AAscidian Therapeutics focuses on RNA exon editing to treat diseases. It develops technologies that edit RNA transcripts after they are made from DNA, enabling precise changes while keeping natural gene expression patterns. This approach aims to produce full-length, functional proteins and offer durable therapeutic effects without direct DNA editing or gene replacement. The company monetizes by licensing its RNA exon editing technology to healthcare providers and research institutions and may also participate in development and sale of therapies based on its platform. Compared with competitors, it emphasizes transcriptome re-engineering in human cells to achieve steady gene expression without altering the genome, reducing certain genetic risks while maintaining normal regulatory control. The overall goal is to translate RNA exon editing into accessible therapies for patients by partnering for licensing and therapy development.
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2020
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Ascidian bets on Exegenesis to optimize AAV delivery for RNA Exon Editors. Ascidian and Exegenesis partner to combine RNA exon editing with AI-engineered AAV capsids, advancing targeted delivery for genetic medicines. Ascidian Therapeutics and Exegenesis Bio have entered a strategic agreement to combine RNA exon editing with AI-enabled adeno-associated virus (AAV) capsid engineering, advancing targeted delivery strategies for genetic medicines. The collaboration will evaluate program-specific combinations of RNA exon editors and next-generation AAV capsids, with ongoing scientific work focused on generating in vivo data and supporting progression toward clinical development. Addressing the delivery challenge in genetic medicines. Efficient and tolerable delivery remains a major obstacle in genetic medicine development. Although therapeutic payloads may demonstrate biological potential, their clinical utility depends on reaching the appropriate cells and tissues at sufficient levels without exposing non-target tissues to unnecessary treatment. AAV vectors offer an established approach to delivering genetic material into target cells. However, conventional capsids often face limitations in tissue specificity and delivery efficiency. Engineered AAV capsids can potentially improve tissue targeting, reduce off-target exposure outside the intended tissues, and support therapeutic delivery at lower, more tolerable doses. For Boston-based Ascidian Therapeutics, a clinical-stage biotechnology company pioneering RNA exon editing (including its lead clinical candidate ACDN-01 for Stargardt disease), solving this delivery challenge is essential because its platform targets diseases caused by genetic abnormalities across multiple tissue types. The company conducted a global search for AAV capsid engineering partners before selecting Exegenesis Bio, a clinical-stage gene therapy developer with operations in the United States and China, to evaluate delivery technologies compatible with its therapeutic platform. Combining RNA Exon editing with ai-enabled capsid discovery. Ascidian's platform uses post-transcriptional RNA editing to replace large mutated exons and generate full-length, functional proteins. Unlike conventional DNA editing approaches, which alter the underlying genetic sequence, RNA exon editing acts at the RNA level, providing an alternative strategy for correcting the consequences of genetic mutations without introducing permanent changes to the genome. The company has partnered and wholly owned programs spanning retinal, renal, neurological, neuromuscular, and other genetically defined diseases. Because the RNA exon editor is delivered via a durable AAV vector that persists in target cells, the approach could potentially enable long-term protein restoration from a single dose - while overcoming the strict ~4.7 kilobase cargo-size limits that restrict conventional gene replacement and larger DNA-editing systems. The clinical applicability and durability of this strategy across broader tissue types will require further validation. Exegenesis will contribute its proprietary AAVarta(R) platform, which integrates artificial intelligence algorithms with high-throughput experimental screening to discover and optimize AAV capsids with distinct delivery profiles. The platform will support the identification of capsids with tissue-specific delivery characteristics suited to individual RNA exon editing programs, helping address differences in tissue accessibility and delivery requirements across disease indications. Non-Exclusive agreement preserves development flexibility. Under the agreement, the companies will evaluate Exegenesis' existing AAV capsids and capsid discovery capabilities against the delivery requirements of individual RNA exon editing programs. Scientific assessments and predefined target capsid profiles will guide the selection of compatible RNA exon editor-capsid combinations for further in vivo evaluation and potential clinical development, with Ascidian retaining responsibility for the subsequent execution of the resulting combinations. The non-exclusive structure allows Ascidian to continue working with other capsid engineering providers when necessary. It also preserves the company's flexibility to select clinical and commercial manufacturing partners according to individual program requirements. Exegenesis retains the right to independently or collaboratively develop the relevant capsids outside Ascidian's RNA exon editing programs. The companies have not disclosed specific target indications, individual program details, or financial terms for the agreement. Delivery optimization becomes a key development priority. Daniel Rosan, President, Chief Financial and Business Officer of Ascidian Therapeutics, highlighted the importance of matching delivery strategies to individual patient populations and therapeutic programs. Exegenesis CEO Zhenhua Wu, Ph.D., emphasized the potential of AI-assisted capsid engineering to expand delivery options across different tissue types. The agreement reflects a broader development reality for genetic medicines: therapeutic editing capabilities alone are insufficient without reliable, targeted delivery to the intended cells. For Ascidian, access to programmable capsid discovery could expand the potential application of RNA exon editing across multiple disease areas. However, the partnership's value will ultimately depend on whether the selected capsids demonstrate sufficient tissue targeting, delivery efficiency, and tolerability in preclinical studies. The next major milestone for the collaboration will be the generation of in vivo proof-of-concept data supporting specific RNA exon editor-capsid combinations. Neither company has disclosed a clinical development timeline for the partnered programs. Reference. Ascidian and Exegenesis Bio Enter Strategic Agreement to Accelerate Development of RNA Exon Editors with Next-Generation AAV Capsids, Ascidian Therapeutics, 12 November 2026 About the Writer Creola Gonsalves (Linkedin) is an M.S. Biotechnology postgraduate with a strong interest in clinical research, evidence interpretation, and healthcare writing, with a focus on translating life-science knowledge into meaningful real-world insights. She is trained in Good Clinical Practice (GCP), clinical research principles, and critical interpretation of randomized clinical trials, with certifications from NIH and Stanford University. Her research background in biotechnological applications and microbial research strengthens her ability to understand scientific evidence and develop clear, accurate, and research-driven healthcare content.
Ascidian, Exegenesis Bio to collaborate on therapeutic RNA exon editors. November 12, 2026 03:28 PM EST NEW YORK - Ascidian Therapeutics and Exegenesis Bio said on Thursday that they are collaborating to develop a suite of therapeutic RNA exon editors for several unnamed diseases. Get the full story with GenomeWeb Premium. Premium Access Trial - only $95 for the first 90 days
GLP-1/GIP dual target weight loss innovative drug. Jul 26, 2026 View: 104 Leave a message GLP-1 Injections 1.General Specification(in stock) (1)API(Pure powder) (2)Tablet (3)Capsule (4)Injection (5)Liquid Drops 2.Customization: Shaanxi BLOOM Tech Co., Ltd will negotiate individually, OEM/ODM, No brand, for secience researching only. Internal Code:BM-3-094 GLP-1 CAS 87805-34-3 Main market: USA, Australia, Brazil, Japan, Germany, Indonesia, UK, New Zealand, Canada etc. Manufacturer: BLOOM TECH Xi'an Factory Analysis: HPLC, LC-MS, HNMR Technology support: R&D Dept.-4 Shaanxi BLOOM Tech Co., Ltd provide GLP-1 injections, please refer to the following website for detailed specifications and product information. Hanson Pharmaceutical's GLP-1/GIP dual target weight loss innovative drug, Olepotide, has been accepted for application and marketing for weight loss indications. On June 4, 2026, Hanson Pharmaceutical Group Co., Ltd. (hereinafter referred to as "Hanson Pharmaceutical", 03692. HK) announced that the company's innovative drug Olepotide Injection Market Authorization Application (NDA) has been accepted by the National Medical Products Administration (NMPA) of China for long-term weight management in obese or overweight adults. Olepotide is a glucagon like peptide-1 (GLP-1)/glucose dependent insulinotropic polypeptide (GIP) dual receptor agonist independently developed by Hanson Pharmaceuticals. By selectively activating GLP-1/GIP receptors, it regulates metabolic pathways related to appetite control, glucose metabolism, and energy balance, producing biological effects such as sugar control and weight loss. Its administration method is once a week, subcutaneous injection. In March 2026, the first phase III clinical study (HS-20094-301) of olepotide in overweight or obese adult subjects in China achieved its primary endpoint. After 48 weeks of treatment with olepotide, the average weight loss from baseline was as high as 19.3%, and the proportion of subjects who achieved a 25% weight loss was as high as 97.2%. Research has shown that the treatment group with oxaliplatin exhibits excellent gastrointestinal tolerance, with an average nausea incidence rate of<10% and an average vomiting incidence rate of<5%. Compared with the phase III trial data of GLP-1 related dual agonist drugs that have been published, the incidence of gastrointestinal adverse events and treatment discontinuation rate are lower. The dual target CAR-T cell injection of mRNA LNP from Shiyao Group has been approved for clinical use in China. On June 2, 2026, Shiyao Group (1093. HK) announced that its first dual target chimeric antigen receptor (CAR) - T-cell injection (SVS6063 injection) based on mRNA LNP has been approved by the National Medical Products Administration of the People's Republic of China for clinical trials in China. This product is the world's first approved dual target cell therapy product based on mRNA LNP. By expressing CAR that can specifically recognize leukocyte differentiation antigen 19 (CD19) and B-cell maturation antigen (BCMA), it accurately identifies and eliminates CD19 and BCMA positive cells in patients' bodies, thereby achieving therapeutic goals. The synergistic effect of dual targets can eliminate pathogenic cells from the source, fundamentally improve the patient's condition, and solve the pain points that traditional treatments cannot cure and are prone to recurrence. Preclinical studies have shown that this product can significantly kill CD19 and BCMA positive cells, and has good safety and efficacy. The clinical indication approved this time is relapsed and refractory systemic lupus erythematosus. In addition, this product is expected to be used for the treatment of other B cell/plasma cell-mediated autoimmune diseases such as myasthenia gravis (MG), rheumatoid arthritis (RA), and anti neutrophil cytoplasmic antibody (ANCA) - related vasculitis. It can also be extended to relapsed/refractory multiple myeloma (MM), B-cell lymphoma, and other hematological tumors, and has high clinical development value. The approval of the clinical trial of this product is another important achievement of its group's layout in the field of cell therapy, laying a good foundation for the development of cell therapy products including in vivo generated CAR-T. -Six months of needle therapy, Roche/Alnylam's new SiRNA antihypertensive drug is planned to be included in breakthrough therapies. On June 2, 2026, the Center for Drug Evaluation (CDE) of the National Medical Products Administration announced that Roche's Zilebesiran is intended to be included as a breakthrough therapy for adult hypertensive patients with cardiovascular disease or high risk of cardiovascular disease, in order to reduce the risk of cardiovascular death, non fatal myocardial infarction, non fatal stroke, and heart failure events (hospitalization or emergency treatment for heart failure). Zilebesiran is a subcutaneous RNAi therapy developed by Alnylam based on its enhanced stable chemical PLUS (ESC+) GaINAc conjugate technology. It reduces angiotensin levels by targeting the synthesis of angiotensinogen (AGT), the most upstream component of the renin angiotensin aldosterone system (RAAS), a classic pathway for blood pressure regulation. Ultimately, it leads to a sustained decrease in blood pressure. In July 2023, Roche and AInylam reached an agreement to obtain the cooperative development and commercialization rights of the drug for a total transaction value of up to $2.8 billion. In September 2025, the two parties launched Zilebesiran's first phase IV clinical trial. ZENISH). This study is a global, randomized, double-blind, placebo-controlled clinical trial (n=11000) aimed at evaluating the effectiveness and safety of Zilebesiran (300mg, subcutaneous injection, once every 6 months) combined with standard treatment (OC) compared to placebo combined with SOC in reducing the incidence of major cardiovascular adverse events (MACE) in hypertensive adult patients with diagnosed cardiovascular disease or poor blood pressure control at high risk of cardiovascular disease. The primary endpoint of the study is the time to the first occurrence of cardiovascular death, non fatal myocardial infarction (MI), non fatal stroke, or heart failure (HF) within 5 years of treatment. 1.9 billion US dollars! Eli Lilly and Ascidian reach a global agreement to develop RNA exon editing drugs. On June 3, 2026, Ascidian Therapeutics announced today that it has reached a global research collaboration and licensing agreement with Ei Lily and Company. Both parties will jointly discover and develop therapies for undisclosed monogenic kidney diseases, and Ei Lily has the right to choose to expand the collaboration to other targets. Ascidian's RNA exon editor can edit multiple complete exons at the kilobase scale to repair genetic instructions that cause diseases. This technology is designed specifically to address large genes or genes with high mutation diversity, expanding the boundaries of gene drugs. According to the terms of the agreement, Eli Lilly will obtain exclusive, target specific rights to Ascidian's RNA exon editing technology for undisclosed kidney disease targets. Ascidian will lead drug discovery and selected preclinical activities, while Eli Lilly will be responsible for additional preclinical research, clinical development, production, and commercialization. Ascidian is eligible for up to $1.9 billion in payments, including prepayments, development and commercialization milestone payments, and tiered royalties based on global sales. Ascidian reserves the right to independently or jointly develop other targets in the field of kidney with other partners. Alnylam and Inceptive have reached a strategic AI collaboration to accelerate the development of RNAi therapy. On June 3, 2026, Alnylam Pharmaceuticals (NASDAQ: ALNY) and lnceptive Nucleics, a company dedicated to building life science foundation models, announced today that they have signed a strategic partnership agreement aimed at accelerating the development of innovative therapeutic drugs. The total value of the partnership project is up to $2 billion, including a $30 million advance payment (including cash and Inceptive equity subscription). In addition, Inceptive is eligible for additional interim payments based on the achievement of milestones such as preclinical research, regulatory approvals, and commercial sales. This collaboration combines AInvlam's leadership position in the RNAi field with lnceptive's foundational models and AI expertise, aiming to catalyze and accelerate the development of nucleic acid drug design. Innovative focuses on developing models for stem based drugs, such as RNAi therapy pioneered by Alnylam. Alnylam's platform: a research and development engine that has produced six marketed drugs and has over 20 years of proprietary siRNA data support. Inceptive Life Foundation Model: Suitable for A! Models based on sequential drugs, it can generalize and continuously self optimize across different projects. The aim of this collaboration is to model the target mRNA and jointly explore sequence space and novel chemical modifications to enhance efficacy and therapeutic effects, while predicting the optimal therapeutic candidate molecules in preclinical models for further development by AInyiam, thereby promoting advances in SiRNA design. Its goal is to help Alnylam prioritize the screening of the most promising molecules and improve experimental efficiency. No Information Send Inquiry
Eli Lilly continues genetic medicine push with $1.9bn Ascidian partnership. Lilly's deal with Ascidian is one of several that the pharma giant has cut within the realm of genetic medicines. Eli Lilly has penned a research collaboration deal with Massachusetts-based biotech, Ascidian Therapeutics, to discover and develop drugs for kidney diseases - adding another bead to the string of genetic medicine-focused deals Lilly has signed over the past few months. Through this partnership worth up to $1.9bn for Ascidian, the pair will initially look to discover and develop therapies for kidney diseases caused by a singular gene, though Lilly can choose to expand to additional targets if the company so chooses. In exchange for an undisclosed upfront payment and its commitment to potential milestone and tiered royalty payments, Lilly will receive the exclusive, target-specific rights to Ascidian's RNA exon editing technology for certain kidney disease targets, which currently remain undisclosed. As per the deal, Ascidian will take charge of the discovery and some of the preclinical activities associated with any targets identified through the partnership, while Lilly will take the helm for additional preclinical work, as well as clinical R&D, manufacturing and commercialisation. The key driver of these efforts will be Ascidian's RNA-based exon editors, which are designed to correct faulty genes linked to disease at the kilobase scale. According to Ascidian's president and CEO, Michael Ehlers, this approach offers the opportunity to rewrite genes at their source without altering DNA, which he says could "open the door to diseases long out of reach" with current gene and base editing approaches. Ascidian also touts the potential of the RNA exon editing approach in lowering the risks linked to direct DNA editing and gene replacement, while providing a durable response similar to those seen with gene therapies. Lilly's dealmaking spree continues. Lilly's deal with Ascidian is the big pharma company's third this week alone, following its $1.26bn licensing deal with Hanmi Pharm for its glucagon-like peptide 2 (GLP-2) agonist, sonefpeglutide, and its $3bn deal with Haisco Pharmaceutical to develop innovative medicines. While Lilly has been casting the net wide with its dealmaking strategy, the company has heavily invested in genetic medicines over the last year, with the company penning multiple partnerships, licensing deals and acquisitions with biotechs in the space. As the pharma giant waits to see if these approaches will bear fruit, it is also making active efforts to bolster its genetic medicines manufacturing output, having pledged $4.5bn to upgrade its dedicated Indiana-based facility on its Lebanon campus back in May 2026. These hefty investments come as Lilly looks to expand its commercial success by building a diversified pipeline - funded by the capital generated from strong sales of its weight loss portfolio, which continued on its strong growth trajectory in Q1 following a stellar 2025. Outside of the genetic medicines realm, notable Lilly deals from 2026 include its $7.8bn acquisition of sleep drug biotech, Centessa, as well as its $8.8bn pact with Chinese biotech, Innovent, focused on developing new cancer and immune disorder therapies.
Eli Lilly, Ascidian sign $1.9 billion deal to develop kidney-disease treatments. Published on 06/03/2026 at 09:08 am EDT By Katherine Hamilton Eli Lilly signed a collaboration and licensing agreement worth up to $1.9 billion with Ascidian Therapeutics to research and develop kidney-disease treatments. Ascidian, a Boston-based biotechnology company, said Wednesday it granted Eli Lilly exclusive, target-specific rights to its RNA-exon-editing technology for undisclosed kidney-disease targets. The RNA-exon editors are capable of altering parts of genetic code to repair genetic instructions that cause disease. Ascidian said it will lead discovery and certain preclinical activities, while Eli Lilly will be responsible for other preclinical work, clinical development, manufacturing and commercialization. Ascidian is eligible to receive up to $1.9 billion, including an upfront payment, development and commercial milestone payments, and tiered royalties on commercial sales worldwide, it said. Ascidian is retaining the right to pursue other targets in kidney disease, it said. Write to Katherine Hamilton at [email protected] (END) Dow Jones Newswires 06-03-26 0907ET (C) Dow Jones - 2026
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Industries
Company Size
11-50
Company Stage
Series A
Total Funding
$90M
Headquarters
Boston, MA
Founded
2020
Find jobs on Simplify and start your career today
Don't see your dream role? Check out thousands of other roles on Simplify.Browse all jobs