Clinical Trials
40
14 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1975
Active, not recruiting
11
27.5%
Completed
17
42.5%
Enrolling By Invitation
1
2.5%
Not yet recruiting
2
5.0%
Recruiting
9
22.5%
No approval data available
- MEDIPAL and JCR announced the first participant has been dosed in the global Phase I/II trial of JR-446 for mucopolysaccharidosis type IIIB, also known as Sanfilippo syndrome type B. - JR-446 is a blood-brain barrier-penetrating alpha-N-acetylglucosaminidase developed by JCR using its proprietary J-Brain Cargo technology, with MEDIPAL holding commercialization rights outside Japan. - MPS IIIB is an ultra-rare lysosomal storage disorder affecting an estimated 500 to 1,000 people worldwide, with no approved treatment currently available. - The open-label trial enrolls patients under six years of age, while a separate Phase I/II trial continues in Japan and JR-446 holds orphan drug designation in the US, EU and Japan.
- Mesoblast drew US$50 million from a five-year non-dilutive credit facility provided by shareholder and director Dr. Gregory George, with cash reserves of US$122 million as of March 30, 2026. - The facility carries a fixed 8.00% annual interest rate with a five-year interest-only period, secured solely by the Temcell royalty, and includes no early prepayment or exit fees. - Proceeds will retire the higher-cost NovaQuest Capital Management LLC debt facility, eliminating short-term debt obligations and optimizing the company's capital structure. - CEO Silviu Itescu stated the facility does not encumber material assets or intellectual property, preserving flexibility for strategic partnerships and licensing transactions.
