XellSmart Secures Fourth FDA and NMPA Clearance for iPSC-Derived Cell Therapy Targeting MSA-P
XellSmart receives US FDA and China NMPA clearance for Phase I/II trials of its iPSC-derived neuron progenitor therapy for MSA-P, expanding its CNS pipeline spanning PD, ALS and spinal cord injury.
Amyotrophic Lateral Sclerosis | 03/03/2026 | By News Bureau | 203
VectorY Doses First Patient in Phase I/II PIONEER-ALS Trial of TDP-43 Targeting Therapy VTx-002
VectorY initiates clinical evaluation of VTx-002, a first-in-class vectorised antibody therapy targeting TDP-43 pathology in ALS, with first patient dosed at Mass General Brigham.
Amyotrophic Lateral Sclerosis | 11/02/2026 | By News Bureau | 189
Under the terms of the partnership, Transition Bio is responsible for the discovery and optimisation of small molecules targeting TDP-43 until nomination of a development candidate, upon which Voyager will have an option to license the worldwide exclusive rights to develop and commercialise the programme.
Amyotrophic Lateral Sclerosis | 15/11/2025 | By Dineshwori | 289
Ractigen Therapeutics Doses First Patient in Phase I Clinical Trial for RAG-17 in SOD1-ALS
RAG-17 received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) in March 2023, followed by the FDA's clearance of its Investigational New Drug (IND) application.
Amyotrophic Lateral Sclerosis | 26/12/2024 | By Aishwarya | 563
MTPA Signs Deal with Dewpoint to Advance Small Molecule Condensate Modulator
The collaboration focuses on developing a novel TDP-43 small molecule condensate modulator (c-mod) for amyotrophic lateral sclerosis (ALS), furthering MTPC's commitment to advancing innovation in ALS and central nervous system (CNS) diseases.
Amyotrophic Lateral Sclerosis | 19/12/2024 | By Aishwarya | 320
Ractigen Therapeutics Secures FDA Orphan Drug Designation for RAG-21
RAG-21 is an siRNA therapy specifically designed to target FUS-ALS, one of the most aggressive subtypes of ALS.
Amyotrophic Lateral Sclerosis | 20/11/2024 | By Aishwarya | 661
Gland Pharma Receives Nod from US FDA for Rare Muscle Disorder
Gland Pharma Ltd. has received approval from the US health regulator for its generic Edaravone injection to treat amyotrophic lateral sclerosis.
Amyotrophic Lateral Sclerosis | 08/05/2024 | By Aishwarya | 883
MTPA Presents Healthcare Resource Utilization Data in RADICAVA ORS at AMCP 2024
Mitsubishi Tanabe Pharma America, Inc. (MTPA) has recently presented one abstract on amyotrophic lateral sclerosis (ALS) at the Academy of Managed Care Pharmacy (AMCP) 2024 Annual Meeting.
Amyotrophic Lateral Sclerosis | 16/04/2024 | By Aishwarya | 488
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