Rare diseases

LEXEO Therapeutics Receives Rare Pediatric Disease Designation and Orphan Drug Designation for LX1004 for the Treatment of CLN2 Batten Disease

Retrieved on: 
Wednesday, July 28, 2021

Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.

Key Points: 
  • Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS), said R. Nolan Townsend, Chief Executive Officer of LEXEO Therapeutics.
  • The FDA grants Rare Pediatric Disease designation for serious and life-threateningdiseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
  • CLN2 Batten disease is an autosomal recessive lysosomal storage disease with approximately 1,000 cases worldwide.
  • LEXEOs current pipeline consists of adeno-associated virus (AAV)-mediated gene therapies in rare cardiac diseases, CLN2 Batten disease, and APOE4-associated Alzheimers disease.

Hypoparathyroidism Epidemiology Research Report 2021-2030 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, July 28, 2021

This 'Hypoparathyroidism Epidemiology Forecast-2030' report delivers an in-depth understanding of the Hypoparathyroidism, historical and forecasted epidemiology as well as the Hypoparathyroidism trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Key Points: 
  • This 'Hypoparathyroidism Epidemiology Forecast-2030' report delivers an in-depth understanding of the Hypoparathyroidism, historical and forecasted epidemiology as well as the Hypoparathyroidism trends in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
  • Among the major types of hypoparathyroidism, i.e., Chronic and transient hypoparathyroidism, the former one accounts for a higher number of prevalent cases of hypoparathyroidism.
  • The report assesses the disease risk and burden and highlights the unmet needs of Hypoparathyroidism.
  • The report provides the segmentation of the disease epidemiology for 7MM by Total Prevalent Cases of Hypoparathyroidism, Diagnosed Prevalent Cases of Hypoparathyroidism, Gender-specific Diagnosed Prevalent Cases of Hypoparathyroidism, Type-specific Diagnosed Prevalent Cases of Hypoparathyroidism, Cause-specific Diagnosed Prevalent Cases of Hypoparathyroidism, and Age-specific Diagnosed Prevalent Cases of Hypoparathyroidism.

Abeona Therapeutics Announces New MRI Data Showing Increased Brain Volume in Young Patients with Sanfilippo Syndrome Type A (MPS IIIA) After Treatment with ABO-102 Gene Therapy

Retrieved on: 
Monday, July 26, 2021

The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.

Key Points: 
  • The new data was presented during an oral presentation at the 16th International Symposium on MPS and Related Diseases.
  • Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability.
  • Secondary endpoints include brain volume, behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and liver volume.
  • The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA).

Health Canada releases What We Heard Report from the Public Engagement on the National Strategy for Drugs for Rare Diseases

Retrieved on: 
Monday, July 26, 2021

From January 27 to March 26, 2021, Health Canada undertook public and stakeholder engagement to seek ideas and views on what a national strategy could look like.

Key Points: 
  • From January 27 to March 26, 2021, Health Canada undertook public and stakeholder engagement to seek ideas and views on what a national strategy could look like.
  • Health Canada will use the What We Heard Report to closely review the ideas and suggestions gathered from the consultations to inform the development of the national strategy.
  • In January 2021, Health Canada launched a national online engagement to let Canadiansespecially patients with rare diseases, their families and other interested stakeholdersshare their views and ideas for a national strategy.
  • This includes working with willing provinces, territories and stakeholders to establish a national strategy for drugs for rare diseases.

Eiger BioPharmaceuticals to Host Conference Call for Second Quarter 2021 Financial Results and Business Update on Thursday, August 5

Retrieved on: 
Thursday, July 22, 2021

PALO ALTO, Calif., July 22, 2021 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc.(Nasdaq: EIGR), a commercial-stage biopharmaceutical company focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that it will host a conference call onThursday, August 5, 2021at4:30 PM ET to discuss its financial results and provide a business update for the second quarter 2021.

Key Points: 
  • PALO ALTO, Calif., July 22, 2021 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc.(Nasdaq: EIGR), a commercial-stage biopharmaceutical company focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that it will host a conference call onThursday, August 5, 2021at4:30 PM ET to discuss its financial results and provide a business update for the second quarter 2021.
  • Eigeris a commercial-stage biopharmaceutical company focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases.
  • Lonafarnib is a first-in-class, oral prenylation inhibitor in a global Phase 3 trial.
  • Zokinvyfor the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient progeroid laminopathies is the Company's first FDA approved product.

Zogenix to Release Second Quarter 2021 Financial Results and Host Conference Call and Webcast on August 5

Retrieved on: 
Thursday, July 22, 2021

Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The companys first rare disease therapy, FINTEPLA (fenfluramine) oral solution, has been approved by the U.S. FDA and the European Medicines Agency and is in development in Japan for the treatment of seizures associated with Dravet syndrome, a rare, severe lifelong epilepsy.
  • The company has two additional late-stage development programs: one in a rare epilepsy called Lennox-Gastaut syndrome and one in a mitochondrial disease called TK2 deficiency.
  • Zogenix also plans to initiate a study of FINTEPLA in a genetic epilepsy called CDKL5 Deficiency Disorder (CDD) and is collaborating with Tevard Biosciences to identify and develop potential next-generation gene therapies for Dravet syndrome and other genetic epilepsies.

Novo Holdings co-leads Hemab's US$ 55M Series A to Advance Next Generation Therapeutics for Bleeding and Thrombosis Disorders

Retrieved on: 
Thursday, July 22, 2021

Dr Sorensen brings a wealth of patient-centred R&D experience in many therapeutic areas including thrombosis and hemostasis.

Key Points: 
  • Dr Sorensen brings a wealth of patient-centred R&D experience in many therapeutic areas including thrombosis and hemostasis.
  • The pipeline will initially focus on underserved people living with rare bleeding disorders such asGlanzmann's Thrombasthenia (GT), with plans to expand intomore common disorders of hemostasis and thrombosis.
  • Jrgen Sberg Petersen, Partner at Novo Holdings, and Camilla Petrycer Hansen, Principal at Novo Seeds will continue as Chairman and Independent Director on the Board.
  • Hemab is an emerging biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders.

Hemab Raises US$ 55M Series A to Advance Next Generation Therapeutics for Bleeding and Thrombosis Disorders

Retrieved on: 
Thursday, July 22, 2021

COPENHAGEN, Denmark and BOSTON, July 22, 2021 /PRNewswire/ --Hemab ApS ("Hemab"), a biotech company developing next generation therapeutics for serious underserved bleeding and thrombosis disorders, today announces the successful closing of a US$ 55M Series A financing.

Key Points: 
  • COPENHAGEN, Denmark and BOSTON, July 22, 2021 /PRNewswire/ --Hemab ApS ("Hemab"), a biotech company developing next generation therapeutics for serious underserved bleeding and thrombosis disorders, today announces the successful closing of a US$ 55M Series A financing.
  • The pipeline will initially focus on underserved people living with rare bleeding disorders such as Glanzmann's Thrombasthenia (GT),with plans to expand into more common disorders of hemostasis and thrombosis.
  • This financing will enable us to accelerate the development of innovative therapeutics for long underserved patients with serious bleeding and thrombosis disorders.
  • Hemab is an emerging biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders.

Novo Holdings co-leads Hemab's US$ 55M Series A to Advance Next Generation Therapeutics for Bleeding and Thrombosis Disorders

Retrieved on: 
Thursday, July 22, 2021

COPENHAGEN, Denmark and BOSTON, July 22, 2021 /PRNewswire/ -- Novo Holdings, a leading international life science investor, today announces that it has co-led alongside HealthCap and RA Capital Management a US$ 55M Series A financing in Hemab ApS ("Hemab"), a biotech company developing next generation therapeutics for serious underserved bleeding and thrombosis disorders.

Key Points: 
  • Dr Sorensen brings a wealth of patient-centred R&D experience in many therapeutic areas including thrombosis and hemostasis.
  • The pipeline will initially focus on underserved people living with rare bleeding disorders such asGlanzmann's Thrombasthenia (GT), with plans to expand intomore common disorders of hemostasis and thrombosis.
  • Jrgen Sberg Petersen, Partner at Novo Holdings, and Camilla Petrycer Hansen, Principal at Novo Seeds will continue as Chairman and Independent Director on the Board.
  • Hemab is an emerging biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders.

Hemab Raises US$ 55M Series A to Advance Next Generation Therapeutics for Bleeding and Thrombosis Disorders

Retrieved on: 
Thursday, July 22, 2021

COPENHAGEN, Denmark and BOSTON, July 22, 2021 /PRNewswire/ -- Hemab ApS ("Hemab"), a biotech company developing next generation therapeutics for serious underserved bleeding and thrombosis disorders, today announces the successful closing of a US$ 55M Series A financing. The investment was led by Novo Holdings, HealthCap and RA Capital Management.

Key Points: 
  • The pipeline will initially focus on underserved people living with rare bleeding disorders such as Glanzmann's Thrombasthenia (GT),with plans to expand into more common disorders of hemostasis and thrombosis.
  • The financing will also enable Hemab to further build the team and expand its operational footprint in Denmark and the US.
  • This financing will enable us to accelerate the development of innovative therapeutics for long underserved patients with serious bleeding and thrombosis disorders.
  • Hemab is an emerging biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders.