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¾È°ú¿ë ¼¼Æ÷Ä¡·á ¹× À¯ÀüÀÚ Ä¡·áCell and Gene Therapies in Ophthalmology |
ÇöÀç ¾È°ú¸¦ ÀûÀÀÁõÀ¸·Î ÇÏ´Â ¼¼Æ÷Ä¡·á ¹× À¯ÀüÀÚ Ä¡·áÁ¦(CGT)°¡ 8 Á¾·ù Ãâ½ÃµÇ°í ÀÖ½À´Ï´Ù. ÀÌ Áß Spark Therapeutics Luxturna(voretigene neparvovec)´Â ¸Á¸·»ö¼Òº¯¼ºÁõ(¸Á¸·¿°)°ú LCA(Leber congenital amaurosis)¸¦ ÀûÀÀÁõÀ¸·Î ÇÏ¸ç ¼¼°è¿¡¼ ÆÇ¸ÅµÇ°í ÀÖÀ¸¸ç, ¾È°ú ¿µ¿ª¿¡¼ Ãâ½ÃµÇ°í ÀÖ´Â À¯ÀÏÇÑ À¯ÀüÀÚ Ä¡·áÁ¦ÀÔ´Ï´Ù. ´Ù¸¥ ¸ðµç CGT´Â ¼¼Æ÷Ä¡·áÀÔ´Ï´Ù. Holostem Terapie Avanzate Holoclar´Â º¯¿¬ Áٱ⼼Æ÷ °á¼ÕÁõ(LSCD)À» ÀûÀÀÁõÀ¸·Î Çϸç À¯·´¿¡¼ ÆÇ¸ÅµÇ°í ÀÖ½À´Ï´Ù. FujifilmÀÇ Nepic, J.TecÀÇ Ocural, Hirosaki Lifescience InnovationÀÇ Sakracy´Â ¸ðµÎ LSCD¸¦ ÀûÀÀÁõÀ¸·Î Çϸç ÀϺ»¿¡¼ ÆÇ¸ÅµÇ°í ÀÖ½À´Ï´Ù. Aurion BiotechnologiesÀÇ Vyznova´Â °¢¸· ºÎÁ¾À» ÀûÀÀÁõÀ¸·Î Çϸç ÀϺ»¿¡¼ ÆÇ¸ÅµÇ°í ÀÖ½À´Ï´Ù. Reliance Life SciencesÀÇ ReliNethra´Â LSCD¸¦, ReliNethra C´Â ¾Ë·¹¸£±â¼º °á¸·¿°, ¾È°Ë °á¸·¿°, ÀÍ»óÆíÀ» ÀûÀÀÁõÀ¸·Î ÇÏ¸ç ¸ðµÎ Àεµ¿¡¼ ÆÇ¸ÅµÇ°í ÀÖ½À´Ï´Ù.
ÀϹÝÀûÀ¸·Î ½ÃÆÇµÇ°í ÀÖ´Â CGT´Â ´ëü Ä¡·á°¡ ¾ø´Â ÁßÁõ ¹× Èñ±Í ÁúȯÀ» ´ë»óÀ¸·Î Çϰí ÀÖ½À´Ï´Ù. °ü·Ã ¿¬±¸°³¹ß ºñ¿ë ¹× ȯÀÚ ¼ö°¡ ºñ±³Àû Àû±â ¶§¹®¿¡ 1°³´ç Ä¡·áºñ´Â »ó´çÈ÷ °í¾×ÀÔ´Ï´Ù. °¡°Ý°áÁ¤°ú »óȯÀº CGTÀÇ ÀáÀçÀû ÆÇ¸Å°í¸¦ Á¦ÇÑÇÒ °¡´É¼ºÀÌ ÀÖ½À´Ï´Ù.
CGT´Â 38°³ ¾È°ú ÀûÀÀÁõ¿¡ ´ëÇØ °³¹ßÁßÀ̸ç, ¹ß°ßºÎÅÍ µî·ÏÀü ´Ü°è±îÁö ¾à 500°³ÀÇ Àڻ꿡 °ÉÃÄ ÀÖ½À´Ï´Ù. CGT ÆÄÀÌÇÁ¶óÀÎ ÀÚ»êÀÌ ¸¹Àº ¾È°ú ÀûÀÀÁõ TOP 5´Â ³ëÀÎȲ¹Ýº¯¼º(AMD), ¸Á¸·¿°, ³ì³»Àå, LCA, ¸Á¸·ÁõÀÔ´Ï´Ù. ÆÄÀÌÇÁ¶óÀÎÀº ÀÓ»ó½ÃÇè ¾à(IND)/ÀÓ»ó½ÃÇè ½Åû(CTA), ÀüÀÓ»ó, Ž»ö µîÀÇ ÀǾàǰ °³¹ß ´Ü°è¿¡ ÁýÁߵǾî ÀÖ½À´Ï´Ù.
¼¼°èÀÇ ¾È°ú¿ë ¼¼Æ÷Ä¡·á ¹× À¯ÀüÀÚ Ä¡·á ½ÃÀåÀº ¿Ï¸¸ÇÑ ¼ºÀåÀ» ´Þ¼ºÇÒ °ÍÀ¸·Î ¿¹ÃøµÇ°í ÀÖÀ¸¸ç, 2023³â 4,600¸¸ ´Þ·¯¿¡¼ 2029³â¿¡´Â 30¾ï ´Þ·¯·Î Áõ°¡Çϸç, CAGRÀº 50.8%¿¡ ´ÞÇÒ °ÍÀ¸·Î º¸À̰í ÀÖ½À´Ï´Ù. À¯ÀüÀÚ Ä¡·á´Â ¾È°ú ¿µ¿ªÀÇ CGT ½ÃÀåÀ» µ¶Á¡ÇÒ °ÍÀ¸·Î ¿¹»óµË´Ï´Ù. À̰ÍÀº ÆÄÀÌÇÁ¶óÀο¡ ÀÖ´Â À¯ÀüÀÚ Ä¡·á ÀÚ»êÀÇ ¼ö°¡ ¸¹Àº °Í(ÃÑÀÚ»êÀÇ 74.6%)°ú ÆÇ¸Å Á¦Ç° Luxturna¸¦ º¸À¯ÇÑ Spark Therapeutics¿Í °°Àº °·ÂÇÑ Âü¿© ±â¾÷ÀÌ ÀÖ´Â °Í¿¡ ÀÇÇÕ´Ï´Ù.
¾È°ú¿ë ¼¼Æ÷Ä¡·á ¹× À¯ÀüÀÚ Ä¡·á ½ÃÀå¿¡ ´ëÇØ Á¶»çÇßÀ¸¸ç, ¾È°ú ÀûÀÀÁõ »óÀ§ 5¿¡ ÃÊÁ¡À» ¸ÂÃß°í ÀÖ½À´Ï´Ù. ¾È°ú ¿µ¿ª¿¡¼ ÁÖ¿ä CGT ¾àÁ¦ÀÇ °æÀï ±¸µµ, ½ÃÀå¿¡¼ÀÇ °úÁ¦¿Í ±âȸ, ÇâÈÄ Àü¸Á µîÀ» Á¤¸®ÇÏ¿© ÀüÇØµå¸³´Ï´Ù.
Currently, there are eight cell and gene therapies (CGTs) on the market for various ophthalmology indications. These comprise Spark Therapeutics' Luxturna (voretigene neparvovec), which is indicated for retinitis pigmentosa (retinitis) and Leber congenital amaurosis (LCA), is marketed globally, and is the only gene therapy on the market within the ophthalmology space. All other marketed CGTs are cell therapies. Holostem Terapie Avanzate's Holoclar is indicated for limbal stem cell deficiency (LSCD) and marketed in Europe. Fujifilm's Nepic, J.Tec's Ocural, and Hirosaki Lifescience Innovation's Sakracy are all indicated for LSCD and marketed in Japan. Aurion Biotechnologies' Vyznova is indicated for corneal edema and marketed in Japan. Reliance Life Sciences' ReliNethra is indicated for LSCD and ReliNethra C is indicated for allergic conjunctivitis, blepharoconjunctivitis, and pterygium, and both are marketed in India.
Generally, marketed CGTs target severe and rare diseases for which there are a lack of alternative treatments. The course per therapy is considerably high due to the associated R&D costs and the relatively small patient populations. Pricing and reimbursement could limit the potential sales revenue of CGTs.
Throughout the eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and India), CGT guidelines have been published covering: Chemistry, Manufacturing, and Controls (CMC), preclinical assessment, and clinical trials. The FDA provides a guidance specifically for the application of gene therapy for retinal disorders.
CGTs are in development for 38 ophthalmology indications and span almost 500 assets from discovery to pre-registration phases. The top five ophthalmology indications with the most CGT pipeline assets are age-related macular degeneration (AMD), retinitis, glaucoma, LCA, and retinopathy. The pipeline is concentrated towards the stages of drug development, such as investigational new drug (IND)/clinical trial application (CTA) filed, preclinical, and discovery.
GlobalData anticipates that the global CGT market value in the ophthalmology space will experience moderate growth. Sales are expected to increase from $46 million in 2023 to $3.0 billion in 2029, at a compound annual growth rate (CAGR) of 50.8%. Gene therapies are expected to dominate the CGT market in ophthalmology. This is attributed to the high number of gene therapy assets (74.6% of total assets) in the pipeline, and strong players such as Spark Therapeutics, with its marketed product Luxturna.
Overview of cell and gene therapies (CGT) in ophthalmology. The report focuses on the marketed CGT assets addressing ophthalmology indications as well as the top five ophthalmology indications with the most CGT pipeline assets. Clients will gain insight into the competitive landscape of leading CGT agents in ophthalmology including launch date projections, analyst consensus forecasts, likelihood of approval analysis, and commentary on current and future players. The report also includes outlook from three key opinion leaders in the 5EU, and US, and analyses key challenges and opportunities in the application of CGTs in ophthalmology. Additionally, the report includes commentary on the regulatory landscape of CGTs and on the reimbursement environment.