ATP, a global leader in life sciences venture capital, is developing the next generation of transformative biotechnology companies. ATP creates science-based companies starting with assets at various stages: from working with scientists on pre-IP ideas, to spinouts from existing company pipelines. The firm provides flexible capital, strategic insight, and operational resources to build sustainable, research-driven enterprises. ATP invests in its portfolio companies from seed stage through IPO and beyond and supports their management teams in strategic transactions and synergistic business combinations.

Apple Tree Partners

Ascidian Therapeutics

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

Ascidian Therapeutics is a biotechnology company focused on treating human diseases by rewriting RNA. Founded in 2020 and headquartered in Boston, the company has developed a proprietary RNA exon editing platform that enables precise post-transcriptional editing of genes. Ascidian's approach allows for the replacement of mutated exons with wild-type exons in a single reaction, without modifying DNA or requiring the introduction of exogenous enzymes. This technology is designed to address large genes and genes with high mutational variance while maintaining native gene expression patterns and levels.

Ascidian Therapeutics Announces Three Key Development Team Appointments as It Advances Its Lead Program Toward the Clinic

Ascidian Therapeutics Launches to Rewrite RNA

Ascidian Therapeutics to Present Preclinical Data for RNA Exon Editor, ACDN-01, Supporting an Open IND for Phase 1/2 Clinical Testing at ASGCT 2024 Annual Meeting

Ascidian Therapeutics Launches With RNA Exon Editing Platform

Ascidian Therapeutics launches with $50M to rewrite RNA

Ascidian starts up with $50M and a twist on RNA editing

Roche partners with RNA editing biotech Ascidian

Boston startup Ascidian Therapeutics charts vision for therapies that ‘rewrite RNA’

Ascidian Nets $50M to Rewrite RNA in Stargardt Disease and Beyond

FDA Clears Ascidian for First-Ever RNA Editing Trial in US

RNA Therapeutics

Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing Therapeutics Targeting Neurological Diseases

Roche

To give Roche access to Ascidian's exon-editing platform to develop drugs for undisclosed neurological targets.

Reinventing gene therapies with next-generation technologies

Human Gene Editing

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

Ascidian Therapeutics is a biotechnology company focused on treating human diseases by rewriting RNA. Founded in 2020 and headquartered in Boston, the company has developed a proprietary RNA exon editing platform that enables precise post-transcriptional editing of genes. Ascidian's approach allows for the replacement of mutated exons with wild-type exons in a single reaction, without modifying DNA or requiring the introduction of exogenous enzymes. This technology is designed to address large genes and genes with high mutational variance while maintaining native gene expression patterns and levels.
The company's lead program, ACDN-01, targets ABCA4 retinopathies, including Stargardt disease, the most common form of inherited macular degeneration affecting approximately 30,000 individuals in the US. In January 2024, Ascidian received IND clearance from the FDA and Fast Track Designation for ACDN-01, becoming the first RNA editing therapeutic to be cleared for clinical investigation in the US. The company plans to initiate a Phase I/II open-label study, called STELLAR, in the first half of 2024 to evaluate the safety and efficacy of a single dose of ACDN-01 administered via subretinal injection.
Ascidian's technology platform combines high-throughput molecular biology with computational biology to design novel RNA exon editor molecules. The company has demonstrated successful RNA exon editing across multiple genes and is advancing programs in ophthalmology, neurological and neuromuscular disorders, and rare diseases. In April 2024, Ascidian was granted Rare Pediatric Disease Designation for ACDN-01 by the FDA.

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 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.
 
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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.

INCUMBENTS_PRESENT

PROMINENT_CATEGORY

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 Ascidian Therapeutics, a company specialising in RNA-based therapeutic development, has entered a partnership with global healthcare organization Roche. The deal gives Roche exclusive access to Ascidian's exon-editing platform, intended to be used for the development of undisclosed neurological drugs.
 As part of the contract, Roche and Ascidian will engage in a collaborative effort to develop the latter's RNA exon-editing technology. Roche is set to continue specific preclinical studies, manufacturing, and further clinical development, while also overseeing the commercialisation of the drugs produced. As compensation, Ascidian Therapeutics will receive an upfront payment of USD 42 million, potential milestone payments amounting up to USD 1.8 billion, alongside royalties on sales.
 Ascidian Therapeutics operates with a focus on harnessing its proprietary RNA exon-editing technology to target and treat genetic diseases. The company aims to do this by accurately editing and replacing faulty exons in RNA. Its leading product, ACDN-01, is the first RNA exon editor to be used in clinical trials. The product targets Stargardt disease, a genetic eye disorder. The primary objective of Ascidian's technology is to provide the benefits of gene therapy, while mitigating risks associated with direct DNA editing, thus ensuring the preservation of normal protein functions.​
 
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Ascidian Therapeutics

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