HONG KONG, Dec. 23, 2024 /PRNewswire/ -- A research led by Hong Kong Baptist University (HKBU) and the Shanghai Sixth People's Hospital Affiliated to School of Medicine at Shanghai Jiao Tong University (Shanghai Sixth People's Hospital) has discovered that an aptamer developed by HKBU can be used to treat X-linked hypophosphatemia (XLH), a rare bone disease. The aptamer, originally developed to treat osteogenesis imperfecta, has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the U.S. Food and Drug Administration (FDA).
XLH ("X" denotes X-chromosome, a sex-determining chromosome) is a rare bone disease characterised by hypophosphatemia (i.e., low phosphate in blood). It is caused by a mutation in the phosphate regulating endopeptidase homolog X-linked (PHEX) gene, and its inheritance pattern follows an X-linked dominant mode. The bone tissue of XLH patients cannot mineralise properly, thereby affecting the hardness of the bones. The cartilage between the ribs of children with XLH grows and connects like beads strung together. They also result in limb deformities and growth retardation. Adult patients experience symptoms such as osteomalacia, bone pain, changes in body shape, shorter stature and pseudo-fractures, leading to reduced mobility or even disability.
Professor Zhang Ge, Associate Dean (Research) of Chinese Medicine and Director of the Law Sau Fai Institute for Advancing Translational Medicine in Bone and Joint Diseases at HKBU, and Professor Zhang Zhenlin, Director of Osteoporosis and Bone Diseases of the Shanghai Sixth People's Hospital, along with Aptacure Therapeutics Ltd., have developed a long-acting sclerostin-loop3 oligonucleotide aptamer for the treatment of XLH. This aptamer has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the U.S. FDA for treatment of XLH earlier this year.
Researchers conducted serum tests on 51 patients with XLH from the Shanghai Sixth People's Hospital. The results showed that their serum sclerostin levels were approximately 4.5 times higher than those of healthy individuals of the same age and gender.
Sclerostin is a protein that inhibits bone growth while playing a protective role in the cardiovascular system. Genetic evidence shows that sclerostin deficiency significantly elevates serum phosphorus levels and increases bone mass in mice. However, the monoclonal sclerostin antibody currently used increases the risk of heart attacks, stroke and cardiovascular diseases.
The research team discovered that sclerostin inhibits bone formation and protects the cardiovascular system through different domains of the protein. When sclerostin loses the "loop3 domain", bone mass and strength increase, but its cardiovascular protective effect remains unaffected. The "loop3 domain" of sclerostin can therefore serve as a therapeutic molecular target to promote bone formation, with no safety concern in the cardiovascular system.
The researchers then screen out oligonucleotide aptamers Apc001 that can inhibit the sclerostin loop3. Animal experiments showed that Apc001 can promote bone formation without increasing cardiovascular risk. It can also significantly increase the blood phosphorus levels of mice with XLH. This is expected to provide a drug candidate for the precise treatment of XLH patients.
According to the regulations of the U.S. FDA, once a drug under development obtains Orphan Drug Designation, its subsequent research and development may allow for a reduction in the number of clinical trial samples, be exempt from new drug marketing authorisation fees, and enjoy seven years of market exclusivity. Pediatric Rare Disease Designation allows drugs to be reviewed with priority. Having been designated as both an orphan drug and a pediatric rare disease by the U.S. FDA, Apc001 can accelerate its clinical translation and is expected to benefit patients with XLH sooner.
Currently, the pilot scale production of Apc001 has been completed and is undergoing the preclinical toxicological assessment by a third party. Apc001 is scheduled to enter clinical trials in both Mainland China and the U.S.
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Aptamer developed by HKBU for treating rare bone disease 'X-linked hypophosphatemia' receives Orphan Drug Designation and Rare Pediatric Disease Designation by U.S. FDA
HONG KONG, Dec. 23, 2024 /PRNewswire/ -- CNN is gearing up for another unforgettable New Year's Eve celebration, leading its global audience into 2025 with music, laughter, and live coverage from around the world. At 7:00 AM ET, CNN kicks off its live reporting from various international cities as they ring in the new year in the Asia Pacific, with Kristie Lu Stout anchoring live from Victoria Harbor in Hong Kong starting at 9:00 AM ET/10:00 PM HKT to 12:00 PM ET/1:00 AM HKT. The festivities will culminate with Anderson Cooper and Andy Cohen co-hosting CNN's New Year's Eve Live with Anderson Cooper and Andy Cohen from New York City's Times Square for an eighth consecutive year.
CNN's line-up of international correspondents will also make appearances during the broadcast, including Lynda Kinkade from Luna Park in Sydney, Hanako Montgomery in glittering Tokyo, Mike Valerio from the iconic Bosingak Belfry in the heart of Seoul and Will Ripley overlooking the Chao Phraya River in Bangkok.
Joining our correspondents will be a star-studded line-up of guests from across the worlds of entertainment, fashion, arts and sports. Kristie Lu Stout has a full slate with Marie Kondo, Japanese "professional tidier", organization expert and Konmari founder, Japanese contemporary artist Takashi Murakami, Chinese-American fashion designer Vivienne Tam, the Philippines' double gold medalist, Carlos Yulo, viral Japanese dance troupe Avantgardey, Pakistani actor and filmmaker Usman Mukhtar, Indian-American comic Zarna Garg, and Panda keeper at Hong Kong's Ocean Park, Matt Leung.
In Australia, Lynda Kinkade chats with music legend Sting, Colin Hay from 80's band Men at Work, fashion designer Rebecca Vallance, and actress and voice of Chilli from Bluey, Melanie Zanetti.
Thailand will provide the backdrop as Will Ripley talks to international rapper, singer, dancer and style icon, LISA, Chinese-American double gold medalist in freestyle skiing, Eileen Gu, Pakistani actress Hania Aamir and Taiwanese-American drag performer and winner of season 16 of RuPaul's Drag Race, Nymphia Wind.
From Japan, Hanako Montgomery speaks with K-pop superstar, TAEYANG, Japanese comedian Naomi Watanabe and Japanese two-time Olympic gold medalist skateboarder, Yuto Horigome, while in Korea, Mike Valerio interviews Indonesian all-female rock trio, Voice of Baceport.
CNN's New Year's Eve Live with Anderson Cooper and Andy Cohen begins at 8:00 PM ET, with music, comedy and some surprise guests, including Curtis "50 Cent" Jackson, live from his residency at Planet Hollywood Resort & Casino in Las Vegas, Meghan Trainor, Shania Twain, Diplo, Lil Jon, Mickey Guyton who will perform "Imagine" live from Times Square, Patti LaBelle, and comedians Whitney Cummings, Amy Sedaris, Sasheer Zamata, Ziwe, Adam Devine, and more.
CNN's New Year's Eve coverage will stream live on Max for subscribers and on CNN.com, CNN connected TV and mobile apps for pay TV subscribers.
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CNN rings in 2025 from across the Asia Pacific, culminating with Anderson Cooper and Andy Cohen in New York
