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Gildeuretinol

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Gildeuretinol

Retin-20,20,20-d3-ol

CAS118139-35-8

MF C20H272H3O, MW 289.5 g/mol

(2E,4E,6E,8E)-3-(2H3)methyl-7-methyl-9-(2,6,6-trimethylcyclohex-1-en-1-yl)nona-2,4,6,8-tetraen-1-ol; (20,20,20-2H3)retinol

(2E,4E,6E,8E)-7-methyl-3-(trideuteriomethyl)-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraen-1-ol
vitamin A analogue, Orphan Drug, Stargardt disease, breakthrough therapy, Pediatric Rare Disease designations, ALK-001, KL-49, ALK 001, KL 49

  • OriginatorColumbia University
  • DeveloperAlkeus Pharmaceuticals
  • ClassEye disorder therapies; Retinoids; Vitamins
  • Mechanism of ActionDimerisation inhibitors; Vitamin A replacements
  • Orphan Drug StatusYes – Stargardt disease
  • Phase II/IIIDry age-related macular degeneration
  • Phase IIStargardt disease
  • No development reportedRetinal dystrophies
  • 08 Sep 2025Gildeuretinol – Alkeus Pharmaceuticals receives Orphan Drug status for Stargardt disease in European Union
  • 09 Jan 2025Alkeus Pharmaceuticals announces intention to submit an NDA to US FDA for Stargardt disease in 2025
  • 09 Jan 2025Efficacy and adverse event data from phase II trial for Stargardt disease released by Alkeus Pharmaceuticals

Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals, Inc. for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is caused by a defect in the ABCA4 gene that clears toxic byproducts resulting from the dimerization of vitamin A. Gildeuretinol is new molecular entity designed to reduce the dimerization of vitamin A in the eye without affecting the visual cycle.[1]

Gildeuretinol has received breakthrough therapyorphan drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration.[2]

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References

  1.  Zaydon YA, Tsang SH (July 2024). “The ABCs of Stargardt disease: the latest advances in precision medicine”Cell & Bioscience14 (1) 98. doi:10.1186/s13578-024-01272-yPMC 11282698PMID 39060921.
  2.  Fitch J (22 November 2024). “Gildeuretinol for Stargardt disease receives Rare Pediatric Disease, Fast Track Designations”Contemporary Pediatrics.
Clinical data
Other namesALK-001, KL-49
Identifiers
IUPAC name
CAS Number118139-35-8
PubChem CID169490774
UNIIPSZ7W5NR24
KEGGD12713
ChEMBLChEMBL5314606
Chemical and physical data
FormulaC20H30D3O
Molar mass292.500 g·mol−1
3D model (JSmol)Interactive image
SMILES
InChI

/////////Gildeuretinol, vitamin A analogue, Orphan Drug, Stargardt disease, breakthrough therapy, Pediatric Rare Disease designations, ALK-001, KL-49, ALK 001, KL 49, PSZ7W5NR24


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DR ANTHONY CRASTO

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DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO Ph.D

DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with AFRICURE PHARMA, ROW2TECH, NIPER-G, Department of Pharmaceuticals, Ministry of Chemicals and Fertilizers, Govt. of India as ADVISOR, earlier assignment was with GLENMARK LIFE SCIENCES LTD, as CONSUlTANT, Retired from GLENMARK in Jan2022 Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 32 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 32 PLUS year tenure till date Feb 2023, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 100 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 100 Lakh plus views on dozen plus blogs, 227 countries, 7 continents, He makes himself available to all, contact him on +91 9323115463, email amcrasto@gmail.com, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 38 lakh plus views on New Drug Approvals Blog in 227 countries......https://newdrugapprovals.wordpress.com/ , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc He has total of 32 International and Indian awards

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