Malin Corporation plc (“Malin”) is an Irish incorporated public limited company whose head office is in Dublin, Ireland with subsidiary offices in New Haven, Connecticut and Research Triangle, North Carolina, USA.   Malin’s purpose is to create shareholder value through the selective long-term application of capital and operational expertise to private, pre-IPO, pre-trade sale operating businesses in dynamic and fast growing segments of the life sciences industry. Through its operational involvement, Malin works with its investee companies to enable them to reach the full potential of their value proposition and to achieve commercial success.

Malin Corporation

Longitude Capital specializes in making venture growth investments in biotechnology and medical technology companies that seek to improve clinical outcomes, enhance the quality of life, and/or reduce system costs. They invest in both privately held and publicly traded life science companies through a variety of investment approaches. Since 2006, Longitude Capital has raised over $1.2 billion across three funds and demonstrated an ability to source, manage and exit attractive investments across multiple market cycles. Their venture growth strategy is rooted in building a balanced portfolio of clinical-stage and commercial-stage companies with clinically de-risked assets, favorable valuations relative to risk profile, and attractive expected returns within three to five years from the initial investment. Longitude Capital seeks to identify new investment opportunities by tapping into their broad network of industry relationships or through the execution of intensive thematic research into emerging therapeutic areas or industry sub-sectors. They utilize a variety of investment structures, including traditional venture capital, asset spin-outs, recapitalizations, PIPEs, open market purchases, royalties, and other equity and equity-linked instruments.

Longitude Capital

Tavistock Group is an international private investment organization comprised of a family office, trading operation, and a portfolio of public investments and private companies. These investments are concentrated in nine primary sectors, including: real estate, hospitality, restaurants, sports, energy, retail, biotechnology and life sciences, agriculture, and finance.

Tavistock Life Sciences

The firm is a 25-year-old global investment firm focused on healthcare with approximately $5.8 billion in AUM, which we have invested in over 290 public and private companies worldwide. Headquartered in Palo Alto, California, with offices in Asia.  Our team consists of 50 multi-disciplinary professionals, including, physicians, scientists, entrepreneurs, operating executives, and industry experts. The firm operates as a multi-fund investment platform, covering growth equity, private equity including buyout, venture capital, and public equity. Vivo invests broadly in healthcare across all fund strategies, including biotechnology, pharmaceuticals, medical devices, and healthcare services, with a focus on the largest healthcare markets.

Vivo Capital

Novartis is a global healthcare company based in Switzerland that provides solutions to address the evolving needs of patients worldwide.

Novartis

Aisling Capital believes the next decade will be marked by a revolution in healthcare driven by new therapeutics generated by biotechnology. The completion of the human genome has given scientists new insights into the causes of human disease. These insights, combined with the past 20 years of developments in the biotechnology industry, are leading to rapid expansion of novel approaches toward the diagnosis, prevention, and treatment of life-threatening illnesses. These advances have led to an ever-increasing demand for capital to complete the development and commercialization of new therapeutics. Our goal is to support the leading global healthcare companies that are building on these technical and medical breakthroughs to commercialize new healthcare products.

Aisling Capital

Perceptive Advisors is a privately owned hedge fund sponsor. The firm invests in the public equity markets across the globe. It employs a long/short equity strategy as a hedging technique while investing. The firm typically invests in the biotechnology and life sciences sectors to make its investments. Perceptive Advisors is based in New York, New York.

Perceptive Advisors

Boxer Capital caters to public companies based in the biotechnology industry.

Boxer Capital

Rapha Capital Management  focused on identifying and growing revolutionary ideas in medicine, healthcare and technology before others can see their value. For inquiries, telephone and contact details are used.

Rapha Capital Management

Pentwater Capital Management Is an investment firm

Pentwater Capital Management

Fidelity is a privately owned investment management firm that serves a wide range of clients. It offers services including IRAs, rollovers, annuities, discount brokerage, retirement planning, estate planning, wealth management, securities execution and clearance, and life insurance. They serve individual investors, businesses, financial advisors, and institutions. The firm manages equity, fixed-income, and balanced mutual funds and invests in public equity and fixed-income markets. It also conducts in-house research to inform its investment decisions.

Fidelity

Adage Capital Management is a Boston-based $8.9B Money Management firm focused on managing S&P 500 assets predominantly for Endowments and Foundations such as Harvard University, Northwestern University, Dartmouth College, the American Red Cross and the Getty Foundation. Adage and its predecessor, the Select Equity Group at the Harvard Management Company, has outperformed the S&P index in each of the last 15 years by an average of 3.5%.   The fund is run by Phill Gross, who prior to founding Adage, was a Healthcare and Retail analyst, equity research director and partner at the Harvard Management Company for 18 years..

Adage Capital Management

Schonfeld Strategic Advisors LLC (''Schonfeld'') is a multi-manager platform that invests its capital with Internal and Partner portfolio managers, primarily on an exclusive or semi-exclusive basis, across quantitative, fundamental equity and tactical trading strategies. Schonfeld and its predecessors have successfully capitalized on inefficiencies and opportunities within the equity markets. We have developed and invested heavily in proprietary technology, infrastructure and risk analytics. Our portfolio exposure has expanded across the Americas, Europe and Asia as well as multiple asset classes and products.

Schonfeld Strategic Advisors

Poseida Therapeutics

Poseida Therapeutics is a clinical-stage biopharmaceutical company utilizing genome engineering capabilities to develop targeted therapeutics for patients with high unmet medical needs. The company develops a pipeline of autologous and allogeneic chimeric antigen receptor T cell or CAR-T, product candidates focusing on the treatment of hematological malignancies and solid tumors.

Poseida Therapeutics specializes in gene editing to develop novel therapeutics for a range of liquid and solid tumors using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops autologous (using cells from the patients themselves) and allogeneic (using cells from other individuals) gene-edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells.&nbsp;

Reinventing gene therapies with next-generation technologies

Human Gene Editing

<div>Poseida Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapeutics with the potential to cure various diseases. The company utilizes proprietary genetic engineering platform technologies, including the non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System, and nanoparticle- and AAV-based gene delivery technologies. These platforms, either alone or in combination, enable Poseida to engineer a portfolio of product candidates designed to overcome limitations of current cell and gene therapeutics.</div>

Poseida Therapeutics Announces Proposed Public Offering of Common Stock

Poseida Therapeutics Appoints Brent Warner as President, Gene Therapy

Poseida Therapeutics Hosts Second Annual Virtual R&D Day Highlighting Novel Pipeline Assets and Latest Technology Innovations

Therapeutics that target specific areas/tissues to minimize treatment side effects.

Tissue Targeting Therapeutics

Poseida Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for cancer and rare diseases. The company's technology platform includes its proprietary non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-Specific Gene Editing System, and nanoparticle and hybrid gene delivery technologies. Their gene therapy pipeline includes P-KLKB1-101 for hereditary angioedema and P-FVIII-101 for hemophilia A. The company's cell therapy portfolio features allogeneic CAR-T cell therapy candidates targeting both solid and liquid tumors.&nbsp;

Novel therapies to reboot cells and organs

Cell & Gene Therapy

Roche: launch of takeover bid for Poseida Therapeutics

Roche

To enhance Roche's portfolio in advanced immune cell therapies, addressing declining oncology sales and expanding capabilities in hematologic malignancies and solid tumors.

Chimeric antigen receptor T-cell (CAR-T) therapy involves genetically engineering T cells to target and kill cancer cells

Astellas Pharma

To develop two allogeneic cell therapies for solid tumors. Astellas will make an upfront payment of USD 50 million to Poseida, with an additional USD 550 million possible based on meeting certain goals.

Companies that develop novel therapeutics based on the CRISPR/Cas9 gene-editing technology

AstraZeneca

Poseida Therapeutics receives USD 50 million investment from Astellas Pharma

To enter ia strategic collaboration and license agreement that is focused on developing allogeneic CAR-T therapies directed to hematologic malignancies including P-BCMA-ALLO1 (RG6538).

Takeda

The companies will collaborate to develop up to six in vivo gene therapy programs using Poseida’s tech platforms, including piggyBac, Cas-CLOVER, and biodegradable nanoparticles.

<div>
 
 Poseida Therapeutics specializes in gene editing to develop novel therapeutics for a range of liquid and solid tumors using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops autologous (using cells from the patients themselves) and allogeneic (using cells from other individuals) gene-edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells.&nbsp;
 
 
 Poseida operates a suite of platforms including non-viral piggyBac DNA Modification System for gene insertion, Cas-CLOVER for site-specific gene editing, and gene delivery technologies (in vivo and ex vivo) based on nanoparticle and adeno-associated virus (AAV) vectors. The company has demonstrated low to almost no off-target activities in the company&rsquo;s preclinical studies using its Cas-CLOVER platform, which it considers to be an advantage over other existing gene editing systems.
 
 
 As of March 2024, the company has six programs in its CAR-T pipeline focused on oncology, of which three have reached Phase I clinical trials. It also has three gene therapies to treat Hemophilia A, Ornithine transcarbamylase deficiency (OTCD), and Phenylketonuria, all of which were in the preclinical stages . The company fully owns two of its CAR-T therapies for solid tumor indications and all of its gene therapies, while its remaining cell therapies are a part of a collaboration with Roche.&nbsp;
 
 
 Key customers and partnerships
 
 
 The company announced in August 2022 that it had entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. The global collaboration covered the research and development of multiple existing and novel &quot;off-the-shelf&quot; cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications. 
 
 
 Funding and financials
 
 
 Poseida Therapeutics went public on the Nasdaq Global Select Market in July 2020, raising gross proceeds of USD 224 million. The net proceeds were earmarked to accelerate its clinical development efforts. For 2021, it recorded revenue of USD 31.2 million earned through collaborations.
 
 
 
 
 The company last raised funds in August 2023, when it raised USD 50 million through a strategic investment from Astellas. The funding comprised of the purchase of shares of common stock for an aggregate purchase price of USD 25 million and an additional USD 25 million one-time payment for certain strategic rights, and provided a business update. The company did not disclose what the funds would be used for.
 
</div>

Poseida Therapeutics specializes in gene editing to develop novel therapeutics for a range of liquid and solid tumors using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops autologous (using cells from the patients themselves) and allogeneic (using cells from other individuals) gene-edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells.&nbsp;Poseida operates a suite of platforms including non-viral piggyBac DNA Modification System for gene insertion, Cas-CLOVER for site-specific gene editing, and gene delivery technologies (in vivo and ex vivo) based on nanoparticle and adeno-associated virus (AAV) vectors. The company has demonstrated low to almost no off-target activities in the company’s preclinical studies using its Cas-CLOVER platform, which it considers to be an advantage over other existing gene editing systems.As of March 2024, the company has six programs in its CAR-T pipeline focused on oncology, of which three have reached Phase I clinical trials. It also has three gene therapies to treat Hemophilia A, Ornithine transcarbamylase deficiency (OTCD), and Phenylketonuria, all of which were in the preclinical stages . The company fully owns two of its CAR-T therapies for solid tumor indications and all of its gene therapies, while its remaining cell therapies are a part of a collaboration with Roche.&nbsp;Key customers and partnershipsThe company announced in August 2022 that it had entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies. The global collaboration covered the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications.

<div>
 
 Editas Medicine specializes in gene editing to develop novel therapeutics for a variety of areas including oncology, ocular diseases, and blood diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 and Cas12a enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, Editas has seven gene editing programs in its pipeline.&nbsp;
 
 
 One of the company&rsquo;s candidates was in the late clinical trial stages as of March 2024. The Phase I/II clinical trials on Editas&rsquo; in vivo gene editing therapy EDIT-101 have been ongoing since 2019 and the company announced that it had achieved proof of concept and identified a responder population in November 2022. For the first time ever (in April 2022), the company delivered its experimental in vivo CRISPR therapy to a 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-dosing-first-pediatric-patient">
 pediatric patient
 </a>
 to treat Leber congenital amaurosis 10 (LCA10), an inherited early childhood eye disease. Its ex vivo gene editing therapy EDIT-301 holds two rare pediatric disease designations granted by the FDA for transfusion-dependent beta-thalassemia and sickle cell disease. The candidate also received 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-receives-fda-orphan-drug-designation-edit-301">
 orphan drug designation
 </a>
 in May 2022 for beta-thalassemia.
 
 
 Key customers and partnerships
 
 
 Founded in September 2013, Editas has secured several collaborations with other pharmaceutical and drug developers including 
 <a href="https://www.catalent.com/catalent-news/catalent-enters-into-strategic-partnership-with-editas-medicine-to-support-gene-editing-medicine-pipeline/">
 Catalent
 </a>
 (for therapy development and manufacturing support),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/adverum-biotechnologies-and-editas-medicine-announce">
 Adverum Biotechnologies 
 </a>
 (to develop technologies to deliver gene editing medicines to the eye), 
 <a href="https://www.askbio.com/editas-medicine-and-askbio-enter-strategic-research-collaboration-to-explore-in-vivo-delivery-of-genome-editing-medicines-to-treat-neurological-diseases/">
 Asklepios BioPharmaceutical
 </a>
 (AskBio) (to research in vivo gene editing therapies for neurological diseases),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-bluerock-therapeutics-enter-strategic">
 BlueRock Therapeutics
 </a>
 (to develop medicines by combining cell therapy and gene editing technologies), and big pharma such as Bristol-Myers Squibb (to develop cell therapies for oncology).&nbsp;
 
 
 The company expanded its partnership with Azzur Cleanrooms on Demand (COD) in July 2023 with regard to continuing to manufacture Editas' candidate EDIT-301. 
 
 
 Funding and financials
 
 
 The company last raised funds in June 2023 when the company raised USD 125 million in a common stock offering. Editas Medicine did not disclose what the raised funds would be used for. The company has been listed on the Nasdaq since February 2016 and was the first CRISPR gene editing company to go public. 
 
</div>

Editas Medicine

<div>
 
 CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline. 
 
 
 In a milestone event in November 2023, the company&rsquo;s gene-editing therapy exagamglogene autotemcel, CASGEVY 
 <a href="https://sp-edge.com/updates/24093">
 (exa-cel)
 </a>
 , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world&rsquo;s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA&rsquo;s decision expected on March 30, 2024.&nbsp;
 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend">
 Vertex Pharmaceuticals
 </a>
 (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and 
 <a href="https://viacyte.com/press-releases/crispr-therapeutics-and-viacyte-announce-strategic-collaboration-to-develop-gene-edited-stem-cell-derived-therapy-for-diabetes/">
 ViaCyte
 </a>
 (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
 
 
 Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.&nbsp;&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 
 
 Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
 
 
 
 
 
</div>

CRISPR Therapeutics

<div>
 
 Intellia Therapeutics specializes in gene editing to develop novel therapeutics for various areas, including immuno-oncology, autoimmune, and genetic diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, it was working on 3 internal drug programs and four collaborative programs. As of March 2024, one program was in the late clinical stage, one program was in the early clinical stage, and five were in the research and preclinical stage. The company considers its lipid nanoparticle delivery system to be a key strength in minimizing genotoxicity risk and random insertion.
 
 
 Intellia acquired biotechnology company Rewrite Therapeutics for USD 45 million in February 2022 to leverage its gene editing and DNA-writing technologies. Intellia would also pay up to USD 155 million in additional milestone payments.
 
 
 
 
 
 Key customers and partnerships
 
 
 Intellia has partnered with a number of biotech and pharma companies, including: 
 1) Big pharma Novartis (January 2015), to research and co-develop CRISPR/Cas9-edited therapies to develop an ex-vivo treatment for sickle-cell disease and a number of undisclosed programs 
 2) Kyverna Therapeutics (January 2022), for the development of a next-generation CD19 CAR T-cell therapy for autoimmune diseases 
 3) ONK Therapeutics (February 2022), to develop engineered natural killer (NK) cell therapies for cancer treatment.&nbsp;
 
 
 In October 2023, the company expanded its research collaboration with 
 <a href="https://sp-edge.com/updates/22377">
 Regeneron Pharmaceuticals
 </a>
 (entered in April 2020) to develop gene-editing therapies for neurological and muscular diseases.
 
 
 
 
 
 Funding and financials
 
 
 Founded by Caribou Biosciences and Atlas Ventures in November 2014, Intellia has been listed on Nasdaq since May 2016. In 
 <a href="https://sp-edge.com/updates/15140">
 November 2022
 </a>
 , the company raised USD 300 million via an underwritten public offering&mdash;it did not disclose the purpose of the financing. For 2022, the company reported a revenue of ~USD 52&nbsp; million, earned through collaborations.
 
</div>

Intellia Therapeutics

<div>
 
 Based in the US and France, Cellectis develops off-the-shelf gene editing cancer immunotherapies based on allogeneic Chimeric Antigen Receptors T (CAR-T) cells dubbed UCARTs by leveraging the transcription activator-like effector nucleases (TALEN) technology.
 
 
 As of March 2024, the company had seven therapeutics in development; two targeting Leukemia, one for the treatment of non-Hodgkin lymphoma, one for Large B Cell Lymphoma, two for Multiple Myelnoma, and one for Renal Cell Carcinoma. Its Leukemia therapeutics were in Phase I of clinical trials, while its non-Hodgkin lymphoma solution was just entering Phase I of clinical trials. The company's therapeutics for Multiple Myeloma, another for Lymphoma, and Renal Cell Carcinoma, had been licensed out to other pharmaceutical companies.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 In 
 <a href="https://sp-edge.com/updates/23389">
 November 2023
 </a>
 , Cellectis entered into a partnership with AstraZeneca to accelerate the development of gene editing therapeutics, with a focus on oncology, immunology, and rare diseases.&nbsp;
 
 
 Cellectis also partnered with Primera in January 2023 to develop in-vivo gene therapies for mitochondrial disease. Through the partnership, both entities would collaborate to genetically edit mutations in mitochondrial DNA and develop an engineering toolbox that can be used to develop therapeutics for mitochondrial diseases. 
 
 
 
 
 
 
 Funding and financials
 
 
 The company entered into several strategic collaboration agreements with AstraZeneca in November 2023. These agreements included an investment agreement relating to an initial equity investment of USD 80 million by subscribing for 16 million ordinary shares. Cellectis stated that it planned to use the proceeds received to develop gene editing tools, for research and development expenses incurred in developing its programs, and other general corporate purposes.
 
 
 
</div>

Cellectis

Caribou Biosciences specializes in gene editing to develop novel therapeutics for hematologic oncology indications using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops allogeneic off-the-shelf gene edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells and CAR Natural Killer (CAR-NK) cells.The company leverages its proprietary platform CRISPR hybrid RNA-DNA (chRDNA), which uses hybrid guides—both ribonucleic acid (RNA) and DNA nucleotides—to edit DNA. As of March 2024, Caribou had four candidates in its clinical pipeline, of which its lead candidate CB-010, a CAR-T therapy to treat relapsed/refractory B cell non-Hodgkin lymphoma, was in the Phase I clinical trial stage (March 2024). Its other CAR-T candidates, CB-011 for the treatment of relapsed/refractory Multiple Myeloma and CB-012, for the treatment of relapsed/refractory Acute Myeloid Leukemia, were also in Phase I of clinical trials. Its alternate application for CB-010 to treat autoimmune diseases was still in the discovery stage.The company intended to extend ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies toward other indications outside oncology and implement its Cas12a chRDNA platform in in vivo (gene editing done inside the body) applications.Key customers and partnershipsThe company entered into a partnership with big pharma company <a href="https://news.abbvie.com/news/press-releases/abbvie-and-caribou-biosciences-announce-collaboration-and-license-agreement-for-car-t-cell-products.htm">Abbvie </a>in February 2021 for licensing and co-development of two CAR-T therapies. Caribou also entered into licensing agreements with <a href="https://www.businesswire.com/news/home/20201118005128/en/Caribou-Biosciences-Announces-Licensing-Agreement-for-scFvs-Targeting-CD371-to-Enable-Development-of-Allogeneic-Cell-Therapies">Memorial Sloan Kettering Cancer Center </a>(MSKCC) (November 2020), Corteva Agriscience, and Intellia Therapeutics. It also has a sales and assignment agreement with ProMab Biotechnologies Inc. (February 2020).Funding and financialsCaribou Biosciences went public on the Nasdaq Global Select Market in July 2021 and raised USD 304 million. The IPO proceeds were earmarked to fuel the company’s clinical development. For 2021, it recorded revenue of USD 9.6 million earned through licensing and collaborations.The company last raised funding in July 2023, when it raised USD 125 million in an upsized underwritten public offering. Caribou did not disclose what the funds would be used for.

Caribou Biosciences

Companies that develop novel therapeutics based on other types of gene editing technologies including TALEN and ZFN

Companies that develop systems that enable the diagnosis of gene-edited therapies. Also includes systems that deliver gene-editing components into the human body

Companies that offer their gene-editing platforms to other pharma companies to develop therapeutics

Companies that offer support services to gene-editing companies, which include genomic research companies, gene-editing component manufacturing, and designing tools

Startups in the hub develop gene-editing treatments to cater to specific patients and patient profiles

Genomics enables precise changes in the DNA sequence of living organisms to develop treatments

<div>
 
 Poseida Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapeutics with the potential to cure various diseases. The company utilizes proprietary genetic engineering platform technologies, including the non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System, and nanoparticle- and AAV-based gene delivery technologies. These platforms, either alone or in combination, enable Poseida to engineer a portfolio of product candidates designed to overcome limitations of current cell and gene therapeutics.
 
 
 
 
 Poseida's pipeline includes both autologous and allogeneic CAR-T cell therapy programs targeting various indications. The company's P-PSMA-101 is an autologous CAR-T program for patients with metastatic castrate-resistant prostate cancer. In the allogeneic space, Poseida is developing P-BCMA-ALLO1, P-MUC1C-ALLO1, and P-CD19CD20-ALLO1, leveraging its proprietary technology to create off-the-shelf CAR-T therapies. The company's approach focuses on generating T-stem cell memory (Tscm) cells, which are associated with improved clinical responses and tolerability profiles.
 
 
 
 
 In gene therapy, Poseida is advancing P-OTC-101, a liver-directed program for the treatment of urea cycle disease caused by ornithine transcarbamylase (OTC) deficiency. The company's hybrid delivery approach aims to achieve durable expression at lower doses, potentially offering a more effective and tolerable treatment option. Additionally, Poseida is developing P-FVIII-101, a liver-directed gene therapy for Hemophilia A in partnership with Takeda, utilizing the piggyBac DNA Delivery System and biodegradable nanoparticle delivery.
 
 
 
 
 Poseida continues to innovate with emerging technologies such as the Site-Specific Super PiggyBac DNA Delivery system for highly specific DNA integration, and a TCR-T platform combining piggyBac and Cas-CLOVER technologies for off-the-shelf TCR-T product candidates. The company is also exploring a CAR 3.0 approach using genetically modified hematopoietic stem cells to create next-generation anti-cancer therapeutics.
 
</div>

<div>
 
 Generation Bio is a biotechnology company developing non-viral genetic medicines for rare and prevalent diseases. The company's proprietary platform combines immune-quiet DNA (iqDNA), a novel variant of closed-ended DNA, with a cell-targeted lipid nanoparticle (ctLNP) delivery system and a rapid enzymatic synthesis (RES) manufacturing process. This platform is designed to create gene therapies with drug-like properties, offering durable gene expression, the ability to re-dose for individual patient titration, and the potential to sustain effect over a lifetime. Generation Bio's approach aims to enable multi-year durability from a single dose, deliver large genetic payloads, and allow titration and redosing to adjust or extend expression levels in each patient. The company's pipeline includes programs for liver diseases such as hemophilia A, phenylketonuria (PKU), and Wilson's disease, as well as eye disorders like Stargardt disease and Leber congenital amaurosis type 10. In October 2023, Generation Bio announced the development of iqDNA, which demonstrated cytokine levels and tolerability comparable to chemically modified messenger RNA in mice and non-human primates. Generation Bio's RES manufacturing process has the potential to produce ceDNA at a scale of hundreds of millions of doses.
 
 
 
 
 Key customers and partnerships
 
 
 
 
 In March 2023, Generation Bio entered into a strategic partnership with Moderna to develop non-viral genetic medicines. The collaboration combines Moderna's expertise with Generation Bio's platform technologies to expand their respective non-viral genetic medicines pipelines. Under this partnership, Moderna is using Generation Bio's ctLNP delivery system and iqDNA technology to advance two immune cell programs and two liver programs. The deal included a USD 40 million upfront cash payment and a USD 36 million equity investment from Moderna. Generation Bio will also receive future development, regulatory, and commercial milestone payments, along with royalties on worldwide net sales of immune cell-targeted and liver-targeted products. The partnership aims to develop new lipid nanoparticles and expand in vivo immune cell targeting as a new class of genetic medicines.
 
</div>

Generation Bio

Oncopeptides AB is a biotech company focused on developing therapies for difficult-to-treat hematological diseases, particularly multiple myeloma. The company's lead product, PEPAXTO (melphalan flufenamide, also known as melflufen), received FDA approval in February 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy. PEPAXTO is the first anticancer peptide-drug conjugate approved by the FDA for this indication. The drug works by leveraging aminopeptidases, which are overexpressed in multiple myeloma cells, to release cytotoxic agents.
In August 2022, the European Commission granted marketing authorization for Pepaxti (melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies. Oncopeptides' research is based on two technology platforms: Peptide Drug Conjugates (PDC) and Small Polypeptide based Innate Killer Engagers (SPiKE). The PDC platform allows for efficient drug distribution in the body, enabling a wide therapeutic window and an optimized benefit-risk profile. The SPiKE platform utilizes the immune system to fight cancer by targeting natural killer cells, potentially reducing dose-limiting side effects while maintaining clinical efficacy.
As of 2023, Oncopeptides was conducting one clinical phase 3 study and six clinical phase 2 studies. The company's pipeline includes innovative drug candidates for difficult-to-treat hematological diseases, with the potential to expand into new indications. Oncopeptides' approach is science and data-driven, focusing on bringing innovation to patients with rare hematological diseases.

Oncopeptides

Neurona Therapeutics is a clinical-stage biotherapeutics company focused on developing regenerative cell therapy candidates for the treatment of neurological disorders. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing Phase I/II clinical trial for drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. It consists of fully-differentiated neural cells called interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, these human interneurons are intended to integrate on-target and durably silence seizure activity in the epileptic region of the brain. In December 2023, Neurona presented early data from five participants in the ongoing study of NRTX-1001. Two patients who received the therapy more than a year prior experienced at least a 95% reduction in overall seizure frequency. NRTX-1001 is manufactured in Neurona's in-house GMP facility using proprietary methods. Beyond epilepsy, Neurona is also evaluating NRTX-1001 for potential application in Alzheimer's disease. The company's pipeline includes other cell therapy candidates such as investigational myelinating glial cells and gene-edited cells for undisclosed indications.

Neurona Therapeutics

Evox Therapeutics is a biotechnology company specializing in exosome therapeutics. The company utilizes its proprietary DeliverEX technology to engineer exosomes for drug delivery to specific organs, including the brain and central nervous system. Evox's approach aims to overcome limitations of genetic medicines such as gene therapy, gene editing, and RNA therapeutics by enabling safe, non-immunogenic repeated delivery to cells and tissues that are challenging to reach with other drug delivery technologies.
The company's technology focuses on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop a new class of therapeutics for treating severe rare diseases. Evox's intellectual property portfolio includes more than 100 granted patents across key jurisdictions, covering various aspects of exosome-based drug design, development, and manufacturing. In May 2023, Evox was granted a US patent for a method of purifying engineered exosomes using ultrafiltration and size-based separation steps, which is widely used in exosome therapeutics.
In June 2023, Evox acquired Codiak Biosciences' engEx-AAV technology platform, which enables active loading of adeno-associated virus (AAV) into exosomes. This technology has demonstrated improved AAV delivery, increased expression breadth in preclinical models, and shielding of AAVs from neutralizing antibodies. The acquisition aligns with Evox's strategy to enhance exosome-mediated delivery for genetic medicines such as gene therapy and genome editors.

Evox Therapeutics

Aro Biotherapeutics is a clinical-stage biotechnology company based in Philadelphia that specializes in developing tissue-targeted genetic medicines. The company's core technology is a proprietary protein platform called Centyrins, which enables efficient and tissue-specific delivery of siRNA and other genetic drugs. Aro's lead candidate, ABX1100, is being developed for the treatment of Pompe disease, a rare genetic disorder characterized by toxic glycogen accumulation in muscles. ABX1100 comprises a CD71 receptor-binding Centyrin conjugated to a small interfering RNA (siRNA) that targets the Gys1 gene, reducing levels and activity of the Gys1 enzyme in muscle tissues. The therapy has shown promising results in preclinical studies, demonstrating potent and muscle-specific reductions in Gys1 mRNA and protein in the Pompe mouse model. In October 2023, Aro initiated a Phase 1 clinical trial of ABX1100 in Canada, with initial data expected in 2024. The US Food and Drug Administration granted ABX1100 Orphan Drug Designation and Rare Pediatric Disease status. Beyond Pompe disease, Aro is exploring the potential of its Centyrin-siRNA conjugate programs for developing new treatments for autoimmune disorders.

Aro Biotherapeutics

Therapeutics that are designed to target specific cells to reach designated target sites

Therapeutics that are designed to target specific protein or protein receptors to reach designated target sites

Therapeutics that use different targeting approaches, such as bone-targeting or pressure-based, to help therapeutics identify specific target sites

Companies that offer contract research and manufacturing facilities that focus on tissue-targeting therapeutics for life sciences organizations

Poseida Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for cancer and rare diseases. The company's technology platform includes its proprietary non-viral piggyBac DNA Delivery System, Cas-CLOVER Site-Specific Gene Editing System, and nanoparticle and hybrid gene delivery technologies. Their gene therapy pipeline includes P-KLKB1-101 for hereditary angioedema and P-FVIII-101 for hemophilia A. The company's cell therapy portfolio features allogeneic CAR-T cell therapy candidates targeting both solid and liquid tumors.&nbsp;As of November 2024, the company had 10 programs in its pipeline, comprising eight CAR-T programs and two genetic medicines, with three programs in Phase I clinical trials.Key customers and partnershipsThe company has established strategic collaborations with major pharmaceutical companies. In August 2022, Poseida partnered with Roche to develop allogeneic cell therapies for blood malignancies. In May 2024, the company entered into a research collaboration with Xyphos Biosciences, an Astellas Pharma subsidiary, to develop new allogeneic cell therapies for cancer. Additionally, Poseida has formed a partnership with Takeda.

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 Tenaya Therapeutics is a developer of therapeutics for heart disease. The company develops its therapeutics using precision medicine, gene therapy, and cellular regeneration. Tenaya produces therapeutics that also use proprietary in-vivo and in-vitro models. Additionally, the company has developed in-house adeno-associated virus (AAV) capsid engineering capabilities and has screened over one billion variants from more than 30 diverse, proprietary AAV libraries. The company is publicly listed on the Nasdaq and trades under the ticker symbol TNYA.&nbsp;&nbsp;
 
 
 In October 2023 Tenaya Therapeutics (Tenaya) received FDA clearance for the company&rsquo;s investigational new drug application (TN-401) to initiate clinical testing. TN-401 is Tenaya&rsquo;s adeno-associated virus serotype 9-based investigational gene therapy drug candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy caused by mutations in the plakophilin-2 gene. 
 
 
 As of May 2024, the company&rsquo;s clinical pipeline included two candidates in Phase I clinical trials, a gene therapeutic for the treatment of Hypertrophic Cardiomyopathy, and a small molecule inhibitor for the treatment of heart failure with preserved ejection fraction. Additionally, Tenaya had one gene therapy candidate and two genetic medicine candidates in preclinical testing phases, along with five genetic medicine candidates in the discovery stage. 
 
 
 
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Tenaya Therapeutics

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 Sarepta Therapeutics is a gene therapy provider for conditions such as Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy 
 (LGMD), and Charcot-Marie-Tooth Disease. The company&rsquo;s therapeutics incorporate technology related to gene therapy, RNA technologies, and gene editing. The company leverages a proprietary phosphorodiamidate morpholino oligomer (PMO) platform for the development of its RNA-based therapeutics. As of August 2023, the company had four FDA-approved treatments for DMD: ELEVIDYS, EXONDYS 51, VYONDYS 53, and AMONDYS 45. Sarepta Therapeutics is also listed on the Nasdaq, trading under the symbol SRPT.&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 As of May 2024, the company had several RNA, gene, and gene editing therapeutics in various clinical stages. Sarepta had two RNA therapeutics, both for the treatment of DMD, one in clinical testing, and the other in pre-clinical testing. Additionally, the company had six gene therapeutics, one for the treatment of DMD (both in clinical testing), four for the treatment of LGMD (three in clinical testing, one in preclinical testing) and one for multiple targets (in preclinical testing). Also, Sarepta had two gene editing-based therapeutics in development for the treatment of DMD, both in preclinical testing phases. 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 Over the years since its founding, Sarepta has partnered with several industry and academic partners. The list includes Dyno Therapeutics, Genethon, Genevant Sciences, Hansa Biopharma, Roche, Columbia University, Harvard University, Translational Laboratory of Murdoch University, University of Florida College of Medicine, The Abigail Wexner Research Institute at Nationwide Children&rsquo;s Hospital, University of Massachusetts Medical School, and the University of Western Australia.&nbsp;&nbsp;
 
 
 
 
 In October 2021, Sarepta and healthcare giant Roche announced they would collaborate on carrying out a clinical study of Sarepta&rsquo;s therapeutic candidate SRP-9001 for the treatment of DMD.&nbsp;
 
 
 
 
 Sarepta and pharmaceutical company Catalent announced that in January 2023, the companies were signing a commercial supply agreement. Through the agreement,&nbsp; Catalent would manufacture Sarepta&rsquo;s gene therapeutic for the treatment of DMD; delandistrogene moxeparvovec (SRP-9001).
 
 
 
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Sarepta Therapeutics

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 Mesoblast is a cell therapy manufacturer that provides therapeutics for oncology, cardiovascular, orthopedic, hematology, immunity, and inflammation-related conditions. The company is listed on the Australian Stock Exchange and trades under the ticker symbol &ldquo;MSB.&rdquo; Mesoblast develops therapeutics using its proprietary mesenchymal lineage cell technology platform, which is used to produce cryopreserved, off-the-shelf, and cellular-based solutions. The company also incorporates 3D bioreactor technology and automation into its production process to produce higher yields.&nbsp;
 
 
 
 
 As of May 2024, the company had five candidates that are in various stages of clinical testing. Mesoblast&rsquo;s steroid-refractory candidate for Acute Graft Versus Host Disease is currently in late-stage Phase III clinical testing and is the closest candidate to commercialization. The company&rsquo;s drug candidates for acute respiratory distress syndrome, chronic low back pain, and chronic heart failure and reduced ejection fraction are also in Phase III clinical testing while its candidate for inflammatory bowel disease is in Phase II of clinical testing.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured several agreements with international companies with regard to development and commercialization of Mesoblast&rsquo;s candidates, including Gr&uuml;nenthal GmBH, JCR Pharmaceuticals, Tasly Pharmaceutical Group, TiGenix NV, and the Lonza Group.&nbsp;
 
 
 
 
 Additionally, in April 2020, Mesoblast partnered with The US NIH&rsquo;s National Heart, Lung, and Blood Institute to conduct testing of its allogeneic mesenchymal stem cell (MSC) product candidate, &ldquo;remestemcel-L.&rdquo;&nbsp;&nbsp;&nbsp;&nbsp;
 
 
 
 
 
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Mesoblast

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 Ocugen is a gene therapy manufacturer that develops vaccine, cell, and gene-based therapeutics for infectious, eye, and orthopedic diseases. Its cell therapy includes a three dimensional tissue-engineered cartilage disk through the growth of chondrocytes &ndash; the cells responsible for maintaining cartilage health. Its gene therapy solution consists of creating a copy of the patient&rsquo;s gene in order to replace mutated/damaged genes. The company is publicly listed on the Nasdaq, and trades under the symbol &lsquo;OCGN&rsquo;.&nbsp;
 
 
 As of May 2024, the company had five therapeutic solutions being clinically tested. The company was developing four gene therapies for the treatment of retinitis pigmentosa, Leber congenital amaurosis, geographic atrophy, and orphan disease. Its treatment candidate for retinitis pigmentosa was in Phase III of clinical study, while its other gene therapies were in Phase I/II. 
 Ocugen was also developing a biological material, in the IND stage, for diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. The company's cell therapy for the treatment of articular cartilage defects in the knee was in Phase III of clinical study. Finally, the company was also developing three vaccines, all in IND stages, for prevention of diseases caused by the flu, Covid-19, and a combination of the flu and Covid-19.
 
 
 Key customers and partnerships
 
 
 Ocugen partnered with biopharmaceutical manufacturer Jubilant HollisterStier of Spokane in June 2021 to manufacture its Covid-19 therapeutic COVAXIN. Through the partnership, Jubilant would be the potential commercial manufacturer of Ocugen&rsquo;s vaccine for the US and Canadian markets.
 
 
 
 
 
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Ocugen

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 MeiraGTx is a developer of gene therapies that aim to treat ocular diseases and problems, neurodegenerative disorders, and salivary gland and autoimmune disorders. MeriaGTx is also developing riboswitch-inducible expression programs for the treatment of inherited obesity and Wilson disease, along with producing small-molecule riboswitch inducers. The company is listed on the Nasdaq and trades under the symbol MGTX.&nbsp;&nbsp;
 
 
 As of May 2024, the company had four gene-based therapeutic candidates in Phase II of their clinical trials for the treatment of (1) xerostomia, Parkinson's Disease, and RPE65-Associated retinal dystrophy and (2) achromatopsia. The company also had one therapeutic being developed to treat Sjogren&rsquo;s Syndrome that was in Phase I/II of its clinical trials. Additionally, MeiraGTx had seven other therapeutics in discovery/preclinical stages. The company was also developing a therapeutic for X-linked retinitis pigmentosa in collaboration with Janssen, which was in Phase III of clinical trials. 
 
 
 Key customers and partnerships
 
 
 MeiraGTx partnered with the International Institute of Risk and Safety Management (IIRSM) in November 2022 to influence risk agendas, raise awareness, the standards of risk, and safety management across the industry.&nbsp;
 
 
 Funding and financials
 
 
 Meira last raised funds in October 2023, when the company secured a strategic investment of USD 30 million from Sanofi. As part of the transaction, Sanofi was granted the right of first negotiation for MeiraGTx's gene regulation technology in certain targets.&nbsp;
 
 
 
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MeiraGTx

Companies producing cells, genes, tissues, and biomaterials such as exosomes and extracellular matrix for drug developers and pharmaceutical companies developing regenerative medicine

Companies conducting R&D on therapies that modify or introduce new genes into the body to treat diseases

Includes therapies that use cells (other than CAR-T and stem cells) such as Adipose-Derived Regenerative Cells (ADRCs), cardiosphere-derived cells, and gamma delta T cells (GDT cells)

Stem cell therapy involves using stem cells usually extracted from the bone marrow and umbilical cord blood to repair a variety of diseases

Startups in the hub consider individual patient profiles, involving variability in genes, environment, and lifestyle

Genomics helps understand the genetic information stored in the DNA to develop targeted treatments

INCUMBENTS_PRESENT

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 Swiss healthcare company Roche will be acquiring gene editing firm Poseida Therapeutics for $1.5 billion. This purchase builds upon a collaboration between the two companies, which began in August 2022. 
 With this acquisition, Roche aims to further enhance its own allogeneic cell therapy research by integrating Poseida's expertise. The company also anticipates benefiting from Poseida’s production capacities and research and development resources, including a CAR T program and various preliminary genomic medicine candidates. Ultimately, this transaction is projected to bolster Roche's ongoing endeavors in creating innovative therapies in the areas of oncology, immunology, and neurology. The acquisition is set to be finalized in the first quarter of 2025.
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Poseida Therapeutics

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