Decorin gel don rage tabo (2019)

Domin 2023.

Ƙungiyar Kafa FIBRX Inc:
Mark de Souza, PhD

• Kafa Shugaba da Shugaba, Chromaderm, Inc. Mataki na 2 Mai hana PKC-beta na Topical don maganin ciwon huhu.
• Tsohon Shugaban Hukumar, PellePharm. Mataki na 3 mai hana bushiya na Topical don maganin ciwon basal cell nevus syndrome, wanda ya sami nasara nadi daga FDA.
• Co-kafa, tsohon shugaban kasa da Shugaba, Lotus Tissue Repair, ya samu ta Shire kan dala miliyan 320 domin recombinant collagen VII furotin maye far for rare kwayoyin fata cuta, dystrophic epidermolysis bullosa.
• Shekaru 15+ na farkon matakin ƙwarewar kasuwanci. VP Ci gaban Kasuwanci, Dyax. Ya jagoranci ƙungiyar ci gaban kasuwanci a Dyax tun daga 2003. An ƙirƙiri fiye da dala miliyan 200 a cikin tallafin da ba na dilutive ba don Dyax daga fasahar 50+ da ma'amalar lasisin samfur.

James W. Fordyce

• Kafa EVP da CFO, Chromaderm, Inc. Mataki na 2 na PKC-beta inhibitor don maganin ciwon huhu.
• Co-kafa, tsohon EVP da CFO, Lotus Tissue Repair, wanda Shire ya samu akan dala miliyan 320 don sake dawo da furotin na collagen VII don cututtukan fata na ƙwayoyin cuta, dystrophic epidermolysis bullosa.
• Wanda ya kafa, Prince Ventures LP> shekaru 25 na ƙwarewar saka hannun jari na kamfani. Mai alhakin yawancin saka hannun jari a matakin farko a kimiyyar rayuwa: Genentech, Inc., Applied Biosystems Inc., Centocor, Inc., Regeneron Pharmaceuticals, Inc., Dyax Corp.

Erkki Ruoslahti, MD, PhD

• Babban Farfesa da tsohon shugaban kasa, Sanford Burnham Medical Research Institute.
• Memba na National Academy of Sciences, Cibiyar Magunguna, AAAS, da EMBO.
• Wanda ya kafa Telios, aikin majagaba a cikin ilimin halitta na matrix, integrins, RGD peptides, decorin a cikin cututtukan fibrotic.

Ungiyar Shawara
Jean Tang, MD, PhD

• Farfesa na Dermatology, Jami'ar Stanford.
• Co-kafa, PellePharm.
• Ya jagoranci gwaji na asibiti da yawa a EB Research Clinic, Jami'ar Stanford.

Hal Landy, MD (Mai Ba da Shawarar Likitoci)

• Tsohon Mashawarcin Likita, Gyaran Nama na Lotus.
• Tsohon CMO, Enobia Pharma, ya jagoranci gwaji da yawa a Genzyme (ciki har da scleroderma), Serono.

Deborah Ramsdell (Mai Bayar da Shawara)

• Mai Ba da Shawarar Ka'ida, PellePharm, Chromaderm.
• Shekaru 30+ na shawarwarin tsari, gami da shekaru 10 a FDA-CBER.

Sheila Magil, PhD (Mataimakin CMC)

• Tsohon mashawarcin CMC, Gyaran Nama na Lotus.
• Babban Mashawarci, Masu Ba da Shawarar Fasaha ta BioProcess.

Fred Reno, PhD (Mai Ba da Shawarar Magungunan Magunguna na Farko da Toxicology)

• Pharmacology da Toxicology Advisor, PellePharm, Chromaderm.
• 30+ shekaru gwaninta.

Maganin rigakafin tabo kamar decorin, wanda ke da ingantaccen tsarin aiki, na iya samun yuwuwar inganta ingancin rayuwa ga marasa lafiya da DEB ta shafa ta hanyar rage tabo da ke hade da hawan keke na yau da kullun na kumburin fata da warkar da rauni.

Taken Aikin: Haɓaka Decorin na Topical Recombinant Decorin a matsayin Maganin Ƙunƙarar Taso don dystrophic epidermolysis bullosa.

Marasa lafiya da dystrophic epidermolysis bullosa (DEB), musamman wadanda ke da nau'in cutar (RDEB), suna da fata mai rauni sosai da mucosa na baka wanda ke haifar da fata mai tsanani da blisters na baki wanda ke warkarwa tare da tabo mai yawa. Yawancin marasa lafiya na RDEB suma suna fama da matsananciyar esophageal, da kwangila da haɗuwa da lambobi, wanda ke haifar da pseudosyndactyly, (misali, 'nakasar mitten' na hannaye da ƙafafu, wanda yatsu da yatsu suke haɗuwa).

Wannan bincike yana binciken yuwuwar wani nau'i na injiniya na furotin ɗan adam da ke faruwa a zahiri, decorin, azaman wakili na rigakafin tabo. Decorin yana taimakawa wajen samar da kulawa da haɓaka matrix na waje a cikin fata wanda ke ba da tallafi da ayyuka daban-daban ciki har da sadarwa tsakanin sel.

A baya, DEBRA UK ta tallafawa bincike ta Farfesa Zambruno (2013), wanda ya gano cewa TGF-β (mai canza girma factor), kwayar halitta wanda ke taimakawa wajen aikin kwayoyin halitta, ya zama kamar yana hade da mafi tsanani bayyanar cututtuka, yana haifar da tabo. A cikin wannan binciken kuma an gano cewa decorin ya hana matakan TGF-β. Wani binciken da aka yi game da maza tagwaye iri ɗaya tare da RDEB, ya nuna cewa an bayyana decorin kusan sau biyu a cikin tagwayen, wanda ke da matsakaicin scarring phenotype (halayen da ake gani a cikin jiki "hoton asibiti"), idan aka kwatanta da ɗan'uwansa tagwaye. wanda ke da mummunan tabo phenotype. Dukansu suna da maƙasudin matakan C7 (collagen 7). An yi hasashen cewa decorin na iya taimakawa wajen sarrafa ayyukan TGF-β wanda ke haifar da tabo.

Maganin rigakafin tabo kamar decorin, wanda ke da ingantaccen tsarin aiki, na iya samun yuwuwar inganta ingancin rayuwa ga marasa lafiya da DEB ta shafa ta hanyar rage tabo da ke hade da hawan keke na yau da kullun na kumburin fata da warkar da rauni.

Waɗannan bayanan kwanan nan sun jagoranci ƙungiyar FIBRX don yin la'akari da cewa ƙirar (gel) na ƙirar ɗan adam recombinant decorin (hrDecorin), wani nau'i na injiniyan furotin na asali, zai zama ingantaccen magani na rigakafin tabo don DEB (dukansu masu rinjaye da DEB mai koma baya). Wannan binciken zai fara aiwatar da binciken ko wani hrDecorin gel na zahiri zai rage tabo a cikin fata na DEB.

Babban manufar wannan mataki na bincike:

• Don ƙira, haɓakawa da gwada sauƙi, kwanciyar hankali, ƙirar gel na hrDecorin wanda ya dace da gwajin asibiti na ɗan adam.
• Gudanar da ilimin toxicology mai mahimmanci da nazarin aminci na farko don tallafawa ƙaddamar da aikace-aikacen Sabbin Magunguna (IND) zuwa Hukumar Abinci da Magunguna ta Amurka (FDA). Ana buƙatar IND don ci gaba zuwa gwajin asibiti na ɗan adam.

Bayar da kuɗin wannan aikin na farko zai ba da damar FIBRX don ci gaba da duk ayyukan da ake bukata don shigar da IND tare da FDA, Dermatology & Dental Division, wanda ke haifar da gudanar da gwaji na farko a cikin mutum na asibiti tare da tsarin gel na hrDecorin. FIBRX yana shirin tara kuɗi don wannan binciken na asibiti a nan gaba. A halin yanzu, aikin da ake buƙata na IND mai ba da izini na asibiti da ake buƙata yana samun tallafi daga wasu masu ba da kuɗi ciki har da Shirin PRMRP na Ma'aikatar Tsaro ta Amurka. Shawarar ta haɗu da ainihin buƙatun dabarun PRMPR, "maganin jiyya don haɓaka warkar da rauni a cikin EB" da "haɓaka sabbin hanyoyin kwantar da hankali don rage alamun EB da haɓaka ingancin rayuwa". Babban dabarun PRMPR jagora ne don taimakawa abokan aikin masana'antu suyi aiki don gano magunguna da jiyya don EB.

Domin 2023.