Severe Combined Immunodeficiency

As global healthcare continues to demand innovative therapies, February 2025 has emerged as a critical period witnessing the robust growth of the biopharmaceutical sector. This month has seen significant capital inflows supporting preclinical-stage research and multiple high-impact collaborations and licensing deals. For example, CSPC Pharmaceutical, through its subsidiary Megalith Biopharmaceutical, transferred the overseas rights of its ROR1-targeting antibody-drug conjugate (ADC), SYS6005, to Radiance Biopharma in a transaction valued at up to $1.24 billion. 1. Chiome Bioscience Partners with OmniAb for Efficient Bispecific Antibody Screening Chiome Bioscience has announced a collaborative research agreement with OmniAb, a company headquartered in Emeryville, California, to explore the synergistic effects of their technologies in bispecific antibody generation. This collaboration will integrate Chiome’s proprietary ADLib® system-based cell surface display technology with OmniAb’s OmniClic® transgenic chicken platform, which is designed to discover candidate monoclonal antibody arms. The partnership aims to establish a high-throughput screening system for bispecific antibodies, facilitating rapid evaluation of various combinations and providing strong technological support for future drug discovery. As part of this collaboration, Chiome plans to apply its new technology to its upcoming Integrated Drug Discovery (IDD) business, which focuses on antibody development platforms. By combining these two advanced technologies, Chiome seeks to enhance its profitability and provide more efficient solutions to its partners. Although the financial impact of this agreement on Chiome’s fiscal results for the year ending December 31, 2025, is expected to be minimal, it marks a significant step toward next-generation antibody drug discovery. Bispecific antibodies, an emerging antibody format designed based on novel mechanisms of action, have rapidly evolved in recent years, becoming a core component of next-generation antibody drug discovery. These antibodies can flexibly bind two different antigens, either targeting two distinct epitopes or identifying the most suitable monoclonal antibody pairs, which is critical for effective screening. Through this collaboration, Chiome and OmniAb aim to achieve breakthrough advancements in this field. OmniAb is renowned for licensing its cutting-edge discovery research technologies to pharmaceutical companies, biotech firms, and academic institutions to advance next-generation therapeutics. The OmniClic® technology, which features a common light chain, is specifically designed to facilitate bispecific antibody generation, significantly simplifying the design of complex antibody structures. Chiome's collaboration with OmniAb is based on the potential of OmniClic® to accelerate antibody drug development. Both parties aspire to drive innovation and development in this sector through their combined expertise. For Chiome’s IDD business, this research outcome will be a key driver of growth. The IDD business serves the diverse R&D needs of partner companies—ranging from pharmaceutical firms leveraging knowledge, experience, and technology—with the goal of achieving milestone-based revenue through collaborative antibody drug discovery. By integrating OmniAb’s technology, Chiome can not only enhance its own technical capabilities but also better serve its global partners, contributing to addressing unmet medical needs. 2. Magnet Biomedicine and Eli Lilly Join Forces to Pioneer Molecular Glue Therapy for Cancer Magnet Biomedicine, a biopharmaceutical company specializing in the discovery of molecular glues through rational screening and design, has announced a significant collaboration and licensing agreement with Eli Lilly and Company. The partnership aims to jointly discover, develop, and commercialize molecular glue therapies in the field of oncology. Leveraging Magnet’s TrueGlue™ discovery platform, the collaboration seeks to identify molecular glues that induce protein proximity and cooperativity, enabling novel mechanisms of action to tackle previously undruggable targets across various diseases and address unmet medical needs. Under the terms of the agreement, Magnet will receive up to $40 million in upfront, near-term payments, and equity investments. Additionally, Magnet is eligible for over $1.25 billion in milestone payments related to development, regulatory, and commercialization achievements, along with tiered royalties based on global net sales. This agreement not only validates Magnet’s technology but also reflects Lilly’s confidence in the future potential of this field. Through this collaboration, Magnet will further advance its innovative platform, while Lilly can leverage cutting-edge technology to provide more effective treatment options for patients. Molecular glues are small-molecule compounds that can alter the surface properties of target proteins, facilitating or inducing interactions with other proteins to achieve specific biological functions, such as protein degradation, pathway inhibition, or activation. Magnet’s TrueGlue™ platform emphasizes rational design over traditional serendipitous discovery methods, offering a more precise approach to identifying therapeutically promising molecular glues. In oncology, the application of molecular glues is particularly significant, as they can target disease-driving proteins that conventional drugs cannot effectively address, opening new avenues for cancer treatment. Lilly’s involvement extends beyond financial investment—it brings extensive technical expertise and experience to the partnership. As a leader in small-molecule drug development, Lilly will contribute its knowledge and resources in drug development, which is crucial for accelerating the transition from laboratory discoveries to clinical applications. This collaboration also underscores the growing potential of molecular glue technology as a revolutionary therapeutic approach, particularly in addressing traditionally "undruggable" targets. The partnership between Magnet Biomedicine and Eli Lilly represents a major advancement in the field of molecular glues, signaling the dawn of a new era in targeted therapies. With similar collaborations continuing to emerge, molecular glues have the potential to provide novel treatment options for various hard-to-treat diseases, including cancer. This innovation is not just a complement to existing therapies—it could fundamentally change the way certain diseases are treated, offering new hope for patients. Through such strategic collaborations, both companies are committed to driving scientific progress and improving global health outcomes. 3. Medigene and EpimAb Biotherapeutics Collaborate to Develop TCR-Guided T-Cell Engagers On February 27, 2025, Medigene AG and EpimAb Biotherapeutics announced a strategic collaboration to jointly research and develop off-the-shelf TCR-guided T-cell engagers (TCR-TCEs) for the treatment of immune-related diseases, including solid tumors. This collaboration leverages Medigene’s expertise in generating and characterizing TCRs with sensitivity, specificity, and safety (3S) alongside EpimAb’s proprietary CD3 antibody and T-FIT platform. Through this partnership, the two companies aim to develop highly targeted bispecific therapies that minimize off-target effects and improve patient outcomes. The bispecific therapy market presents significant opportunities in oncology, addressing critical unmet needs in both solid and hematologic malignancies. More than five million cancer patients globally face low five-year survival rates each year, underscoring the urgent demand for innovative treatments. Bispecific TCR-TCEs offer a promising solution by enabling the immune system to target and eliminate cancer cells with greater precision. The market for these therapies is projected to grow at a compound annual growth rate (CAGR) of 40.9% from 2023 to 2030, with an estimated market size exceeding $80 billion by 2030. 4. Unravel Biosciences and COMBINED Brain Join Forces to Advance Drug Discovery for Rare Neurological Diseases Unravel Biosciences and COMBINED Brain have announced a collaborative initiative aimed at identifying promising drug candidates for over 110 rare genetic neurological disorders represented by member patient advocacy groups. This collaboration represents one of the largest partnerships between the biopharmaceutical industry and patient communities, leveraging Unravel’s BioNAV™ drug discovery platform and COMBINED Brain’s extensive patient biosample repository. Through this partnership, the two organizations aim to rapidly identify therapies for clinical trials and provide timely treatment options for children with rare diseases. Unravel Biosciences’ BioNAV™ platform is an AI-powered target and drug discovery system that utilizes human health gene expression networks to identify small molecule modulators with potential therapeutic effects for complex diseases. Meanwhile, COMBINED Brain maintains a unique large-scale patient biosample collection, enabling the generation of RNA datasets to facilitate novel drug target discovery. This collaboration is not only focused on developing new drugs but also on repurposing existing approved medications to accelerate clinical translation and deliver patient benefits as quickly as possible. 5. HBM Alpha Therapeutics Enters Strategic Partnership to Advance Novel Endocrine Therapies HBM Alpha Therapeutics (HBMAT), an innovative biotechnology company incubated by HBM Holdings, has entered into a strategic collaboration and licensing agreement with a business partner to advance the development of therapies targeting corticotropin-releasing hormone (CRH). Under the agreement, the partner has secured exclusive development and commercialization rights for HAT001/HBM9013 outside Greater China. HAT001/HBM9013 is a potent and selective neutralizing antibody, and through this partnership, HBMAT is expected to receive up to $395 million in revenue, including upfront payments, milestone payments, and tiered royalties on future net sales. HAT001/HBM9013 is specifically designed to target CRH, reducing the adrenocorticotropic hormone (ACTH)-induced effects mediated by CRH. Preclinical studies have demonstrated significant efficacy, and the therapy is now advancing toward clinical development. This treatment holds potential for conditions such as congenital adrenal hyperplasia (CAH), a rare genetic disorder. Currently, CAH relies on treatment approaches developed over 70 years ago, with no significant updates in therapeutic options. The emergence of HAT001/HBM9013 could provide a breakthrough solution for patients. This partnership marks a significant milestone for HBMAT in its mission to develop innovative therapies for endocrine disorders and represents an important step in HBM Holdings’ global company incubation strategy. By collaborating with an experienced drug development partner, HAT001/HBM9013’s clinical progress is expected to accelerate, offering more effective treatment options for patients worldwide. Additionally, HBMAT holds warrant rights to acquire a minority equity stake in its partner, further strengthening its financial position and growth potential. HBMAT focuses on developing antibody therapies for rare genetic disorders such as congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS), addressing significant unmet medical needs. Beyond these indications, HAT001/HBM9013 may also be applicable to other disorders related to the hypothalamic-pituitary-adrenal (HPA) axis, highlighting its broad therapeutic potential and significant market opportunity. With the signing of this agreement, HBMAT will be able to concentrate resources and technological capabilities to accelerate the development of HAT001/HBM9013 and other promising therapies. This initiative represents not only a proactive response to current medical challenges but also a crucial step in future medical advancements. Through continuous technological innovation and strategic collaborations, HBMAT aims to establish itself as a leading biopharmaceutical company in the field of endocrine and related disorders, contributing to the improvement of human health. 6. IPA and RIBOPRO Collaborate to Advance mRNA-Driven Antibody Discovery, Ushering in a New Era of Immunotherapy ImmunoPrecise Antibodies Ltd. (IPA) and RIBOPRO have announced a strategic partnership aimed at revolutionizing the discovery and development of therapeutic antibodies. By integrating RIBOPRO's expertise in mRNA sequence optimization and lipid nanoparticle (LNP) delivery technology with IPA's advanced B-cell screening, single-cell analysis, and AI-driven discovery processes, the collaboration seeks to accelerate the development of novel therapeutics while enhancing antigen expression and immune response quality—critical steps in the antibody discovery process. Traditional immune-based antibody discovery platforms often face challenges in properly expressing complex antigens, which can hinder their ability to elicit effective immune responses. However, by leveraging RIBOPRO’s proprietary mRNA and LNP technologies, this collaboration enables precise and efficient antigen expression, potentially leading to stronger immune responses and a faster path to novel antibody therapeutics. This partnership introduces an innovative approach to overcoming long-standing challenges in antigen expression, opening new possibilities for precision medicine. IPA’s approach combines cutting-edge AI-driven analysis with highly specialized laboratory techniques to design and optimize antibodies with the highest clinical relevance. Incorporating mRNA immunization into IPA’s workflow is expected to further enhance the accuracy and efficiency of its antibody discovery pipeline. This collaboration exemplifies the convergence of modern biotechnology and artificial intelligence, playing a crucial role in accelerating drug development and improving success rates. 7. WuXi XDC and LigaChem Biosciences Strengthen Partnership to Accelerate ADC Therapeutic Innovations On February 25, 2025, WuXi XDC, a global leader in end-to-end CRDMO services for antibody-drug conjugates (ADCs) and other bioconjugates, announced the signing of an expanded Memorandum of Understanding (MOU) with LigaChem Biosciences. This agreement builds upon their existing collaboration since 2021 and aims to further accelerate the development of next-generation ADC therapies. Through this partnership, LigaChem will leverage WuXi XDC’s cutting-edge technologies and extensive expertise to significantly enhance the efficiency of its ADC development programs. Under the expanded MOU, WuXi XDC’s fully integrated ADC discovery platform will play a pivotal role in expediting LigaChem’s ADC research and development. By utilizing WuXi XDC’s advanced technologies and deep industry expertise, LigaChem can more effectively navigate all stages of ADC development, from discovery to clinical translation. Additionally, LigaChem will leverage WuXi XDC’s comprehensive ADC development and manufacturing platform to support multiple ADC projects, ensuring streamlined and efficient processes throughout the development cycle. Since initiating their partnership in 2021, WuXi XDC and LigaChem have made significant strides in advancing ADC and other bioconjugate innovations. This latest agreement further solidifies their shared commitment to driving the next wave of ADC breakthroughs, with the goal of delivering meaningful benefits to patients worldwide. As their collaboration deepens, both companies remain focused on leveraging continuous technological advancements and service innovations to accelerate the development of novel therapeutic solutions and improve patient outcomes. 8. BridGene and Takeda Partner to Advance Small Molecule Drug Discovery in Immunology and Neurology On February 25, 2025, BridGene Biosciences announced a strategic collaboration and licensing agreement with Takeda, a global pharmaceutical leader. Under this agreement, BridGene will utilize its proprietary chemoproteomics platform, IMTAC™, to discover novel small-molecule drug candidates in the fields of immunology and neurology. This collaboration targets historically "undruggable" targets, signifying both the recognition of BridGene’s technological capabilities and the potential for groundbreaking medical advancements. Through this partnership, BridGene stands to receive up to approximately $770 million in total payments from Takeda, including an upfront payment and early milestone payments totaling $46 million. Furthermore, if all milestones are achieved, BridGene may receive additional clinical and commercial milestone payments, along with tiered royalties on future sales. This financial structure not only provides strong funding support for BridGene but also incentivizes continuous innovation throughout the research and development process. For Takeda, this collaboration grants exclusive rights to develop and commercialize any potential products emerging from the partnership. IMTAC™, BridGene’s core technology platform, employs a chemoproteomics approach to identify small-molecule ligands for "undruggable" targets in a live-cell environment. What sets this technology apart is its ability to screen across the entire proteome, enabling the discovery of new disease-relevant targets and mechanisms. In the fields of immunology and neurology, IMTAC™ offers a promising avenue for identifying novel therapeutic strategies, bringing hope to patients with complex diseases. The collaboration is expected to investigate multiple targets, with both parties working together from the initial discovery of small-molecule candidates to the development of early lead compounds. If successful, this initiative will significantly expand the range of druggable targets, particularly for key molecular markers previously deemed intractable. With the emergence of more innovative therapies, this partnership is poised to enhance patient outcomes and pave new directions for the pharmaceutical industry. 9.ReCerise and NCCS Collaborate to Advance Innovative Hepatocellular Carcinoma Research On February 21, 2025, ReCerise Therapeutics, a company dedicated to developing first-in-class therapies for cancer, announced a partnership with the National Cancer Centre Singapore (NCCS) to develop innovative treatments for hepatocellular carcinoma (HCC) through multi-omics data analysis. This collaboration specifically focuses on RCT1213, an investigational candidate currently under development for HCC treatment. Through this joint research, both parties aim to conduct a multi-layered study on the potential of RCT1213. The collaboration will take place under the PLANet Project (Precision Medicine in Liver Cancer in the Asia-Pacific Network), an initiative led by NCCS based on the concept of intra-tumor heterogeneity and the highly dynamic tumor microenvironment (TME) of liver cancer. This longitudinal study will conduct comprehensive multi-omics analyses on tumor and blood samples from HCC patients. The project will leverage the extensive multi-omics data collected under the PLANet Project and will be led by world-renowned HCC expert Professor Pierce Chow. Professor Chow is a Senior Consultant in the Department of Surgery and Surgical Oncology at Singapore General Hospital (SGH) and NCCS, and he also serves as the Principal Investigator of the PLANet Project. While the specific mechanism of action of RCT1213 has not been fully disclosed, it is hypothesized that the therapy may target specific liver cell proteins or pathways that have shown promise as potential treatment targets. Through its collaboration with NCCS, ReCerise aims to investigate the expression of these targets in real-world HCC patient populations and assess their impact on the liver microenvironment. This effort will not only provide deeper insights into the mechanism of action of RCT1213 but also lay the groundwork for future clinical trials. Hepatocellular carcinoma is a highly prevalent cancer with poor prognosis, primarily due to late-stage diagnosis and the limited efficacy of existing immunotherapy options. ReCerise was founded with the mission of exploring and developing novel therapeutic approaches to address this significant unmet medical need. With promising preclinical research and proof-of-concept studies on a specific liver protein, ReCerise now has the opportunity to expand its research through this collaboration, paving the way for potential future commercialization. Notably, this research and development initiative has received financial support from the South Korean government and is managed and supervised by the Korea Evaluation Institute of Industrial Technology (KEIT). This public sector support, in addition to private investment, plays a crucial role in accelerating new drug development. Yong-Bae Kim, CEO of ReCerise Therapeutics, expressed excitement about collaborating with NCCS to advance the development of innovative liver cancer treatments. As more data is gathered and technological advancements continue, ReCerise is optimistic about bringing more effective treatment options to HCC patients. 10.GeneCradle Technology and Buyang Gene Technology Join Forces to Pioneer a New Era in Duchenne Muscular Dystrophy Gene Therapy On February 19, 2025, GeneCradle Technology and Buyang Gene Technology officially signed a strategic cooperation agreement to jointly develop next-generation gene therapies for Duchenne muscular dystrophy (DMD). This collaboration aims to address the urgent clinical needs of DMD patients and bring new hope for treatment. DMD is a severe genetic disorder for which no curative therapies currently exist, and available treatments only provide partial symptom relief. Since 2021, GeneCradle Technology has focused on developing gene therapy drugs for DMD, with a core emphasis on optimizing and engineering the Dystrophin protein and its regulatory elements. The company has innovatively designed Smart-Dystrophin, which, compared to conventional mini-Dystrophin or micro-Dystrophin, exhibits superior biological activity in various DMD animal models, potentially improving disease progression. Meanwhile, Buyang Gene Technology has developed a series of novel AAV capsids through its proprietary DynEvoLib® platform. These capsids offer enhanced muscle-targeting capabilities and higher transgene expression efficiency while reducing immunogenicity and improving safety. This partnership combines GeneCradle Technology's extensive expertise in AAV-based gene drug development with Buyang Gene Technology’s cutting-edge innovations in next-generation AAV capsid engineering. By leveraging their respective strengths, the collaboration is expected to enhance the efficacy and safety of DMD gene therapy while potentially reducing the required dosage, thereby minimizing potential side effects. Additionally, this cooperation will contribute to the advancement of China’s independent gene therapy research and development, bringing hope to rare disease patients both in China and globally. Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder caused by mutations in the dystrophin gene, primarily affecting males. The disease leads to progressive muscle fiber degradation and atrophy, ultimately resulting in the loss of ambulation and life-threatening cardiopulmonary complications. While corticosteroids such as prednisone can temporarily alleviate symptoms, their long-term use is associated with significant side effects, and they do not address the underlying disease mechanism. Consequently, the search for effective gene therapy solutions has become a key focus in DMD research. As GeneCradle Technology and Buyang Gene Technology deepen their collaboration, their efforts are expected to drive further progress in DMD gene therapy, offering patients safer and more effective treatment options. This partnership marks a significant milestone in technological innovation and has far-reaching implications for the field of gene therapy. With continued breakthroughs in this area, there is reason to believe that the quality of life for patients with DMD and other genetic disorders will improve substantially, providing valuable insights and technological advancements for future treatments. 11.Sunshine Guojian and Shenyang Sunshine Deepen Cooperation to Optimize Resource Allocation and Drive High-Quality Development On February 20, 2025, Sunshine Guojian announced that the company and its wholly-owned subsidiary, Dansheng Pharmaceutical, plan to sign a License Agreement with Shenyang Sunshine Pharmaceutical Co., Ltd., granting exclusive rights to Project 612 and Project 708 to Shenyang Sunshine. Under the agreement, Sunshine Guojian will receive an upfront payment of CNY 102 million, along with milestone-based payments and 15% of sales revenue as royalties. This transaction not only provides Sunshine Guojian with immediate cash inflow but also lays the foundation for future revenue growth. Project 612 is a novel anti-HER2 humanized monoclonal antibody, while Project 708 is a bispecific antibody targeting PD-1/TGF-β. Both drugs are currently in the preclinical stage, but research suggests they exhibit synergistic effects when used in combination, demonstrating significant anti-tumor efficacy. Through this collaboration, Shenyang Sunshine has acquired full rights for these projects in Mainland China, covering R&D, manufacturing, and commercialization, which will further accelerate the development of these innovative therapies. It is worth noting that Shenyang Sunshine and Sunshine Guojian are both subsidiaries of 3SBio, with Shenyang Sunshine directly holding 6.42% of Sunshine Guojian’s shares, making this a related-party transaction. Since Sunshine Guojian’s strategic shift to focus on the autoimmune disease field, Shenyang Sunshine has repeatedly acquired its non-core pipeline assets. Notable examples include large-scale transfers in April 2023 and June 2024, with total transaction amounts reaching several hundred million yuan. This model has enabled Sunshine Guojian to realign its business strategy while providing Shenyang Sunshine with new growth opportunities. Read the Rest of the Series Highlights of Financing and Strategic Collaborations in the Preclinical Biopharmaceutical Sector: February 2025(2) How to get the latest progress on drug deals? If you would like to access the latest transaction event information, you can click on the 'Deal' module from the homepage of the Synapse database. Within the Deal module, you can search for global pharmaceutical transaction information using labels such as Drugs, Organization, Target, Drug Type, Deal Date.  Furthermore, you can obtain the original link to the transaction coverage by clicking on the "Deal Name." In the analysis view, you can see the most active assignors, assignees, popular targets, and other dimensions of analysis, as well as the distribution of research and development statuses at the time of the transaction, to help you better understand the search results. The Synapse database also supports the ability to view current transactions from the dimension of "drugs" (by selecting "drugs" from the "Adjust Dimension" dropdown menu above). Targeting transactions involving renowned pharmaceutical companies that are of interest to the industry, such as Merck, Roche, etc., Synapse has identified a group of "leading companies" through drugs that have achieved global sales exceeding 1 billion US dollars in 2022. Transactions involving drugs from these leading companies can be filtered by clicking on the "Leading Company" tag on the left-hand side. In addition to the drug transaction module, you can also view related transaction history on the drug detail page and the institution detail page. Click on the image below to explore new pharmaceutical funding transactions! 

FDA & EMA approvals for rare genetic diseases are fast and require clinical studies with small number of patients Ramat Gan, Israel, March 19, 2025 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced that a Phase II design is completed and preparatory work is undergoing for the initiation of clinical study in the rare genetic disease Lowe Syndrome. Dr. Franchesca Emma from the Division of Nephrology, Bambino Gesù Children's Hospital - IRCCS Rome Italy, will be the principal investigator of the study. The Phase II open study will enroll 5 patients that will be treated twice daily with 3 mg Piclidenoson for 12 months. The study’s primary end point will be the efficacy of Piclidenoson in increasing 99mTc-DMSA renal uptake. The treatment of this rare genetic disease is based on successful pre-clinical work of Dr. Antonella De Matteis, Professor of Biology, Department of Molecular Medicine and Medical Biotechnology at the University of Naples Federico II, and Program Coordinator of the Cell Biology and Disease Mechanisms at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy. Can-Fite and Fondazione Telethon have signed a collaboration agreement for the clinical development of Piclidenoson for the treatment of Lowe Syndrome, a high medical need with no drug available. Lowe Syndrome, also known as oculo-cerebro- renal syndrome (OCRL), an X-linked genetic condition occurring almost exclusively in males, is a multisystem disorder characterized by vision problems including clouding of the lenses of the eyes (cataracts) that are present at birth, kidney problems (consisting of urinary loss of proteins and solutes) that usually develop in the first year of life, and brain abnormalities associated with intellectual disabilities, and a life span that rarely exceeds 40 years. Lowe Syndrome prevalence is estimated at approximately 1 in 500,000. “Having tested thousands of compounds in search of a treatment for Lowe Syndrome, Piclidenoson is the only compound we’ve found to date that has shown to be effective in pre-clinical studies. Importantly, we observed that Piclidenoson treatment in preclinical models of Lowe syndrome leads to a significant decrease of the urinary loss of proteins,” Dr. De Matteis stated. “We chose to investigate Piclidenoson based on the availability of extensive scientific data showing its excellent safety, coupled with efficacy in this disease in pre-clinical studies which involves renal, cerebral, and ocular manifestations.” Can-Fite CSO&Chairperson Dr. Pnina Fishman commented, “We are very much enthusiastic by the breakthrough research of Dr. De Matteis showing that Piclidenoson is efficacious in treating pre-clinical models of Lowe Syndrome. Her impressive results are the basis for implementing Piclidenoson in the treatment of this rare genetic disease”. stated Dr. Pnina Fishman, Can-Fite CSO & Chairperson”. About Piclidenoson Piclidenoson is a robust anti-inflammatory agent, currently being evaluated in a pivotal Phase III psoriasis clinical study under approval of both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Piclidenoson is a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule, orally bioavailable drug with an excellent safety profile demonstrating evidence of efficacy in Phase II and Phase III clinical studies. The drug’s mechanism of action entails inhibition of the inflammatory cytokines interleukin 17 and 23 (IL-17 and IL-23) and the induction of apoptosis of patients’ skin cell keratinocytes involved with the disease pathogenicity. About Fondazione Telethon Fondazione Telethon ETS is one of the main Italian biomedical charities, founded in 1990 on the initiative of a group of patients suffering from muscular dystrophy. Its mission is to achieve the cure of rare genetic diseases through scientific research of excellence, selected according to the best practices shared internationally. Through a unique method in the Italian panorama, it follows the entire "research chain" dealing with fundraising, selection and funding of projects and the research activity itself carried out in the centers and laboratories of the Foundation. Telethon also develops collaborations with public health institutions and pharmaceutical industries to translate the results of research into therapies accessible to patients. Since its foundation, Telethon has invested more than 660 million euros in research, has funded 2,960 projects with 1,720 researchers involved and 630 diseases studied. To date, thanks to Fondazione Telethon, the first gene therapy with stem cells in the world has been made available, thanks to the collaboration with the pharmaceutical industry. This therapy is intended for the treatment of ADA-SCID, a severe immunodeficiency that compromises the body's defenses from birth. In 2023, Fondazione Telethon became responsible for the production and distribution of the drug to eligible patients in the European Union. Another gene therapy resulting from Telethon research made available is the one for a serious neurodegenerative disease, metachromatic leukodystrophy. This therapeutic approach is in an advanced stage of development for another immunodeficiency, Wiskott-Aldrich syndrome. Other diseases on which the gene therapy developed by Telethon researchers has been evaluated in patients are beta thalassemia and two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within the Telethon institutes a targeted therapeutic strategy is being studied or developed for other genetic diseases, such as hemophilia or various hereditary vision defects. In parallel, the study of basic mechanisms and potential therapeutic approaches for diseases still unanswered continues in all laboratories funded by Telethon. About Can-Fite BioPharma Ltd. Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CFBI) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion-dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company's lead drug candidate, Piclidenoson recently reported topline results in a Phase III trial for psoriasis and is expected to commence a pivotal Phase III. Can-Fite's liver drug, Namodenoson, is being evaluated in a Phase III trial for hepatocellular carcinoma (HCC), a Phase IIb trial for the treatment of MASH, and in a Phase IIa study in pancreatic cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company's third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,600 patients in clinical studies to date. For more information please visit: https://www.canfite.com/. Forward-Looking Statements This press release may contain forward-looking statements, about Can-Fite’s expectations, beliefs or intentions regarding, among other things, its product development efforts, business, financial condition, results of operations, strategies or prospects. All statements in this communication, other than those relating to historical facts, are “forward looking statements”. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to any resurgence of the COVID-19 pandemic and the war between Israel and Hamas; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the “Risk Factors” section of Can-Fite’s Annual Report on Form 20-F filed with the SEC on March 28, 2024 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contact Can-Fite BioPharma Motti Farbstein info@canfite.com +972-3-9241114

Results from the first four children to be treated have been detailed in a research paper. Credit: Tom Merton via Getty Images. MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born blind from birth gained significant vision improvements following treatment. The AAV-AIPL1 therapy is designed to address severe sight impairment caused by mutations in the AIPL1 gene. This condition is a form of retinal dystrophy that leads to severe visual impairment from birth, with affected individuals typically only able to distinguish between light and dark. The gene therapy involves injecting functional copies of the AIPL1 gene into the retina using an adeno-associated viral (AAV) vector, aiming to restore retinal cell function and prevent degeneration. Results from four of the 11 children were published in The Lancet on 21 February. The first four children treated were aged one to three years with severe retinal dystrophy linked to mutations in the AIPL1 gene. Each child received the therapy in one eye through subretinal injection. Outcome measures included visual acuity assessments, functional vision evaluations, visual evoked potentials, and retinal structure imaging. Before treatment, the children’s visual acuity was limited to light perception. At an average follow-up of three and a half years post-treatment, the treated eyes showed significant improvement, with visual acuity improving. In contrast, the untreated eyes’ visual acuity deteriorated to unmeasurable levels. Additionally, objective tests confirmed enhanced visual function and electrophysiological assessments indicated increased visual cortex activity specific to the treated eyes. Imaging revealed better preservation of retinal structure in treated eyes compared to untreated ones. MeiraGTx’s CEO Alexandria Forbes said: “These improvements extended outside the meaningful effects on vision and result in life-changing benefits in all areas of development including communication, behaviour, schooling, mood, psychological benefits and social integration.” Following these positive outcomes, a second cohort of seven children received treatment in both eyes. All 11 children treated with AAV-AIPL1 have so far exhibited meaningful visual improvements. One child experienced cystoid macular oedema in the treated eye, which partially improved over time and did not hinder the overall visual gains. No other significant safety concerns have been reported. In light of these findings, MeiraGTx has engaged in discussions with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit AAV-AIPL1 for expedited approval. The company is also in talks with the US Food and Drug Administration (FDA) to explore accelerated approval pathways in the US. If approved, AAV-AIPL1 would be eligible for a priority review voucher (PRV). While gene therapies hold promise for rare diseases, commercialisation remains a challenge. High development costs, complex manufacturing processes, and limited patient populations often deter investment. Earlier this month, Italian charity Telethon Foundation submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for a gene therapy targeting Wiskott-Aldrich syndrome after the withdrawal of its commercial partner. This marks the second time the foundation has stepped in to support an abandoned gene therapy, following its efforts with simoladagene autotemcel for ADA-SCID. The foundation claims to be the first charity worldwide to take responsibility for drug production and distribution, highlighting the financial and logistical hurdles in bringing gene therapies to market. The incidence of AIPL1-associated severe retinal dystrophy is estimated to be around one in every million live births. During a call with investors, Forbes stated that the company is exploring all options to ensure global access to AAV-AIPL1, noting “strategic interest” in the therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy