ARCH invests primarily in companies co-founded by scientists and entrepreneurs, concentrating on bringing to market innovations in life sciences. They enjoy special recognition as a leader in the successful commercialization of technologies developed at academic research institutions and national laboratories. ARCH has raised ten venture funds totaling over $3 billion and has invested in the venture capital rounds for more than 150 companies. ARCH investors include corporations, pension funds, endowment funds, financial institutions, and private investors.

ARCH Venture Partners

Fast Track Initiative is a venture capital firm with an exclusive focus on life sciences and healthcare services.

Fast Track Initiative, Inc.

Codon Capital is a venture capital firm that brings entrepreneurs and researchers to discover strategies for translating innovation into products and startup companies. It focuses on two biology disciplines, Therapeutics and Synthetic Biology, each with unique investment benefits. Together, they represent the new biology design revolution, opportunities that will define healthcare, and agriculture. Founded in 2013 by Karl Handelsman, Codon Capital is headquartered in San Francisco, California.

Codon Capital

+ND Capital (NanoDimension) is a venture capital firm founded in 2002 that invests in disruptive technologies across the life, physical, and data sciences.

+ND Capital

Novalis

Forbion Capital Partners is a Netherlands-based venture capital firm focused on investing in life sciences companies in drug development as well as MedTech companies addressing high medical needs. Forbion's investment team of nine investment professionals has built an impressive performance track record since the late nineties with successful investments in Rhein Biotech, Crucell, Neutec, Glycart, Borean, Impella, Alantos, Acorda, Fovea and PanGenetics. Current assets under management exceed â‚¬400m ($500m), split between three active funds. Finally, Forbion co-manages BioGeneration Ventures, an early stage fund focused on academic spin-outs and seed investments in the Netherlands.

Forbion Capital Partners

Alexandria Venture Investments is a venture capital firm that develops work in real estate technology, and healthcare sectors. The company's strategic venture capital platform is Alexandria Venture Investments. Since its inception in 1996, the company has invested in disruptive life science, agrifood tech, and technology companies that are developing transformative new modalities and platforms to meaningfully improve human health.

Alexandria Venture Investments

Ono Venture Investment is the corporate venture capital arm of Ono Pharmaceutical Co.,Ltd. , an R&D-oriented pharmaceutical company succeeded in delivery of “First in class” medicines to patients such as OPDIVO® (nivolumab).

Ono Venture Investment

AIRNA

AIRNA is a biotech company that is pioneering RNA editing therapies to improve the health of individuals suffering from both rare and common ailments.

<div>AIRNA Therapeutics focuses on RNA-editing therapeutics to restore the health of patients with rare and common diseases. AIRNA&rsquo;s pipeline is driven by its RNA editing platform, RESTORE+, which optimizes therapeutic sequence, chemistry, and delivery for the RNA-editing pipeline. The company is advancing the development of product candidates to treat the inherited genetic disease, alpha-1 antitrypsin deficiency (AATD). ADAR-directed RNA editing delivers a safe oligonucleotide which is programmed to recruit an endogenous cellular enzyme, ADAR, to introduce a precise modification to a patient&rsquo;s RNA, resulting in changes to the encoded therapeutic proteins.&nbsp;</div>

Reinventing gene therapies with next-generation technologies

Human Gene Editing

AIRNA is a biotechnology company focused on developing RNA editing therapeutics for both rare and common diseases. Founded in 2023, the company utilizes its proprietary RESTORE+ platform to generate a pipeline of RNA editing products. AIRNA's technology is based on ADAR-directed RNA editing, which uses oligonucleotides to recruit an endogenous cellular enzyme called ADAR to make precise modifications to a patient's RNA, resulting in changes to encoded therapeutic proteins. This approach aims to provide a safer alternative to traditional gene editing techniques by modifying RNA without altering the original DNA code.

AIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases

Sept. 19 Quick Takes: RNA editing company Airna emerges with $30M round led by Arch

FTI Supports AIRNA’s $30M Launch to Develop RNA Editing Therapeutics

AIRNA: $30 Million Secured To Create New Class Of RNA Editing Therapeutics

5 RNA editing companies you should know about

Startup Airna emerges from stealth to bring RNA editing to a rare disease

RNA Therapeutics

AIRNA: RNA Editing Therapeutics Company Raises $60 Million

<div>
 
 AIRNA Therapeutics focuses on RNA-editing therapeutics to restore the health of patients with rare and common diseases. AIRNA&rsquo;s pipeline is driven by its RNA editing platform, RESTORE+, which optimizes therapeutic sequence, chemistry, and delivery for the RNA-editing pipeline. The company is advancing the development of product candidates to treat the inherited genetic disease, alpha-1 antitrypsin deficiency (AATD). ADAR-directed RNA editing delivers a safe oligonucleotide which is programmed to recruit an endogenous cellular enzyme, ADAR, to introduce a precise modification to a patient&rsquo;s RNA, resulting in changes to the encoded therapeutic proteins.&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 In 
 <a href="https://sp-edge.com/updates/22023">
 September 2023
 </a>
 , the company raised USD 30 million in funding in a round led by ARCH Venture Partners. The funds were used to advance its pipeline of RNA-editing therapeutics using its RESTORE+ platform to treat rare and common diseases. 
 
</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

AIRNA is a biotechnology company focused on developing RNA editing therapeutics for both rare and common diseases. Founded in 2023, the company utilizes its proprietary RESTORE+ platform to generate a pipeline of RNA editing products. AIRNA's technology is based on ADAR-directed RNA editing, which uses oligonucleotides to recruit an endogenous cellular enzyme called ADAR to make precise modifications to a patient's RNA, resulting in changes to encoded therapeutic proteins. This approach aims to provide a safer alternative to traditional gene editing techniques by modifying RNA without altering the original DNA code.
The company's lead program is focused on developing a treatment for alpha-1 antitrypsin deficiency (AATD), an inherited genetic disorder. AIRNA is also pursuing additional programs to address other prevalent diseases with high unmet medical needs. The RESTORE+ platform optimizes each therapeutic's sequence, chemistry, and delivery for precise, efficient, and safe RNA editing. AIRNA's founders, including Thorsten Stafforst from the University of Tübingen and Jin Billy Li from Stanford University, were pioneers in the field of RNA editing, publishing the first peer-reviewed papers on ADAR-mediated RNA editing and the use of oligonucleotides for targeted RNA editing.

<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.

<div>
 
 AIRNA, a company specialising in RNA-editing therapeutics, has successfully secured USD 60 million in an oversubscribed Series A funding round. The round was spearheaded by Forbion and saw participation from new investors Ono Venture Investment and Alexandria Venture Investments along with existing investors ARCH Venture Partners and ND Capital. With this funding, AIRNA's total Series A financing has reached USD 90 million.
 The funds raised will be utilized to drive AIRNA's alpha-1 antitrypsin deficiency (AATD) candidate into clinical trials, as well as to strengthen its development pipeline. The company's goal is to have its first drug reach the clinical stage by 2025.
 
</div>

AIRNA

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