Congenital disorders

Saniona Receives U.S. FDA Orphan Drug Designation for Tesomet in Hypothalamic Obesity

Retrieved on: 
Monday, July 26, 2021
Health, Clinical medicine, Congenital disorders, Pharmaceuticals policy, Amphetamine, Bariatrics, Body shape, Obesity, Appetite, Prader–Willi syndrome, Orphan drug

Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of hypothalamic obesity (HO).

Key Points: 
  • Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of hypothalamic obesity (HO).
  • Saniona is proud to be pioneering a regulatory path forward for people living with HO, and we are thrilled to have received the first-ever FDA orphan drug designation in HO.
  • Wood Foundation and parent of a child living with hypothalamic obesity, commented, The recognition of the first orphan drug designation in hypothalamic obesity is a critical milestone for the HO community.
  • Saniona is advancing Tesomet for hypothalamic obesity and Prader-Willi syndrome, two severe rare disorders characterized by obesity and loss of appetite control.

Saniona Launches New Video Series to Highlight the Impact of Prader-Willi Syndrome and Hypothalamic Obesity on Patients and Caregivers

Retrieved on: 
Monday, May 10, 2021
Clinical medicine, Health, Medicine, Congenital disorders, Amphetamine, Bariatrics, Body shape, Nutrition, Obesity, Prader–Willi syndrome, Prader

The company\xe2\x80\x99s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain.

Key Points: 
  • The company\xe2\x80\x99s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain.
  • Ultimately CP survivors with hypothalamic injury report at least three times higher 20-year mortality than CP survivors without hypothalamic injury.
  • Many HO patients are treated with approaches used for general obesity such as surgery, medication and lifestyle counseling, but these are often ineffective.
  • It occurs most often in children and older adults, creating a burden for both patients and families.\n'

Saniona to Present at Multiple Upcoming Investor Conferences

Retrieved on: 
Monday, April 26, 2021
Health, Branches of biology, Clinical medicine, Congenital disorders, Prader

The company\xe2\x80\x99s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain.

Key Points: 
  • The company\xe2\x80\x99s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain.
  • Saniona\xe2\x80\x99s robust drug discovery engine has generated a library now consisting of more than 20,000 proprietary modulators of ion channels, a significantly untapped drug class that is scientifically validated.
  • Lead candidate SAN711 is entering Phase 1 for rare neuropathic disorders, with SAN903 for rare inflammatory and fibrotic disorders advancing through preclinical development.
  • Led by an experienced scientific and operational team, Saniona has an established research organization in Copenhagen, Denmark and is building its corporate office in the Boston, Massachusetts area, U.S.

Saniona Announces Partnership with the Foundation for Prader-Willi Research (FPWR) to Support its Prader-Willi Syndrome (PWS) Clinical Trial with Tesomet

Retrieved on: 
Monday, April 19, 2021
Clinical medicine, Branches of biology, Medicine, Genetic syndromes, Congenital disorders, Genodermatoses, Prader–Willi syndrome, Appetite, Polyphagia, Prader, Obesity

\xe2\x80\x9cWe look forward to working with FPWR to gather this feedback and to increase awareness of our clinical trial.\xe2\x80\x9d\nSaniona\xc2\xa0previously evaluated Tesomet in a randomized, double-blind, placebo-controlled Phase 2a trial in adults and adolescents with PWS.

Key Points: 
  • \xe2\x80\x9cWe look forward to working with FPWR to gather this feedback and to increase awareness of our clinical trial.\xe2\x80\x9d\nSaniona\xc2\xa0previously evaluated Tesomet in a randomized, double-blind, placebo-controlled Phase 2a trial in adults and adolescents with PWS.
  • Adult patients receiving Tesomet achieved a statistically significant reduction in hyperphagia, as well as a clinically meaningful reduction in body weight at a dose of 0.5 mg per day.
  • The company\xe2\x80\x99s lead product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, severe rare disorders characterized by uncontrollable hunger and intractable weight gain.
  • Saniona is advancing Tesomet for hypothalamic obesity and Prader-Willi syndrome, two severe rare disorders characterized by obesity and loss of appetite control.

DGAP-News: Biotest AG: Biotest accelerates fibrinogen phase III study with additional patient group

Retrieved on: 
Wednesday, April 7, 2021
Blood, Coagulation system, Anatomy, Acute-phase proteins, Blood proteins, Congenital disorders, Fibrinogen, Factor I deficiency, Fibrin

In order to compensate the fibrinogen deficiency and to effectively stop bleeding, Biotest has developed the fibrinogen concentrate BT524.

Key Points: 
  • In order to compensate the fibrinogen deficiency and to effectively stop bleeding, Biotest has developed the fibrinogen concentrate BT524.
  • The AdFIrst study is a prospective, randomized, active-controlled, multicenter phase III study that investigates the efficacy and tolerability of the fibrinogen concentrate BT524 in patients with acquired fibrinogen deficiency.
  • With the AdFIrst study in acquired fibrinogen deficiency, Biotest is further expanding its development program for the fibrinogen concentrate BT524.
  • The AdFIrst study is a prospective, active-controlled, multicentre phase III study investigating the efficacy and safety of the fibrinogen concentrate BT524 in patients with acquired fibrinogen deficiency.

BT524: A Human Fibrinogen Concentrate Purified from Human Plasma - Global Emerging Insight and Market Forecast Report 2021-2030 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 16, 2021
Health, Biotechnology, Blood, Congenital disorders, Anatomy, Branches of biology, Business intelligence, Strategic management, Fibrinogen, Factor I deficiency, SWOT analysis, Market

The "BT524 - Emerging Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "BT524 - Emerging Insight and Market Forecast - 2030" report has been added to ResearchAndMarkets.com's offering.
  • BT524 is a human fibrinogen concentrate purified from human plasma.
  • "BT524- Emerging Insight and Market Forecast - 2030" the report provides comprehensive insights about an investigational product for Hypofibrinogenemia in 7 Major Markets.
  • Further, it also consists of future market assessments inclusive of the market forecast, SWOT analysis, market competitors, and other emerging therapies.

Saniona Announces Oral Presentation of Tesomet Data in Hypothalamic Obesity at ENDO 2021

Retrieved on: 
Monday, March 15, 2021
Branches of biology, Health, Clinical medicine, Congenital disorders, Appetite, Endocrinology, Rare diseases, Prader–Willi syndrome, Obesity, Prader, Endocrine system, Hypothalamus

Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that data from its Phase 2 clinical trial of Tesomet for hypothalamic obesity (HO) will be presented at the Endocrine Societys 2021 Annual Meeting (ENDO 2021), which is being held virtually March 20-23, 2021.

Key Points: 
  • Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that data from its Phase 2 clinical trial of Tesomet for hypothalamic obesity (HO) will be presented at the Endocrine Societys 2021 Annual Meeting (ENDO 2021), which is being held virtually March 20-23, 2021.
  • There is currently no medicine approved for hypothalamic obesity, a rare disease characterized by intractable weight gain and complicated by uncontrollable hunger.
  • The companys lead product candidate, Tesomet, is in mid-stage clinical trials for the rare diseases Prader-Willi syndrome and hypothalamic obesity.
  • Saniona is advancing Tesomet for hypothalamic obesity and Prader-Willi syndrome, two severe rare disorders characterized by obesity and loss of appetite control.

Sio Gene Therapies to Participate in Upcoming Investor & Scientific Conferences

Retrieved on: 
Thursday, March 11, 2021
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Congenital disorders, Applied genetics, Sandhoff disease, Tay–Sachs disease, Gene therapy, Therapy

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Saniona Receives Feedback from U.S. FDA Providing a Regulatory Path Forward for Tesomet in Hypothalamic Obesity

Retrieved on: 
Monday, March 8, 2021
Health, Clinical medicine, Branches of biology, Congenital disorders, Amphetamine, Bariatrics, Body shape, Nutrition, Obesity, Appetite, Prader–Willi syndrome, Prader

Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced it received feedback from the U.S. Food and Drug Administration (FDA) providing further clarity on a regulatory path for Tesomet in the treatment of hypothalamic obesity (HO).

Key Points: 
  • Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced it received feedback from the U.S. Food and Drug Administration (FDA) providing further clarity on a regulatory path for Tesomet in the treatment of hypothalamic obesity (HO).
  • Saniona previously announced that the FDA had highlighted the potential for off-label use of Tesomet in the general obese population.
  • The companys lead product candidate, Tesomet, is in mid-stage clinical trials for the rare diseases Prader-Willi syndrome and hypothalamic obesity.
  • Saniona is advancing Tesomet for hypothalamic obesity and Prader-Willi syndrome, two severe rare disorders characterized by obesity and loss of appetite control.

Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference

Retrieved on: 
Tuesday, February 16, 2021
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Congenital disorders, Medical genetics, Sandhoff disease, Tay–Sachs disease, Gene therapy, Therapy

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.