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Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development
NM3086 is an anti-P humanized monoclonal antibody that binds properdin with high affinity. NM3086 blocks both C3b and membrane-attack complex (MAC) formation, which being invetigated for for PNH patients.
AC-1101 (tofacitinib) topical gel phase I study patients with granuloma annulare. The oral formulation of tofacitinib has already been FDA approved to treat such ailments as rheumatoid arthritis.
TARA-002 is an investigational cell-based immunopotentiator, which is being investigated for the treatment of pediatric patients with lymphatic malformations (LMs).
ABBV-CLS-7262 is an eIF2B activator which targets eIF2B, a guanine nucleotide exchange factor that is essential for protein synthesis and a key regulator of the ISR. It is being investigated for vanishing white matter disease.
OMS906 is a humanized mAb which inhibits MASP-3, the key activator of the alternative pathway of complement, it is advancing in clinical programs for paroxysmal nocturnal hemoglobinuria (PNH), complement 3 (C3) glomerulopathy and other related indications.
The collaboration is for BPGbio’s BPM 31510 for Epidermolysis Bullosa Phase II/III trial. Debra of America will provide support to awareness, understanding, and key information towards patient recruitment and advocacy for the clinical trial.
RCT1100 is a first-in-class, mRNA-based genetic medicine for the treatment of people with primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene that encodes a protein essential for ciliary movement.
RLF-TD011 is a differentiated acid oxidizing solution of hypochlorous acid which shows antimicrobial action with anti-inflammatory properties, allowing infection control, reduction of wound colonization, alleviation of pain and itching and improved wound healing.
BB-301 is currently in development for Oculopharyngeal Muscular Dystrophy. BB-301 results in robust inhibition of mutant PABPN1 and concomitant replacement of the codon-optimized PABPN1 protein. The treatment restores muscle strength and muscle weight.
FT-001 is administered by injection into the subretinal space of the eye that delivers a functional copy of the human RPE65 gene. FT-001, for the treatment of Leber Congenital Amaurosis-2, a severe inherited retinal disease related to RPE65 gene mutation.
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