheresis, immediately transferred to the adjacent GMP compliant HAL for AACT manufacturing. BioCytics HAL is a revolutionary manufacturing platform key to the production of autologous non-genetically modified cells with the highest specificity and functionality. BioCytics partners with cancer researchers to advance cancer research through its biospecimen sourcing efforts.Leukapheresis derived circulating tumor cells (CTCs), tumor derived cell clusters (TDCCs) and patient derived tumor organoids (PDTOs). GMP process optimization and scale up for manufacturing clinical trial cell therapy investigational product. CDMO including IND drafting and submission to FDA for phase 1 clinical trials. Clinical trial site for pan-cancer solid tumor cell therapies. BioCytics offers translational research services leveraging its cellular biology labs including advanced equipment including flow cytometry, video microscopy, impedance cytotoxicity assays and custom assays.Technical area 1: cell-specific delivery;Technical area 2: production and CMC;Technical area 2: preclinical models;
Case Western Reserve UniversityUmut Gurkanumut@case.eduumut@case.eduCleveland, OhioRelevant to EMBODY TA2-Validation/preclinical models: Disease and modality agnostic, IND-enabling advanced in vitro systems and validation assays for native and genetically engineered immune cells. Microengineered, multicellular microfluidic models of immune cell-vasculature and immune cell-tumor microenvironment interactions are ideal validation models. We develop microfluidics-based systems that are rationally designed to incorporate biochemical and microphysiological cues.   These in vitro models reveal the mechanisms behind engineered immune cells’ function, safety, off-target effects, and cellular fitness for individual patients. We have relevant experience to the EMBODY program’s TA2-Validation activities. Current and relevant research focus areas include modality-agnostic potency assay development enabling both ex vivo and in vivo genome editing therapeutics as part of the NIH Somatic Cell Genome Editing Consortium, and in vitro safety and validation assay development as part of a DARPA program. We have experience in developing, translating, and commercializing in vitro safety and potency models for emerging cell and gene therapies.Interested in collaborating with teams developing new gene editing and production strategies as part of the EMBODY program (TA1 and TA2). Technical area 2: preclinical models;
Form BioBrandi Cantarelbrandi@formbio.comclaire@formbio.comAustin, TXForm Bio is focused on developing tools for (i) CRISPR guide design including base and prime editing, (ii) construct design for larger editing applications such as PASTE and (iii) genome editing validation to evaluate on-target efficiencies and off-target effects. Our genome editing design tools generate guides across multiple editing modalities i.e. base, prime, HDR, which are provided as a ranked list to achieve the highest editing yield.  We account for genetic variation in target sites and potential bystander effects in base editing experiments.  For advanced prime editing applications, we host tools to help design engineered pegRNA for higher efficiency and find the best location for large PASTE constructs to be inserted.  Our genome editing validation tools can analyze amplicon and whole genome sequence (WGS) to determine the success of editing and off-target editing.  These in silico technologies for designing the editing-based therapeutic payloads are much faster than lab iterations and thus reduce time and cost to therapeutic development. Additionally, they complement innovations in designing delivery vehicles for ex vivo cell modification and in vivo gene modification, toward a complete immune cell engineering solution for the mitigation of devastating genetic diseases. Form Bio, a software company seeks partners with wet-lab capabilities, including the ability to perform editing experiments across diverse cell lines, animal models.   Additional partners could also include organizations that perform facilitate clinical trials including manufacturing organization and sequencing centers. Leveraging datasets from these experiments, we aim to develop and validate machine-learning models for enhancing the prediction of guide efficiencies and the positions and likelihood of off-target edits in both human and model organism cells. Technical area 1: genetic engineering ;
University of TennesseePaul Dalhaimerpdalhaim@utk.eduosp@utk.eduKnoxville, TNOur laboratory works on delivering nucleic acids to macrophages. We use soft nanocarriers that bind macrophage surface receptors to deliver the nucleic acids. The nucleic acids code for proteins that invigorate macrophages in the aged and obese.We are looking for partners that are interested in using macrophages to combat diseases. These diseases can include chronic inflammation, frailty, and poor immune response to exogenous diseases.   Technical area 1: cell-specific delivery;
Spring ScienceDr. Hope O'Donnellhope@springscience.comrrpv@springscience.comSan Carlos, CASpring Science is putting advanced AI image analysis tools in the hands of researchers across a broad spectrum of applications, using cellular or histologic morphology, phenomics, and proteomics to solve complex mechanisms, screen compound libraries, assess clinical biomarkers, and more. This platform is ideally suited to complex population response comparisons in primary cells, such as PBMC. 


We have developed in vitro predictive models on human PBMC for various applications, including an aging model used to predict which adjuvants could best overcome cellular senescence, and a model of in vivo immunogenicity used for adjuvant development, including formulation selection, and relative potency readouts for complex combinations of innate ligands.


Our platform is ideally suited for the development of predictive preclinical models of the human immune response in a wide variety of disease contexts.		While initial model building can be done with commercially obtained healthy donor material, potential partners should have (or intend to have) access to either relevant patient samples, such as frozen PBMC, or relevant metadata (eg, test results, disease status, treatment outcomes, etc) that can be used to assess and improve models.Technical area 2: preclinical models;
Throne BiotechnologiesYong Zhao, MD, PhDyong.zhao@thronebio.comconnect@thronebio.comParamus, NJThrone Biotechnologies (Throne) is a clinical-stage therapeutic company with a disruptive stem cell technology that can fundamentally reverse type 1 diabetes (T1D), alopecia areata (AA) and other autoimmune-/inflammation-associated diseases (e.g., Lupus, Eczema, and long COVID) through the immune education of Stem Cell Educator therapy (“Educator Therapy”) at the root causes, based on the reprogramming/engineering of different types of immune cells by using novel human cord blood stem cells (CB-SC). Over last 12 years, international multicenter clinical trials in the United States, China, and Spain have strongly demonstrated the clinical safety and efficacy of Educator therapy in more than 300 patients aged from 3 to 77 years old, with over 30 peer-reviewed publications in basic research and clinical studies. Currently, Throne has received three FDA-approved phase 2 INDs by using Educator therapy to treat T1D, AA, and COVID-19 patients. Specifically, Educator therapy is recognized as the leading “Practical Cure Project” for T1D out of 607 global projects. Throne was awarded one of the “5 Best BioTech Companies To Watch” by Silicon Review. Throne has research lab for Quality Control (QC) testing and preclinical studies, the GMP facility for manufacturing Stem Cell Educators and the Outpatient Clinical Facility for treatments of patients. Throne has ongoing clinical trials by using Stem Cell Educator therapy to treat patients with type 1 diabetes and other autoimmune- or inflammation-associated diseases. To discover more biomarkers for diagnosis and improve the clinical efficacy of Stem Cell Educator therapy, Throne is seeking potential partners: 1) that have the capability to perform the single cell RNA sequencing, data analysis, and dissect the gene expressions of different immune cell compartments; 2) that have the capability for the purification of exosomes, microRNAs and proteomics profiling; 3) have the expertise to characterize different types of immune cells such as monocytes/macrophages, T cells and B cells in autoimmune diseases or cancer immunology. Technical area 2: production and CMC;Technical area 2: preclinical models;Technical area 1: genetic engineering ;Technical area 1: cell-specific delivery;
Nanite Inc.Thomas Neenanthomas@nanitebio.comshashi@nanitebio.comBoston, MANanite develops polymer nanoparticles (PNPs) for the safe and efficient delivery of genetic cargoes. The integration of fast synthesis of small focused polymeric libraries, multiplexed in vivo studies, and proprietary machine learning models enables our SAYERTM platform to rapidly explore a design space vastly exceeding what is possible with lipid nanoparticles (LNPs) or viral vectors. We focus on fully excretable/resorbable systems that allow repeat dosing with acceptable safety profiles. We emphasize simple synthetic polymerization and functionalization from readily available starting materials, ensuring acceptable cost of goods for our clinical candidates. Our current focus (with multiple early commercial partnerships) is centered on the development of PNPs that afford efficient delivery to the lung, immune cells (in vivo CAR-T), kidney and the peripheral nervous system. Nanite is interested in partnering with entities who have proprietary genetic cargoes that control gene expression, gene regulation, or gene editing. We have a particular interest in identifying partners focused on indications that require repeat dosing, and where the immunological, toxicity or costs constraints of viral vectors or LNPs may be clinically limiting. We believe that our machine learning driven approach to develop highly specific polymeric delivery vehicles from readily available starting materials offers considerable advantages in cost of goods and speed to market. We have ongoing robust programs in the development of polymeric vectors with specificity for immune and lung tissue but are interested in expanding to other tissues and indications. We are committed to working with partners with innovative biological approaches to together develop practical solutions for the expansion of genetic cures to a much-broadened patient population. Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
Syenex Inc.Jay Rosanellijrosanelli@syenex.comjleonard@syenex.comEvanston, ILSyenex is dedicated to creating and universally disseminating biotechnologies that enable developers of immune and stem cell therapies. Syenex applies its expertise in synthetic biology, extracellular vesicle engineering, and scale up process development to address the specific challenges facing both upstream R&D and downstream clinical challenges for cell and gene therapy developers. Syenex was launched with enabling IP in the fields of: (i) engineering enveloped vectors for targeted, efficient, and specific delivery and (ii) engineering cellular expression technologies including stable cell lines and novel genetic expression control systems. This expertise is supported by a proprietary technology stack which enables ready and broad commercial access to these innovations—unlocking transformative innovations in biotechnology for the entire cell and gene therapy industry.

Of particular relevance for this program, Syenex has developed and offers products for enhanced and specific gene delivery to primary human T cells, including ex vivo, extracorporeal, and in vivo routes of administration. This technology has been validated for use with standard state-of-the-art lentiviral packaging vectors, and it is designed to be extensible to related classes of enveloped vehicles, for delivery of multiple types of cargo, and to target novel immune cell subtypes.		Syenex is interested in expanding our network and teaming structure to collaborate on projects aligned with this program. We are particularly interested in: (1) partners with therapeutic assets in need of a delivery and manufacturing solution and: (2) partners developing novel in vitro and in vivo testbeds, as evaluation frameworks for the vectors, vehicles, and agents we seek to develop. Technical area 1: genetic engineering ;Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
GeneFab, LLCMichelle Hungmichelle.hung@genefab.comyinyinchong@genefab.comAlameda, CAGeneFab, LLC is a CRDMO focused on supporting the design and manufacture of genetic medicines. Our Synthetic Biology R&D facility in South San Francisco (California) specializes in the high throughput design, screening, and optimization of technologies that enhance the performance, precision and safety of cell, gene, and nucleic acid therapies (CGNT). GeneFab's cGMP facilities in Alameda (California) support the manufacture of genetic medicines, including process development, analytical development, QC, and GMP for viral vectors, pDNA, mRNA, LNP, and cell therapies. A third site in Singapore is focused on high quality manufacturing while reducing COGS. 
Our synthetic biology discovery platform supports genetic medicine design, and we have completed successful partnerships with leading cell and gene therapy companies to build potent and compact promoters specific to a desired cell type or cell state, NK and T cell logic gates, small molecule regulated switches, and master regulators of immune cell states. GeneFab’s Research team has a diverse background including expertise in bioinformatics and machine learning, promoter and UTR discovery, high throughput screening, protein engineering, and immunology.
 		We are looking for partners with expertise in design and targeting of in vivo delivery vehicles for genetic cargo (TA1). We are also looking for partners to lead preclinical modeling (TA2), clinical development (TA 1 & 2), and commercialization (TA 1 & 2). 

GeneFab specializes in the design of novel genetic cargoes, and functionally validating these designs via high throughput assays in therapeutically relevant cell types to enhance safety and specificity (TA1: genetic engineering). GeneFab’s team has a proven track record of designing synthetic promoters and UTRs that are highly specific for therapeutically relevant cell types or cell states. These synthetic elements can mitigate potential adverse side effects stemming from ectopic cargo expression and enhance the precision of our collaborator’s in vivo delivery vehicles (TA1: cell-specific delivery). GeneFab’s team can also design payloads that transform cell states (e.g., from anti-inflammatory to pro-inflammatory). To further enable our partner’s clinical development and commercialization of the therapeutic, GeneFab’s manufacturing team would focus on innovations to reduce COGs by improving and optimizing individual steps of the manufacturing process such as vehicle packaging efficiency (TA2: production and CMC).
 		Technical area 1: genetic engineering ;Technical area 2: production and CMC;
USC Keck School of MedicineProfessor Paula Cannonpcannon@usc.edu Los Angeles, CAThe Cannon lab at USC are experts in genome editing of HSC, T cells and B cells. We have developed novel protocols to engineer B cells at the Ig locus to express custom antibodies or non-antibody molecules, in ways that preserve normal B cell functions. We are currently evaluating therapeutic molecules that target HIV, COVID, cancer and neuropathologies, including molecules that could cross the blood-brain barrier. The engineered cells are evaluated using a suite of pre-clinical models based on humanized mice, human tonsil organoids, and NHPs. We also work with delivery vectors capable of in vivo delivery to B cells or T cells. We are therefore interested to further develop in vivo B cell engineering for long-term secretion of therapeutic molecules, and in ways that can be boosted by immunization.1. Partners with candidate therapeutic molecules, or an interest in a specific disease application, that could benefit from the long-term secretion of a molecule from engineered B cells. 

2. Partners developing genetic engineering technologies compatible with site-specific insertion of large gene cassettes, that could be combined with our Ig locus engineering approach.

3. Partners with alternate in vivo delivery technologies that could be used to target B cells.
 		Technical area 1: genetic engineering ;Technical area 1: cell-specific delivery;Technical area 2: preclinical models;
Optimeos Life Sciences, IncChet Markwaltercmarkwalter@optimeos.com Princeton, NJOptimeos Life Sciences is commercializing a non-viral gene therapy platform developed at Princeton University and designed to overcome the targeting and inflammation limitations that plague existing approaches. Our CINC nanoparticles deliver mRNA or DNA to cells with equivalent potency to lipid nanoparticles but with drastically reduced inflammatory responses. Our unique architecture also allows us to stably anchor targeting ligands onto the nanoparticle surface for cell-specific delivery. Crucially, we maintain potency in the target cell type while drastically curtailing non-specific uptake.
We achieve this through two nanoparticle assembly steps that are carried out with a scalable technology called inverse Flash NanoPrecipitation, which uses specially designed mixers to rapidly drive nanoparticle self-assembly. The same mixers have been used by the Gates Foundation and multiple pharmaceutical companies for clinical nanoparticle production. 		We are looking for partners that can (1) contribute genetic cargo design expertise and (2) develop preclinical models.

We are well-positioned to continue development of our cell-specific delivery vehicle but would look to expand our sophistication in cutting-edge cargo design through partnering. Particular experience with non-viral delivery would be most aligned with our platform. We also would welcome partnerships with groups looking to address the preclinical model question.		Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
Senti Biosciences, Inc.Nicholas Frankelnicholas.frankel@sentibio.comthomas.chung@sentibio.comSouth San Francisco, CASenti’s research focuses on designing genetic payloads and regulatory elements for next-generation gene and cell therapies. Our discovery is driven by a high throughput research engine comprising bioinformatics, pooled screening, and liquid handling robotics. We design cargos that imbue cells with therapeutic functions against cancer or autoimmune disease, comprising both protein-coding payloads and non-coding gene regulatory sequences. 

Protein-coding payloads include chimeric antigen receptors targeting tumor- or autoreactive cell-associated antigens, including logic-gating to target multiple subtypes and/or de-target healthy cells. Our cytokine-based systems support expansion and persistence of engineered cells with fine-tuned levels of cytokine release and optional control by dosage of a clinically approved drug. 

Non-coding gene regulatory sequences ensure that expression of transgene(s) is restricted to the intended target cell type and/or cell state, such as activated immune cells. We have engineered promoters to be stronger than commonly used constitutive promoters in cell and gene therapy to achieve higher abundance of CAR molecules, correlating with higher therapeutic function. 

Additionally, Senti also has highly experienced and relevant clinical development, CMC, and regulatory capabilities that it can leverage to advance the EMBODY program into and through the clinic, as demonstrated by our clinical stage SENTI-202 logic-gated CAR cell therapy. 		Delivery vector and manufacturing: We are looking for partners with expertise in delivery vectors for in situ cell therapy that are targeted to immune effector cells and de-targeted to the liver, into which we could load a Senti designed genetic cargo. With our expertise in promoter design, we can provide genetic regulatory elements that further ensure that the cargo is silent in off-target cells and robustly expressed in on-target cells, as well as payload genes that will mediate therapeutic function. The ideal partner would also have CMC capabilities and a vision for achieving sufficiently low COGS. 

In vivo models: We are looking for partners with expertise in developing novel humanized mouse models that recapitulate multiple immune and non-immune cell populations required for evaluating therapies in the context of cancer or auto-immune disorders while de-targeting the liver and avoiding other serious adverse effects such as cytokine storm. Given our expertise in high throughput pooled screening and NGS, we are interested in collaborating on developing novel usages of these models to test large arrays of product candidates simultaneously, dramatically increasing the amount of data per mouse. 		Technical area 1: genetic engineering ;
Laguna Biotherapeutics, Inc.Jonathan Kotulajkotula@lagunabio.comkrcarrington@lagunabio.comSan Francisco, CALaguna expands and activates endogenous gamma delta T cells, and directs them to kill specific cells for oncology, infectious disease, and auto-immune indications. Our method has proven safety and MoA in people, including immune-compromised cancer patients. We have an exploratory platform that offers a unique delivery technology. GMP manufacturing for our method is streamlined and an order of magnitude less complex and less expensive that ex vivo cell expansion. Our delivery method is exploratory and is inclusive of our endogenous immune cell expansion technology, which is an order of magnitude less complex and less expensive than viral vectors or LNP delivery technology. We are looking for partners who have expertise in genetic cargo and immune cell engineering, as well as partners who have expertise in developing humanized in vivo models.Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
University of Colorado - Gates InstituteLaura Borgeltlaura.borgelt@cuanschutz.educhristopher.garbe@cuanschutz.eduAurora, ColoradoGates Institute (https://gates.cuanschutz.edu/) at the University of Colorado Anschutz Medical Campus (CU-AMC) was established with the mission of facilitating translation of academic and industry-partnered novel cell and gene therapies and protein biologics. The Gates Biomanufacturing Facility (GBF; www.gatesbiomanufacturing.com), a state-of-the-art GMP facility, manufactures investigational cell/gene therapy and protein biologic products to support early phase clinical trials. The GBF consists of 25,000ft2 of ISO classified cleanrooms dedicated to cell therapy or protein biologics manufacture, translational sciences, process/analytical development, QC, and material and cryogenic storage. GBF has successfully developed a wide array of cell/gene/protein therapy candidates and manufactured >130 GMP runs in support of multiple FIH INDs. 
Being co-located on the CU-AMC enables Gates Institute/GBF to directly collaborate with the University of Colorado School of Medicine, University of Colorado Health, and Children’s Hospital Colorado to translate and conduct FIH clinical trials. Our current research focus includes immune effector cells coupled with CARs or other engineered modifications, synthetic receptor based cellular therapies, RNA (RNAi, mRNA, CRISPR/Cas based editing), and recombinant proteins. Coupled with exceptional adult and pediatric clinical programs in oncology, autoimmunity, diabetes, etc., our campus is well positioned to address and advance next generation cell/gene therapies.		While the CU-AMC possesses expertise in genetic engineering and cell-specific delivery, we would seek potential partners with complementary approaches that could supplement or enhance our current capabilities and broaden potential clinical targets, whether cell type or disease indication. Furthermore, Gates Institute in collaboration with our investigators continues to develop non-clinical disease models to support fundamental proof of concept, biodistribution, and efficacy studies. The opportunity to partner and develop more universally representative and robust disease models would accelerate early lead target identification and validation, improve and provide more clinically relevant potency/selectivity assessments, and drive efficient translation of in vitro/in vivo demonstrated therapeutic candidates to FIH clinical trials.Technical area 1: genetic engineering ;Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
ImmunoVec Ryan Wong, Ph.D.ryan@immunovec.comluke@immunovec.comLos Angeles, CAImmunoVec provides an innovative solution to cell-type specific targeting of Cell and Gene therapies by designing transcriptional control elements that regulate the expression of therapeutic payloads post-delivery. Our library of cell-type specific gene regulatory elements, including enhancers and promoters, can be incorporated into any delivery vehicle, viral or non-viral, ensuring precise and tunable expression within specific immune cells (T cells, NK cells, M2 macrophages, Tregs, etc.)
ImmunoVec’s gene regulatory elements will express the therapeutic payload only in specific target cells and specific cell states, even if the payload is inadvertently delivered to off-target cells. 
ImmunoVec also has a sequence optimization platform that increases viral titer (production of functional viral particles per mL) and transduction efficiency by over 30-fold. This platform increases the yield of viral particles per GMP manufacturing batch and lowers the dosage of particles required for effective treatment, enabling the development of curative therapies at a fraction of the cost of traditional cell and gene therapies. Using our technology, we have developed multiple cell-type specific gene therapies that are approaching clinical trials.
Our team also has extensive experience in multiple advanced humanized mice models reconstituted with a full human immune system for preclinical evaluation of therapeutic candidates.
 		ImmunoVec, a gene regulation company, is open to exploring partnerships with groups that have in vivo delivery vehicles able to deliver DNA payloads to immune and hematopoietic cells. Additional candidate partners may also include groups with broad tropism delivery vehicles capable of delivering DNA payloads (without immune cell specificity).

ImmunoVec specializes in the design of novel expression cassettes able to drive fine-tuned expression of any DNA encodable payload in specific blood and immune cells. Through our platform we can design highly regulated expression cassettes to be used in both viral and non-viral gene therapies to express a variety of cargos including: chimeric antigen receptors, therapeutic genes, monoclonal antibodies, etc. into specific cell lineages and at fine-tuned expression levels. This progress is driven forward by a stealth team of experienced computational and biological scientists recruited from top academic institutions and industry, world-class physicians with decades of experience leading cell and gene therapy clinical trials, FDA regulatory experts, and top executives from large tech companies including Google, and experienced entrepreneurs with a record of success across multiple companies.
 		Technical area 1: genetic engineering ;Technical area 2: production and CMC;Technical area 2: preclinical models;
The Jackson LaboratoryDr. Leonard ShultzLenny.shultz@jax.orgJenna.Gerrish@jax.orgBar Harbor, METhe Jackson Laboratory is an independent non-profit biomedical research organization encompassing mouse genetics, human genomics, and computational modeling to define the underlying biology of a broad spectrum of diseases. Professor Leonard Shultz directs a research lab focusing on the development, optimization, and validation of immunodeficient mouse models that support engraftment and function of normal and malignant human cells and tissues. Based on the NOD/SCID/Gamma (NSG) mouse model developed in his lab, Dr. Shultz and his colleagues developed and are improving a growing panel of next generation NSG mice expressing human HLA and cytokine transgenes and carrying targeted mutations that eliminate mouse MHC, further depress host innate immunity, and heighten human immunity. These models engraft robustly with human HSC, and PBMC and support the development of functional human immune systems, including T cells, B cells, NK cells, macrophages, and dendritic cells. These humanized mouse models are widely used to study human immunotherapy against cancer as well as human autoimmune diseases in vivo. Ongoing collaborative studies with Drs Michael Brehm and Dale Greiner at the University of Massachusetts Chan Medical School are applying these models in preclinical studies of human malignancy and type I diabetes as well as other human autoimmune diseases.We are looking for collaborators that need to test human therapeutics in vivo. The humanized mouse models that Dr. Shultz has developed are ideal for the evaluation of therapeutic modalities targeting human cell populations without putting patients at risk. These collaborations can also leverage the UMASS Chan Humanized mouse Core, that is co-directed by Drs. Brehm and Greiner. The core has extensive experience in engrafting and validating the models developed by Dr. Shultz and has expertise in the study of human immune system function and homeostasisTechnical area 2: preclinical models;
Southwest Research InstituteJian Ling, Ph.D.jling@swri.org San Antonio, TexasSouthwest Research Institute (SwRI) is a nonprofit contract research organization. We cover almost all areas of physical science and engineering. Relevant to this program, we have production and CMC capabilities. Particularly, we have a innovative manufacturing platform for automatable and scalable manufacturing of cells and viral vectors for gene delivery. We also have a proprietary technology for specific cell delivery of DNA plasmids for in vivo gene therapy.   We looking for partners who are experts in genetic engineering and preclinical models. Technical area 1: cell-specific delivery;Technical area 2: production and CMC;
Velvet TherapeuticsClaudia Leeclaudia.lee@velvettherapeutics.comchris.coker@velvettherapeutics.comHouston, TXSpun out of Baylor College of Medicine, Velvet Therapeutics is a preclinical, non-viral, DNA-driven in vivo cell and gene therapy company focused on solid tumors. We aim to reprogram the tumor microenvironment of immunosuppressing solid tumors in vivo by engaging multiple cytotoxic immune cell types, principally t cells and macrophages (especially tumor-associated macrophages).

Our research program comprises:

• The design of immune modulating chimeric antigen receptors (CARs), including related cell type-specific control elements.
• The design of safe, CAR-encoding, circular, compact, supercoiled, ultrapure DNA particles that afford controlled, durable in vivo expression and are redoseable (DNA-CARs).  
• The design of safe, biodegradable, branched, amino acid polymers (Star) that afford tuned, targeted systemic and local delivery to the lymphatic system, lung, and others.
• The complexation and targeted delivery of DNA-CAR Star polyplexes to t cells and macrophages.		We are looking for partners to 1) identify appropriate preclinical models and to validate CAR targets and 2) to scale up manufacturing, evaluate the quality attributes of different proof-of-concept production schemes and to develop relevant assays. Technical area 1: genetic engineering ;Technical area 1: cell-specific delivery;
KiraGen BioAaron Edwardsaaron@kiragenbio.comryan@kiragenbio.comCambridge MAKiraGen Bio is at the forefront of developing next-generation CAR-T cell therapies aimed at overcoming the complexities of the tumor microenvironment (TME) in solid tumors. Our research centers around two critical areas:

TME Navigation: We focus on understanding and manipulating the TME to enhance the efficacy of CAR-T cells. Our approach involves creating CAR-T cells that are resistant to the suppressive and hostile conditions typically found in solid tumors, enhancing their ability to survive and function effectively.
Combinatorial Gene Editing: Utilizing an AI-driven platform, we are pioneering the rational design of gene edits to produce CAR-T cells optimized for various TME profiles, rather than individual patient genetics. This strategy enables the development of robust CAR-T cells that can adapt to diverse tumor conditions, potentially increasing the success rate of treatments across a broader patient base.
By integrating advanced AI with deep biological insights, KiraGen Bio aims to revolutionize CAR-T cell therapies for solid tumors, focusing on scalability and effectiveness in challenging tumor environments.		KiraGen Bio specializes in understanding the tumor microenvironment (TME) biology and advanced combinatorial gene editing techniques. We are looking for partners with innovative delivery systems capable of:

Targeted Delivery: Technologies that can accurately deliver therapeutic agents to specific immune cell populations within the TME. This precision is crucial for ensuring that our CAR-T cells can reach and exert their effects in the desired locations within solid tumors.

Complex Payload Handling: Systems that can effectively handle and deliver multiple sgRNAs and mRNA payloads. These capabilities are essential for implementing our high-order gene edits, enabling us to program immune cells with multiple genetic instructions simultaneously.

Partners should have proven expertise in nanotechnology, lipid nanoparticles, or other delivery vectors that meet these requirements and are adaptable for clinical use. Collaborators who can contribute to the rapid and efficient scaling of these technologies will help us achieve our goal of transforming CAR-T cell therapies to be more effective and accessible for treating solid tumors.		Technical area 1: genetic engineering ;Technical area 2: preclinical models;
STRM.BIO, Inc.David Raiserdmr@strm.bio Cambridge, MASTRM.BIO has developed a non-viral, extracellular vesicle (EV)-based in vivo delivery technology that is fully liver-detargeted and demonstrates striking bone marrow specificity, with the ability to directly modify T-cells and B-cells in vivo. STRM.BIO EVs are non-immunogenic (i.e. can be repeat dosed) and have larger carrying capacity than most viruses and synthetic nanoparticles. The technology value proposition is supported by strong in vivo proof of concept data across a variety of models and in both small and large animals. The company is currently focused on process optimization (including cost-reduction measures in manufacturing and loading/formulation optimization) and demonstrating therapeutic efficacy in multiple indications.STRM.BIO's core competency is cell-specific delivery, with strong competencies and/or partners in genetic engineering and small-to-medium scale process development and production. To achieve all technical objectives of the EMBODY solicitation, we envision the need to partner extensively, particularly in the areas of (1) large scale production and CMC, (2) genetic engineering/therapeutic cargo development, and (3) preclinical model development.Technical area 1: cell-specific delivery;Technical area 2: production and CMC;Technical area 1: genetic engineering ;
Portal BiotechnologiesMathias Pawlakmathias@portal.bioelizabeth@portal.bioWatertown, MAPortal has developed a next generation intracellular delivery platform for highly efficient, cost-effective cell engineering for a broad range of cell types and cargos. Portal has demonstrated robust delivery of many cargos (mRNA, siRNA, CRISPR, proteins, peptides) into diverse cell types including stem cells and primary human immune cells (T, B, NK cells, HSCs and iPSCs) while maintaining normal cell function. 
As opposed to shipping donor cells to centralized laboratories for cellular engineering, which carries substantial costs and limits accessibility and adoption of novel therapeutics, in vivo   and point-of-care cell engineering will constitute dramatically advanced approaches for cell therapeutics. Enhancing immune cell function in vivo will be an important cornerstone of medicine and the EMBODY program will facilitate this revolution. However, in vivo organ and cell type-specific immune cell engineering may face key obstacles and limitations due to lack of targeting mechanisms. Therefore, point-of-care cellular engineering approaches will make an important contribution to next generation therapeutics in settings that require highly targeted and complex cellular changes - especially to the immune system.		Within the EMBODY program, Portal is specifically looking to collaborate with partners from both academia and industry that envision a point-of-care cellular engineering approach. We are looking for technologies that are synergistic to ours to increase value and accessibility and reduce costs of the therapy. 
Portal Biotechnologies envisions a point-of-care approach that retrieves primary patient cells and engineers them immediately to promptly administer them back. Specifically, in situations in which multiple gene deletions and mRNA libraries are required for therapeutic effect in which in vivo approaches will not be successful, our technology will provide a critical solution, substantially advancing the field of cell therapies.		Technical area 1: cell-specific delivery;