MiNA Therapeutics Ltd.

LONDON--(BUSINESS WIRE)--MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, announced today that it will present new pre-clinical data on its RNA activation program for the treatment of sickle cell disease at the European Hematology Association 2025 Congress. Pre-clinical studies of MTL-HBG drug candidate demonstrated best-in-class activity in a highly translatable model and underscore its transformational therapeutic potential in sickle cell disease. The data will be presented on June 14, 2025 at 5:30 p.m. CEST in an oral presentation. MTL-HBG is an RNAa medicine designed to increase transcription of the gamma globin gene (HBG), enabling people with sickle cell disease to produce enhanced levels of fetal hemoglobin (HbF). HbF is a compensatory form of hemoglobin which, when induced to sufficient levels, protects people with sickle cell disease from a range of symptoms including recurrent vaso-occlusive crises and progressive organ damage. “The extraordinary levels of fetal hemoglobin induced by MTL-HBG underscore its transformational therapeutic potential,” said Robert Habib, CEO of MiNA Therapeutics. “This new pre-clinical data further increases our confidence of translating the potential therapeutic benefits of MTL-HBG to patients with sickle cell disease.” MTL-HBG is administered in vivo without the need for harmful pre-conditioning or complex cell engineering. MTL-HBG comprises an RNAa payload, which directly targets the HBG gene and is encapsulated in NOV340 liposomes. MTL-HBG has previously demonstrated promising biodistribution and in vivo activity in non-human primates. MTL-HBG is currently in IND-enabling studies. The presentation highlights how MTL-HBG induced HbF to 62% as a proportion of total hemoglobin (% HbF) in erythroid progenitor cells derived from four patients with sickle cell disease. These levels represented an absolute increase of 45% HbF compared to negative controls. In addition, induction of HbF was demonstrated to be pancellular, with 82% of erythroid progenitor cells expressing HbF. The studies were conducted with reduced drug doses that replicate the level of cellular uptake of MTL-HBG anticipated in patients following systemic administration. Oral Presentation Details Title: Small activating RNA-mediated induction of HBG via liposome delivery for in vivo treatment of sickle cell disease Session Date and Time: Saturday, June 14, 2025, 5:30 p.m. – 5:45 p.m. CEST Session Title: Sickle cell disease complications and therapeutic strategies Room: Coral 3 Abstract Number: S287 Following the congress, the oral presentation will be available on the MiNA website: www.minatx.com. About MiNA Therapeutics MiNA Therapeutics is the leader in small activating RNA therapeutics. Harnessing innate mechanisms of gene activation, small activating RNA therapeutics are a revolutionary new class of medicines that can restore normal function to patients’ cells. We are advancing a proprietary pipeline of new medicines for genetic diseases, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of therapeutic areas. Based on our unique know-how in RNA activation we are expanding the possibilities of RNA-based medicine for patients. For more information, visit www.minatx.com.

The treatment will be focused on sensorineural hearing loss, which is the most common cause of hearing loss. \n Eli Lilly is further expanding its RNA medicine strategy by tapping South Korea’s Rznomics for a $1.3 billion biobucks deal focused on treating hearing loss.The centerpiece of the agreement is Rznomics’ trans-splicing ribozyme platform, which the Seongham, South Korea-based company has touted as enabling “precise RNA editing and has broad applicability across multiple indications.” The plan is for the companies to use the platform to co-develop a therapy for inherited hearing loss.The treatment will be focused specifically on sensorineural hearing loss, which is the most common cause of hearing loss. Rznomics will conduct early-stage research before handing over responsibility to Lilly for further development and, hopefully, commercialization.While Rznomics will receive an upfront payment from Lilly as part of the financial package, the biotech didn’t break down the details in its May 15 release. The total value of the deal could reach $1.3 billion, with the South Korean biotech also in line for a slice of the royalties, should the therapy make it to market.“We are thrilled to collaborate with Lilly, a global leader in pharmaceutical innovation,” Rznomics CEO Seong-Wook Lee said in the release. “This partnership validates our trans-splicing ribozyme platform and opens the door to treating previously intractable diseases with precision RNA therapeutics.” Rznomics’ lead clinical-stage candidate is RZ-001, an RNA replacement enzyme-based gene therapy aimed at liver and brain cancer.Meanwhile, Lilly has stepped up its RNA therapeutics strategy in recent months, including a $13 million upfront deal with Creyon Bio two weeks ago to develop AI-designed oligonucleotide therapies spanning a “a broad range of diseases.” Oligonucleotides work as short strands of synthetic DNA or RNA that can reduce, restore or modulate RNA through several different mechanisms.In August 2024, the Big Pharma opened a $700 million R&D center in the Boston Seaport, boosting its RNA and DNA research capabilities. Further back, Lilly has clinched RNA-focused deals with the likes of MiNA Therapeutics and ProQR Therapeutics. 

Tadekinig alfa is designed to bind and block IL-18. Credit: TheCorgi/Shutterstock. AB2 Bio has made an option and licencing agreement with Nippon Shinyaku , granting the latter the option to obtain exclusive rights to commercialise Tadekinig alfa in the US. This therapy treats a rare paediatric condition, primary monogenic interleukin-18 (IL-18)-driven hyperinflammatory syndrome, which can lead to rapid multiple organ failure and mortality if not treated. AB2 Bio will receive up to $36m in early payments, including a $6m payment upon signing. The company could also receive up to $150m in development milestone payments and commercial milestone and royalty payments potentially amounting to $500m. AB2 Bio is set to continue its preparations for a US biologics licence application (BLA) approval for the therapy for this indication. Upon Nippon Shinyaku exercising its option rights and following AB2 Bio’s receipt of the BLA approval in the US, NS Pharma, Nippon Shinyaku’s wholly owned subsidiary, will undertake the marketing of the therapy. Nippon Shinyaku will have exclusive rights to commercialise Tadekinig alfa for its primary indication in individuals with NLRC4 mutation and X-linked inhibitor of apoptosis protein (XIAP) deficiency within the US. AB2 Bio retains the rights to the therapy for all other indications within the country, including the US Virgin Islands, Guam and Puerto Rico, as well as for all indications in other parts of the world. AB2 Bio CEO Djordje Filipovic stated: “This partnership will accelerate bringing Tadekinig alfa to young patients suffering from this rare and devastating disease and potentially provide a significant improvement in treatment.” Tadekinig alfa is a recombinant human IL-18 binding protein. In April 2024, MiNA Therapeutics entered a research partnership and licensing agreement with Nippon Shinyaku. The collaboration is focused on developing small activating RNA therapeutics for rare neurodegenerative conditions.