Gazi University

Charles River Laboratories, a Wilmington, Massachusetts-based contract development and manufacturing organization (CDMO), has entered a collaboration with Gazi University Faculty of Medicine, an academic medical institution based in Ankara, Turkey, to support plasmid DNA supply and in vitro efficacy studies for adeno-associated virus production. Under the agreement , Charles River will provide Gazi University with off-the-shelf, research-grade AAV plasmids to support early-stage gene therapy research targeting hyperphosphatemic tumoral calcinosis, a rare inherited disorder associated with elevated blood phosphate levels and calcium phosphate deposits in soft tissue. Gazi University researchers have identified GALNT3 as the gene responsible for the condition. The plasmids supplied through the collaboration are manufactured using animal component-free methods and are royalty-free, offering a pre-manufactured option designed to reduce development timelines and costs for early-stage gene therapy programs, according to the company. “We are excited to build a strong collaboration with Gazi University,” Kerstin Dolph, corporate senior vice president of global manufacturing at Charles River, said in a statement. “Leveraging cell and gene therapy CDMO expertise will enable the Faculty of Medicine to test and ultimately transform ground-breaking concepts into real-world therapies.” Gazi University said the collaboration brings together academic research and manufacturing expertise to advance rare disease gene therapy development. The agreement comes as Charles River continues to adapt its cell and gene therapy CDMO strategy amid a shifting market landscape. The company has been scaling platforms, investing in automation and standardized development approaches, and balancing client demand as biopharma companies rationalize pipelines and focus resources on the most viable advanced therapy programs. Charles River has said it remains focused on improving efficiency, yield and consistency in viral vector manufacturing through platforms such as its nAAVigation and Lentivation systems, while maintaining flexibility to support emerging modalities including nucleic acids and lipid nanoparticle-based therapies.

Cell and Gene Therapy CDMO to bolster rare disease research at Gazi University Faculty of Medicine WILMINGTON, Mass.--(BUSINESS WIRE)-- $CRL #CDMO --Charles River Laboratories International, Inc. (NYSE: CRL) and Gazi University Faculty of Medicine , today announced a gene therapy contract development and manufacturing organization (CDMO) collaboration, providing plasmid DNA for AAV production and in vitro efficacy studies. “We are excited to build a strong collaboration with Gazi University,” said Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River. “Leveraging cell and gene therapy CDMO expertise will enable the Faculty of Medicine to test and ultimately transform ground-breaking concepts into real-world therapies.” Hyperphosphatemic tumoral calcinosis (HTC) is a rare inherited disorder causing high blood phosphate and painful, non-cancerous calcium phosphate deposits in soft tissues, especially around joints like hips, shoulders, and elbows, leading to pain, inflammation, and movement issues. It is caused by genetic defects in regulating phosphate, and Gazi University has identified GALNT3 as the responsible gene. Charles River will supply Gazi University with off-the-shelf research-grade AAV plasmids , offering a pre-manufactured and ready-to-use option for early-stage gene therapy programs. These plasmids are produced with animal component-free methods and are royalty-free, designed to accelerate projects and reduce costs. “We are very happy to enter this strategic collaboration, bringing together academic innovation and CDMO expertise to accelerate the development of advanced therapies,” commented Prof. Fatih Ezgü, MD, Professor of Pediatrics and Head of the Department of Pediatric Genetics at Gazi University Faculty of Medicine. “This partnership marks a significant milestone in our mission to pioneer transformative therapies for rare diseases, and we look forward to the progress we can achieve together.” Cell and Gene Therapy CDMO Solutions Committed to streamlining the development of advanced therapies from concept to cure, Charles River has established an industry-leading cell and gene therapy portfolio, integrating discovery and safety assessment services, biologics testing, and pre-clinical through commercial-scale manufacturing solutions for plasmid DNA, viral vectors, and cell therapies. Explore the challenges and considerations for plasmid DNA throughout CGT development, from plasmid selection in drug discovery and future-proofing strategies, to navigating the translation to clinic and beyond. Access guidebook: About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . Contacts Charles River Investor Contact: Todd Spencer Corporate Vice President, Investor Relations 781.222.6455 todd.spencer@crl.com Charles River Media Contact: Amy Cianciaruso Corporate Vice President, Chief Communications Officer 781.222.6168 amy.cianciaruso@crl.com

ACI SANT'ANTONIO, Italy, July 22, 2024 /PRNewswire/ -- SIFI, a leading international ophthalmic company headquartered in Italy, is pleased to announce that the Committee for Orphan Medicinal Products (COMP) of the European Medicine Agency (EMA) has issued a positive opinion concerning Orphan Drug Designation (ODD) for polihexanide in the treatment of fungal keratitis. This designation follows the positive opinion of Committee for Human Medicinal Products (CHMP) adopted in May and the COMP positive opinion dated March 30 of this year for the medicinal product AKANTIOR® (polihexanide 0.08%) in the treatment of acanthamoeba keratitis. SIFI expects to receive the decision of the European Commission (EC) for marketing authorization in August. Polihexanide received an ODD in fungal keratitis from the US Food and Drug Administration (FDA) in February 2022. Fungal keratitis, a severe infection of the cornea caused by fungi such as Fusarium, Aspergillus, and Candida species, is a significant cause of ocular morbidity and unilateral blindness worldwide1. Although rare, this condition can lead to substantial vision loss if not treated promptly. Currently, no approved treatments exist for fungal keratitis in Europe, Japan, Asia, and most other regions globally. COMP's positive opinion concerning ODD for fungal keratitis is based on SIFI's in vitro, ex vivo and in vivo studies on standardized strains, demonstrating the efficacy of polihexanide against the most recurring species in fungal keratitis, such as Fusarium, Candida and Aspergillus. In vivo study were performed in collaboration with Gazi University (Ankara, Turkey). Additional studies on human ocular isolates are ongoing to potentially confirm the efficacy of polihexanide in fungal keratitis. SIFI plans to initiate clinical trials in patients affected by fungal keratitis in 2025. The company will seek Contract Research Organization (CRO) pitches for a Phase II clinical trial in Q4 2024. SIFI will focus its clinical development program on two major geographies, such as the USA and Europe. A potential CRO partner should demonstrate extensive experience in ophthalmology and rare diseases, and a global presence. "As we are preparing to launch AKANTIOR® (polihexanide 0.08%) in acanthamoeba keratitis across Europe, this COMP positive opinion concerning ODD for fungal keratitis reinforces our commitment to further invest on polihexanide and develop it in such rare disease where there is a high unmet medical need," said Fabrizio Chines, Chairman and CEO of SIFI. "We are eager to advance into the clinical development after encouraging preclinical data and double down on our efforts to develop therapeutic solutions for ophthalmic rare diseases." ABOUT SIFI: SIFI is a leading international ophthalmic company, headquartered in Italy, featuring an integrated business model, from research & development to manufacturing and commercialization both in pharmaceutical and biomedical sectors. Since our foundation in 1935, our mission is to improve people's lives through meaningful innovation in eye care. SIFI exports to more than 40 countries worldwide with a direct presence in major European markets, Mexico and, through joint ventures, in China and the United Arab Emirates. More information is available at . ABOUT AKANTIOR®: AKANTIOR® (polihexanide 0.08%) stands to become the first approved drug for the treatment of acanthamoeba keratitis in the world. It is an anti-amoebic polymer that acts on both the trophozoites and cysts of the protozoan acanthamoeba. It is formulated at a 0.8mg/ml (0.08%) concentration which makes it possible to administer as monotherapy eye drops in single-dose containers. The EC and the FDA granted SIFI Orphan Drug Designation in the treatment of acanthamoeba keratitis. CHMP opinion was received in May, EC decision is expected in August 2024. In addition, COMP confirmed the designated orphan medicinal product status at the time of marketing authorisation of Akantior (polihexanide). ABOUT FUNGAL KERATITIS: Fungal keratitis is a severe corneal infection that often results in blindness and eye loss. The disease is most prevalent in tropical and subtropical climates. According to The Lancet, annual global incidence of fungal keratitis is estimated at 1,051,787 cases, with the highest rates in Asia and Latin America. There are no approved treatments for fungal keratitis in Europe, Japan, Asia and almost anywhere else around the World. In addition to lack of approved medical alternatives, the incidence of fungal keratitis may be on the uptick due the global trend in general resistance to anti-fungal therapeutics. 1. AAO Fungal Keratitis – Europe EPIDEMIOLOGY Contact: Sabrina Zappia +39 3336999669 [email protected]