[JP Morgan] 바카라 가상 머니 사이트: “Prioritizing partnerships for MSC-based muscle disease treatment ‘EN001’”

[by Kang, In Hyo] "On December 18, 2024, the Australian pharmaceutical company Mesoblast received approval from the U.S. Food and Drug Administration (FDA) for 'Ryoncil (remestemcel-L)', a treatment for pediatric steroid-refractory acute graft-versus-host disease (SR-aGVHD). Ryoncil is a cell therapy based on 'mesenchymal stromal cells (MSC)', and as a result of this approval, the atmosphere at local meetings in the U.S. shifted, with more attention being paid to our primary pipeline, '바카라 가상 머니 사이트' (development code name)."

"This is the company's first time participating in this event," said Oh Choong-seob, Director of Strategic Planning at 바카라 가상 머니 사이트, during the '2025 J.P. Morgan Healthcare Conference', the world's largest pharmaceutical and biotechnology investment event held in San Francisco, USA, on January 15. "Approximately 70% of our activities were focused on licensing EN001, a muscle disease treatment candidate developed using umbilical cord-derived mesenchymal stem cells (MSCs), while the remaining 30% was devoted to holding various meetings aimed at establishing a contract development and manufacturing organization (CDMO) network for cell and gene therapy (CGT)."

Founded in March 2018 as a faculty startup by Professor Jang Jong-wook of Samsung Seoul Hospital, 바카라 가상 머니 사이트 made its debut on the KOSDAQ market in August last year through the technology special listing system. The company specializes in advanced biopharmaceutical CDMO service and next-generation CGT novel drug development, focusing on rare and incurable muscle diseases like Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), and sarcopenia. As a CGT CDMO and new drug development specialized company, 바카라 가상 머니 사이트 operates a sustainable business model that generates stable revenue from its CGT CDMO services, which is then reinvested in CGT new drug development.

"We conducted approximately 20 meetings through the 1-on-1 partnering platform at the JP Morgan Biotech Showcase (BTS), with Japanese companies showing the highest response rate," said Oh Choong-seob. "While 바카라 가상 머니 사이트 is a well-established modality (treatment approach) in Korea and Japan, it remains relatively unfamiliar in other countries."

He noted, however, "Foreign companies outside of Japan, particularly those focused on rare disease treatments, have shown interest in 바카라 가상 머니 사이트, our MSC-based muscle disease treatment candidate." He added, "We have reviewed and discussed the possibility of licensing-in (technology introduction) for 바카라 가상 머니 사이트 with a U.S.-based rare disease treatment development company."

바카라 가상 머니 사이트 is developing EN001 as a novel drug for CMT, DMD, and sarcopenia, all of which currently lack fundamental treatments. In Korea, these conditions have received approvals for clinical phase 1b, clinical phase 1/2, and clinical phase 1/2a trials, respectively. CMT and DMD are classified as rare diseases under the Korea Disease Control and Prevention Agency's 'Rare Disease Management Act'. EN001 was designated as an ‘orphan drug (ODD) in development’ for these conditions in April and May 2022.

At this year's JP Morgan Healthcare Conference, 바카라 가상 머니 사이트 concentrated on activities for the licensing-out (technology transfer) of its main pipeline, EN001. However, the company’s core business model revolved around its CGT CDMO business. "In the CGT CDMO industry, we engaged in discussion with Japanese companies interested in building manufacturing facilities and focused on establishing CGT CDMO networks and relationships with U.S.-based companies," Oh remarked.

"Unlike antibody CDMOs, CGT CDMO businesses involve a significant number of autologous cells, which operate in a 'multi-variety, small-quantity' production framework. This allows us to refer customers to CGT CDMOs in different countries, enabling collaboration and fostering mutual growth," Oh furt바카라 가상 머니 사이트r elaborated.

"바카라 가상 머니 사이트's core business area is CGT CDMO, which generates the majority of our revenue. By reinvesting a portion of these earnings into novel drug development, we benefit from significantly lower risks compared to companies solely focused on developing new drugs," he added.

In 2024, on an individual basis, 바카라 가상 머니 사이트 reported sales of KRW 5.1 billion (approximately USD 3.5 million) through the third quarter, reflecting a 4.2% increase compared to the same period of the previous year. The operating loss also decreased by 6.5%, from approximately KRW 10.9 billion in Q3, 2023, to KRW 10.1 billion in Q3, 2024. While sales are increasing, the deficit has been decreasing.

"While the CGT CDMO industry is challenging overall, this year, 바카라 가상 머니 사이트 will maintain strong CDMO sales and ensure continued growth in the company's revenue," Oh concluded. "Since 'process solidification' is crucial for CGT CDMO, we will dedicate ourselves to achieving this goal as well."

"Regarding 바카라 가상 머니 사이트, I believe it will progress smoothly if we adhere to the clinical timeline," Oh further emphasized. "Given the event leading up to this JP Morgan Healthcare Conference, where the first MSC-based treatment (GVHD indication) was approved and successfully commercialized, we focused on building a network by securing as many partnerships as possible at the event."