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QR-1123

QR-1123 is a first-in-class investigational RNA-based oligonucleotide designed to treat autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin (_RHO_) gene.[L46782]

Bifidobacterium Longum Infantis 35624

Bifidobacterium Longum Infantis 35624 is a probiotic being investigated for the treatment of gastrointestinal disorders, such as Crohn's disease and irritable bowel syndrome.[A259872, A259877]

AAV-dXIAP

AAV-dXIAP is a recombinant adeno-associated virus-encoded gene for X-linked mammalian inhibitor of apoptosis protein (XIAP) developed by Neurologix.

Gallium citrate

Prademagene zamikeracel

[L53008] COL7A1 encodes type VII collagen (C7), which is a major component of anchoring fibrils (AF) that are critical for dermal-epidermal cohesion. … Prademagene zamikeracel is a sheet-based gene therapy comprising autologous cells isolated from skin punch biopsies of patients with mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

CEQ508

CEQ508 is a Live Attenuated E. Coli Expressing Beta Catenin ShRNA currently being investigated to treat Familial Adenomatous Polyposis.

Atinumab

ATI-355 is a humanized anti-Nogo-A antibody developed by Novartis.

TTAX01

TTAX01 is a cryopreserved human umbilical cord product being developed by TissueTech.[L47246]

DaxibotulinumtoxinA

It is a botulinum toxin without accessory proteins purified from the bacterium _Clostridium botulinum_ type A,[L47840] the gram-positive anaerobic bacterium primarily present in soil. … [L47870] DAXI or DAXXIFY, a product of daxibotulinumtoxinA, incorporates RTP004, a proprietary synthetic stabilizing peptide for enhanced product stability.[A261080, A261085]

Lexanersen

Lexanersen is an antisense oligonucleotide under investigation for use as a disease-modifying therapy in Huntington's disease.