The Alaska Permanent Fund is a constitutionally established permanent fund managed by a state-owned corporation, the Alaska Permanent Fund Corporation. The fund was established in Alaska in 1976 by Article 9, Section 15 of the Alaska State Constitution under Governor Jay Hammond. From February 1976 until April 1980, the Department of Revenue Treasury Division managed the state's Permanent Fund assets, until, in 1980, the Alaska State Legislature created the APFC.  Shortly after the oil from Alaska’s North Slope began flowing to market through the Trans-Alaska Pipeline System, the Permanent Fund was created. It was designed to be an investment where at least 25% of the oil money would be put into a dedicated fund for future generations, who would no longer have oil as a resource. This does not mean the fund is solely funded by oil revenue. The Alaska Permanent Fund sets aside a certain share of oil revenues to continue benefiting current and all future generations of Alaskans.

Alaska Permanent Fund

SoftBank Vision Fund is a subsidiary of Softbank Group that specializes in growth capital and social impact investments. Sectors that the fund invests in include the internet of things, artificial intelligence (AI), robotics, communications infrastructure, telecoms, computational biology, biotech, cloud technologies and software, consumer internet businesses, financial technology, and mobile apps. The Fund is designed to be a catalyst for technology progress in anticipation that it will expand SoftBank's capabilities, accelerating progress towards SoftBank 2.0. It aims to invest in businesses and foundational platforms that SoftBank believes to revolutionize and innovate the world tomorrow.

SoftBank Vision Fund

Flagship Pioneering conceives, creates, resources, and grows first-in-category ventures to transform human health and sustainability. The company accelerates Flagship Pioneering’s advances in biology, engineering, medicine, energy, and beyond. The firm harnesses science and entrepreneurialism to envision alternative futures, beginning with seemingly unreasonable propositions and navigating to transformational outcomes through an evolutionary methodology. Flagship Pioneering combines innovative capacity, entrepreneurial leadership, and professional capital management in a single enterprise that systematically produces first-in-category companies.

Flagship Pioneering

The QIA was founded by the State of Qatar in 2005 to strengthen the country's economy by diversifying into new asset classes. Building on the heritage of Qatar investments dating back more than three decades, its growing portfolio of long-term strategic investments helps complement the state's wealth in natural resources. Qatar's goal is to become an international center for finance and investment management, a vision shared by its government, people, and institutions. It has a track record of investing in different asset classes, including listed securities, property, alternative assets, and private equity in all the capital markets as well as the newer emerging markets.

Qatar Investment Authority

Philosophy is to focus on life science markets with strong demand and growth potential, and then back the highest-quality entrepreneurs in those markets to build defensible, strong, and long-term businesses.  build portfolio among attractive life science sectors in a manner that balances risk while maximizing potential synergies and ultimate returns. Our focus is on opportunities in biotechnology, medical devices, and diagnostics, but we are continually responsive to the best areas for investing in order to adapt to changing market conditions.  Invest in companies that:      * are pursuing substantial and growing market opportunities     * possess a unique and proprietary technology, with a clear competitive advantage     * are early-stage opportunities     * have or will attract strong management     * have a strong investment syndicate     * offer a defined path to liquidity

Altitude Life Science Ventures

Longevity Vision Fund is a $100M investment fund dedicated to making longevity affordable and accessible to all. The fund accelerates longevity breakthroughs by investing in companies that develop technologies, products, and services that extend healthy human lifespans and overcome the negative effects of aging Longevity Vision Fund provides Seed to Series C funding to biotech and life extension-focused companies that have demonstrated affordable longevity potential and vision of milestones driving up the value of the company and market validation.

Longevity Vision Fund

We are a specialized investment fund in biotechnology that also functions as a company builder. Our mission is to create and support innovative companies, guiding them from inception to commercial launch.  As a dynamic company builder, we provide support to emerging companies in agrowth-focused and innovative environment. We deliver comprehensive services in finance, accounting, legal, compliance, and recruitment to help startups thrive and succeed.

Saya Bio

T. Rowe Price is an investment management firm dedicated to funding technology startups. The firm serves individuals, financial intermediaries, and institutions and offers a full range of investment strategies across a broad spectrum: debt, equities, and private equities.

T. Rowe Price

Abu Dhabi Investment Authority is an investment management agency investing funds on behalf of the Government of Abu Dhabi. ADIA does not directly to manage or take an operational role in the companies in which it invests but through it's PPP Representative Eiosoft Capital Group LLC.

Abu Dhabi Investment Authority

ARTIS Ventures is a financial services firm that finances early-to-late-stage venture investments. The team supports its portfolio companies through their entire life cycle, from initial venture investment to public offering and beyond.

Artis Ventures (AV)

March Capital is a venture growth firm headquartered in Santa Monica, California and investing globally since 2014. March identifies entrepreneurs with a provocative vision to companies poised, then dares to go all in by leading rounds with deep conviction and concentration risk. With $1B+ in capital over 4 funds and across 30 market technology companies, March accelerates the digital transformation of Cloud-based software, automation, AI, and IT infrastructure. Its vision is to create a technology investment platform by combining intense sector focus, patience, access to a network (including founding The Montgomery Summit), and portfolio engagement to inspire and accelerate extraordinary companies.

March Capital

Cormorant Asset Management is an employee-owned hedge fund sponsor that provides its services to pooled investment vehicles. The firm also focuses on both public and private market innovative companies in the biotechnology and life sciences marketplace.

Cormorant Asset Management

SALT Fund

Hanwha Impact Partners is an investment firm that invest in ecofriendly energy and businesses that leverage the convergence of technologies.

Hanwha Impact Partners

Tessera Therapeutics

Tessera Therapeutics is an early-stage life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability.

<div>Tessera Therapeutics develops novel therapeutics, leveraging its proprietary ribonucleic acid (RNA)-based gene writing technologies to address disease indications across neurology, oncology, and auto-immunology. The company&rsquo;s approach revolves around reducing or eliminating the dependence on nucleases in gene editing. It creates synthetic mobile genetic elements (MGEs) called retrotransposons that are capable of copying an RNA sequence into the DNA, without breaking the DNA, with precision. It uses machine learning, generative protein design, and protein engineering techniques to identify, engineer, and optimize elements.&nbsp;</div>

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Tessera Therapeutics is a biotechnology company pioneering Gene Writing, a novel approach in genetic medicine. Founded in 2018 by Flagship Pioneering, Tessera's Gene Writing technology is designed to write therapeutic messages into the genome to treat diseases at their source. The company's platform can change any base pair to any other, make small insertions or deletions, and write entire genes into the genome using only RNA delivery. This technology aims to overcome limitations of current gene therapy and gene editing approaches.

Tessera Therapeutics, Inc.

Gene Writing Technology Company Tessera Therapeutics Emerges from Stealth

Tessera Therapeutics Announces Over $300M Series C Financing to Advance its GENE WRITING Platform

Tessera Therapeutics Announces Over $300M…

Tessera Therapeutics Presents New Data Demonstrating the Potential of Gene Writing™ Technology at the American Society of Gene and Cell Therapy 26th Annual Meeting

A Flagship Pioneering Company

Flagship Pioneering’s Scientists Invent a New…

RNA Therapeutics

Tessera Therapeutics to Present New Preclinical Data From In Vivo Programs for Sickle Cell Disease and T-Cell Therapies at the 66th American Society of Hematology Annual Meeting

<div>
 
 Tessera Therapeutics develops novel therapeutics, leveraging its proprietary ribonucleic acid (RNA)-based gene writing technologies to address disease indications across neurology, oncology, and auto-immunology. The company&rsquo;s approach revolves around reducing or eliminating the dependence on nucleases in gene editing. It creates synthetic mobile genetic elements (MGEs) called retrotransposons that are capable of copying an RNA sequence into the DNA, without breaking the DNA, with precision. It uses machine learning, generative protein design, and protein engineering techniques to identify, engineer, and optimize elements.&nbsp;
 
 
 The company has not revealed its therapeutic pipeline. It announced plans to set up a good manufacturing practices (GMP) standard manufacturing facility by the end of 2022 to produce RNA writers&mdash;that have the potential for redosing&mdash;on a large scale.&nbsp;
 
 
 Key customers and partnerships
 
 
 The company partnered with 
 <a href="https://www.businesswire.com/news/home/20220228005181/en/Serotiny-and-Tessera-Therapeutics-enter-into-collaboration-to-engineer-and-optimize-programmable-Gene-Writer-proteins#:~:text=By%20partnering%20with%20Serotiny%2C%20Tessera,a%20variety%20of%20disease%20states.">
 Serotiny
 </a>
 in February 2022 to engineer programmable gene writer proteins leveraging Serotiny&rsquo;s platform. It also entered into a collaboration with 
 <a href="https://www.businesswire.com/news/home/20211103005184/en/Tessera-Therapeutics-Announces-RD-Collaboration-with-Cystic-Fibrosis-Foundation-as-Part-of-a-Portfolio-of-Complementary-Technologies-Advanced-by-Flagship%E2%80%99s-Pioneering-Medicines-to-Treat-Cystic-Fibrosis">
 Cystic Fibrosis Foundation
 </a>
 in November 2021 to apply its gene writing technology in treating cystic fibrosis.&nbsp;
 
 
 &nbsp;
 Funding and financials
 
 
 In April 2022, Tessera Therapeutics raised over USD 300 million in a 
 <a href="https://www.businesswire.com/news/home/20220419005210/en/Tessera-Therapeutics-Announces-Over-300M-Series-C-Financing-to-Advance-its-GENE-WRITING-Platform">
 Series C
 </a>
 funding round involving a wholly-owned subsidiary of the Abu Dhabi Investment Authority (ADIA), Alaska Permanent Fund Corporation, and Flagship Pioneering (founder of Tessera), among others. The proceeds were earmarked for accelerating the development of its gene writing platforms and clinical development pipeline.&nbsp; 
 
</div>

<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 the CRISPR/Cas9 gene-editing technology

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

Tessera Therapeutics is a biotechnology company pioneering Gene Writing, a novel approach in genetic medicine. Founded in 2018 by Flagship Pioneering, Tessera's Gene Writing technology is designed to write therapeutic messages into the genome to treat diseases at their source. The company's platform can change any base pair to any other, make small insertions or deletions, and write entire genes into the genome using only RNA delivery. This technology aims to overcome limitations of current gene therapy and gene editing approaches.Tessera's Gene Writers are based on mobile genetic elements (MGEs), which are naturally occurring genome architects representing approximately half of the human genome. The company has evaluated thousands of natural and synthetic MGEs to create Gene Writer candidates capable of writing therapeutic messages into the human genome. Tessera's research engine further optimizes these candidates for efficiency, specificity, and fidelity.The company's technology platforms include RNA Gene Writing and DNA Gene Writing. RNA Gene Writing uses an RNA template and Gene Writer protein to either write a new gene into the genome or guide the rewriting of a pre-existing genomic sequence. DNA Gene Writing uses a DNA template to write a new gene into the genome. These approaches offer the potential for a new category of genetic medicines with broad applications both in vivo and ex vivo.

<div>
 
 PYC Therapeutics is a clinical-stage biotechnology company focused on developing RNA therapies for genetic diseases. The company utilizes a proprietary drug delivery platform based on Cell-Penetrating Peptides (CPPs) to enhance the potency of precision medicines within the RNA therapeutic class. PYC's technology aims to overcome the delivery challenges associated with traditional RNA therapeutics, allowing for more effective treatment of genetic ailments.
 
 
 
 
 PYC's lead drug candidate, VP-001, is designed to treat Retinitis Pigmentosa type 11 (RP11), a blinding eye disease that begins in childhood. VP-001 targets the U4/U6 small nuclear ribonucleoprotein (PRPF31) gene and is administered through intravitreal injection. In August 2023, the US Food and Drug Administration (FDA) granted Fast Track designation to VP-001, potentially expediting its development and review process. In May 2024, PYC reported encouraging first clinical data for VP-001 in RP11 patients.
 
 
 
 
 The company's pipeline includes other preclinical programs for Inherited Retinal Diseases and Central Nervous System disorders. In May 2024, PYC received orphan drug designation from the FDA for PYC-001, a drug candidate targeting autosomal dominant optic atrophy (ADOA). PYC is also developing VP-002 and exploring multiple candidates for eye and CNS indications.
 
 
 
 
 In January 2024, PYC announced plans to collaborate with Google Cloud to develop new precision medicines. This project aims to utilize machine learning models, merging AlphaFold and its successors, to predict ideal peptide sequences and structures for delivering precision medicine cargoes to cells in the human body.
 
 
 
 
 For the half-year ended December 31, 2023, PYC reported revenue of AUD 9.12 million, up from AUD 3.49 million in the previous year. The company's net loss for the period was AUD 15.09 million. As of March 31, 2024, PYC had a pro-forma cash balance of approximately AUD 84 million, providing runway into the second half of 2025 for its ongoing clinical trials.
 
</div>

PYC Therapeutics

Flamingo Therapeutics is a biotechnology company specializing in RNA-targeted therapies for oncology. Founded in 2019, the company focuses on developing treatments for difficult-to-treat cancers using state-of-the-art chemistries and a clinical-stage pipeline targeting undruggable transcription factors and splice variants. Flamingo's lead program is danvatirsen, an antisense oligonucleotide (ASO) that selectively targets STAT3 and has shown clinical activity in two Phase II studies. Danvatirsen binds to STAT3 mRNA, inhibiting translation of the transcript, resulting in reduced tumor cell growth and activation of anti-tumor immunity. Flamingo initiated the PEMDA-HN Phase II trial evaluating danvatirsen in combination with pembrolizumab for patients with recurrent/metastatic head and neck squamous cell carcinoma (HNSCC). The company is also advancing FTX-001, its most advanced long non-coding RNA (LncRNA) program targeting MALAT-1, through readiness for Phase 1 trials in solid tumors. Flamingo's proprietary discovery engine, FLAME (Flamingo LncRNA Antisense Mining Engine), addresses lncRNAs, a large and untapped class of disease-causing targets within the "dark matter" of the human genome. In March 2023, Flamingo merged with Dynacure to create a leading RNA therapeutics company focused on clinical oncology, combining their pipelines and expertise.
Key customers and partnerships
Flamingo Therapeutics has established strategic alliances and partnerships to advance its RNA-targeted therapies. The company has an expanded alliance with Ionis Pharmaceuticals, a world leader in RNA-targeted antisense oligonucleotide (ASO) therapeutics. This partnership, announced in September 2021, combines three clinical-stage programs from Ionis with Flamingo's novel lncRNA discovery engine and preclinical lncRNA program. The alliance leverages Flamingo's expertise in oncology and long non-coding RNAs with Ionis' proficiency in RNA-targeted drug discovery and development. Additionally, Flamingo collaborates with academic partners at VIB, KU Leuven, Ghent University, and the University of Michigan, whose pioneering work in non-coding RNA genes in cancer contributed to the company's foundation. In June 2023, Flamingo received a EUR 1.7 million research grant from Flanders Innovation & Entrepreneurship (VLAIO) to advance its RNA-targeting oncology portfolio, further validating its approach in creating a pipeline of programs targeting undruggable transcription factors and non-coding RNA.

Flamingo Therapeutics

Korro Bio is a biotechnology company focused on developing RNA editing therapeutics for both rare and common diseases. Founded in 2018, the company is headquartered in Cambridge, Massachusetts. Korro's proprietary platform, called OPERA (Oligonucleotide Promoted Editing of RNA), leverages the body's natural ADAR (adenosine deaminase acting on RNA) system to make precise edits to RNA transcripts. This approach allows for the correction of genetic mutations without permanently altering DNA, potentially offering a safer alternative to traditional gene editing techniques.
The company's lead program targets alpha-1 antitrypsin deficiency (AATD), a genetic disorder affecting the lungs and liver. Korro's AATD candidate aims to repair inherited mutations and restore production of normal functional alpha-1 antitrypsin (A1AT) protein in the liver. preclinical studies have demonstrated an increase of normal A1AT protein to 85% of total protein in circulation. Korro plans to submit a regulatory filing to initiate clinical trials for this candidate in the second half of 2024.
Beyond AATD, Korro is developing RNA editing therapies for conditions such as Parkinson's disease, severe alcoholic hepatitis, amyotrophic lateral sclerosis (ALS), subsets of pain, and cardiometabolic diseases. The company's initial focus is on liver-targeted therapies, with plans to expand into central nervous system indications and other tissue types.
In July 2023, Korro Bio announced plans to go public through a reverse merger with Frequency Therapeutics. The combined company will operate under the Korro Bio name and trade on the Nasdaq stock exchange under the ticker symbol KRRO. This transaction, along with a concurrent USD 117 million financing, is expected to provide Korro with approximately USD 170 million in cash upon closing, which is anticipated to fund operations into 2026.

Korro Bio

Halo-Bio is a biopharmaceutical company focused on developing innovative therapies. The company has a licensing agreement with Tekmira Pharmaceuticals related to certain lipid nanoparticle (LNP) technologies.

Halo-Bio RNAi Therapeutics

Oncotelic Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing RNA therapeutics and small molecule drugs for cancer and infectious diseases. The company's lead drug candidate is OT-101, a first-in-class RNA therapeutic designed to target TGF-β2, a protein that suppresses immune cell response and contributes to tumor growth in cancer tissues. OT-101 has demonstrated notable single-agent activity in clinical trials for relapsed/refractory cancer patients, with overall survival increasing in a dose-dependent manner at levels of 140mg/m2 and higher.
Oncotelic is currently conducting a Phase 2 billion/Phase 3 clinical trial called STOP-PC to evaluate OT-101 in combination with mFOLFIRINOX compared to mFOLFIRINOX alone for patients with advanced and unresectable or metastatic pancreatic cancer. The company believes this trial could deliver a decisive win against pancreatic cancer based on previous data showing improved survival in patients with low TGF-β2 expression treated with irinotecan.
In addition to its oncology pipeline, Oncotelic has explored OT-101's potential against SARS-CoV-2, completing a Phase 2 trial for Covid-19 in November 2021 with positive top line data for safety and efficacy. The company has also acquired AL-101 for intranasal delivery of apomorphine, which it intends to develop for conditions including Parkinson's Disease, erectile dysfunction, and female sexual disorders.

Oncotelic

Companies developing treatments using molecules like antisense oligonucleotides (ASOs) and phosphorodiamidate morpholino oligomers (PMOs). These molecules target specific genes to treat genetic diseases by either silencing or modifying gene expression.

Companies using RNA interference (RNAi) to target and silence specific genes involved in diseases for treating various conditions by blocking harmful gene activity.

Companies involved in altering RNA molecules to modify gene expression or correct genetic defects. This can include substitutions, insertions, or deletions of nucleotides, leading to alterations in the RNA sequence and the regulation of RNA molecules to control gene expression or cellular functions.

Companies focused on other RNA-based therapies using approaches such as tRNA, circRNA, lncRNA, and saRNA to develop treatments targeting specific RNA molecules to regulate gene expression.

RNA-based developments form a foundation for precision medicine.

RNA plays essential roles in gene expression, regulation, and protein synthesis, making it a fundamental focus in genomic research and understanding genetic mechanisms.

Tessera Therapeutics

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