MPS I
Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH).
SiglionTherapeutics, Development of a Novel Encapsulated Non-Viral CellBased, BBB-Penetrant Therapy for MPS I.
REGENXBIO revealed some positive news on the clinical trials they are conducting for the treatment of severe MPS I with RGX-111
- RGX-111, a potential one-time gene therapy for MPS I, is well-tolerated across two dose levels, with no drug-related serious adverse events.
MPS II
Watch the presentation on MPS II Research Highights >>
Denali Therapeutics Announces Continued Progress in DNL310 (ETV:IDS) Program for MPS II (Hunter Syndrome) Supporting Planned Initiation of Phase 2/3 Clinical Trial.
Homology Medicines announces first presentation of data with HMI-203 In Vivo Gene Therapy development candidate for Hunter Syndrome.
REGENXBIO revealed some positive news on the clinical trials they are conducting for the treatment of MPS II (Hunter Syndrome) with RGX-121.
- RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels.
MPS III
Abeona Therapeutics Inc., a fully-integrated leader in gene and cell therapy, announced new positive data from two ongoing Phase 1/2 clinical trials of the company’s investigational AAV-based gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim data was presented in late-breaking platform oral presentations at the 17th Annual WORLDSymposium™. The presentation slides are available on the company’s website at www.abeonatherapeutics.com.
JCR Pharmaceuticals received the “New Treatment Award” for IZCARGO® (pabinafusp alfa or JR-141), a blood-brain barrier penetrating I2S enzyme approved in Japan last May for the treatment of MPS II. Global phase III study in USA, Brazil and Europe are about to start.
Chairman and President: Shin Ashida announced that European Commission (EC) has granted orphan drug designation to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type III A (MPS IIIA, or Sanfilippo type A). JR-441 is a blood-brain barrier (BBB)-penetrating form of recombinant heparan N-sulfatase that was developed using JCR’s proprietary J-Brain Cargo® BBB-penetrating technology.
Lysogene provides update on ffeicacy, safety and timelines of the AAVance phase 2/3 clinical trial with gene therapy LYS-SAF302 for the treatment of MPSIIIA.
MPS VI
SiglionTherapeutics, Development of a Novel Encapsulated Non-Viral CellBased Therapy for MPS VI.