Leukocyte adhesion deficiency

Rocket Pharmaceuticals Reports Third Quarter 2022 Financial Results and Highlights Recent Progress

Retrieved on: 
Thursday, November 3, 2022
Biotechnology, FDA, Health, Other Health, Pharmaceutical, Other Science, Research, General Health, Genetics, Fitness & Nutrition, Science, COVID-19, Clinical Trials, Cardiology, American Heart Association, Leadership, Morgan Stanley, Company, G&A, Danon disease, Private Securities Litigation Reform Act, Chemistry, Research, J. P. Morgan, Risk, Investment, CGMP, Society, Genetic disorder, FDA, Adult, Safety, Pyruvate kinase deficiency, Bone marrow failure, Conference, SVB, ASH, FA, COVID-19, PKD, Marketing, Gene editing, Leukocyte adhesion deficiency, CMC, Heart Failure Society of America, Medication, NASDAQ, LVV, Acquisition, Disease, Fanconi anemia, AHA, SEC, LV, Patient, AAV, Heart failure, Pharmaceutical industry, Medical device, Air cargo, Rocket

We are now laser focused on advancing Phase 2 pivotal study design and endpoint selection with FDA feedback expected this quarter.

Key Points: 
  • We are now laser focused on advancing Phase 2 pivotal study design and endpoint selection with FDA feedback expected this quarter.
  • Regarding our Pyruvate Kinase Deficiency (PKD) program, we also expect to share updated data from both adult patients at ASH.
  • Based on the data available, the Company expects feedback from the FDA on Phase 2 pivotal study design and endpoints this quarter.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Highlights Anticipated 2022 Milestones Across Lentiviral and AAV Gene Therapy Clinical Programs

Retrieved on: 
Tuesday, January 11, 2022
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, FA, Patient, PKD, Conference, CD18, Genetic disorder, Danon disease, Private Securities Litigation Reform Act, SEC, COVID-19, MBA, Safety, Risk, NASDAQ, LVV, Pyruvate kinase deficiency, Lists of diseases, Medication, Heart failure, Leukocyte adhesion deficiency, Fanconi anemia, Lifting, Marketing, AAV, Pharming, Bone marrow failure, Pharmaceutical industry, EU

The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.

Key Points: 
  • The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Receives EMA Priority Medicines (PRIME) Designation for RP-L201 Gene Therapy for Treatment of Leukocyte Adhesion Deficiency-I

Retrieved on: 
Monday, March 29, 2021
Oncology, Health, FDA, Genetics, Clinical trials, Pharmaceutical, Biotechnology, Branches of biology, Biology, Life sciences, Rare diseases, Biotechnology, European Medicines Agency, European Union, Leukocyte adhesion deficiency, Gene therapy, RP-L201, Leukocyte Adhesion Deficiency-I, Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-L201, the Companys investigational gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-L201, the Companys investigational gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).
  • PRIME designation was granted based on encouraging preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trial of RP-L201.
  • We are delighted that the EMA has awarded PRIME designation to RP-L201 for the treatment of LAD-I.
  • These medicines are considered priority medicines by the EMA and are intended to reach patients earlier.

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy Designation for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I

Retrieved on: 
Tuesday, March 9, 2021
Biotechnology, FDA, Health, Pharmaceutical, Clinical trials, Health sciences, Branches of biology, Life sciences, Emerging technologies, Drug discovery, Food and Drug Administration, Hematology, Gene therapy, Therapy, Leukocyte adhesion deficiency, Regenerative medicine, Clinical trial, Leukocyte Adhesion Deficiency-I, Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).
  • RMAT designation was granted based on encouraging preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trial of RP-L201.
  • RMAT designation was established to help expedite the development and approval of regenerative medicine products, including cell and gene therapies.
  • Rocket also holds Rare Pediatric (U.S.), Orphan Drug (U.S./EU), and Advanced Therapy Medicinal Product (EU) designations for the RP-L201 program.

Orpha Labs AG Initiates Compassionate Use Program for ORL-101 in Leukocyte Adhesion Deficiency Type II (LAD-II)

Retrieved on: 
Monday, February 8, 2021
Medical specialties, Rare diseases, Hematology, Leukocyte adhesion deficiency, LAD, Clinical medicine

BAAR, Switzerland, Feb. 8, 2021 /PRNewswire/ -- Orpha Labs AG today announced its compassionate use program to make ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) patients.

Key Points: 
  • BAAR, Switzerland, Feb. 8, 2021 /PRNewswire/ -- Orpha Labs AG today announced its compassionate use program to make ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) patients.
  • ORL-101 is a novel formulation of an ultra-pure L-fucose currently in development for the treatment of LAD II patients.
  • The Israeli Ministry of Health (MOH) approved the use of ORL-101 for LAD-II patients under Orpha Labs' compassionate use program.
  • Orpha Labs' Founder and Chief Executive OfficerAlp Bugra Basat, MD,said, "We are proud to announce this development.

Orpha Labs AG Initiates Compassionate Use Program for ORL-101 in Leukocyte Adhesion Deficiency Type II (LAD-II)

Retrieved on: 
Monday, February 8, 2021
Medical specialties, Rare diseases, Hematology, Leukocyte adhesion deficiency, LAD, Clinical medicine

BAAR, Switzerland, Feb. 8, 2021 /PRNewswire/ -- Orpha Labs AG today announced its compassionate use program to make ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) patients.

Key Points: 
  • BAAR, Switzerland, Feb. 8, 2021 /PRNewswire/ -- Orpha Labs AG today announced its compassionate use program to make ORL-101 available to physicians providing care for Leukocyte Adhesion Deficiency Type II (LAD-II) patients.
  • ORL-101 is a novel formulation of an ultra-pure L-fucose currently in development for the treatment of LAD II patients.
  • The Israeli Ministry of Health (MOH) approved the use of ORL-101 for LAD-II patients under Orpha Labs' compassionate use program.
  • Orpha Labs' Founder and Chief Executive OfficerAlp Bugra Basat, MD,said, "We are proud to announce this development.

Rocket Pharmaceuticals Presents Positive Clinical Data from its Fanconi Anemia and Leukocyte Adhesion Deficiency-I Programs at the 62nd American Society of Hematology Annual Meeting

Retrieved on: 
Monday, December 7, 2020
Health, Stem cells, Genetics, General Health, Pharmaceutical, Cardiology, Biotechnology, Medical specialties, Clinical medicine, Rare diseases, Integrins, Hematology, Clusters of differentiation, Leukocyte adhesion deficiency, Fanconi anemia, Integrin beta 2, Fanconi, LAD

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today presents updated interim data from its Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs at the 62nd American Society of Hematology (ASH) Annual Meeting.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today presents updated interim data from its Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs at the 62nd American Society of Hematology (ASH) Annual Meeting.
  • Dr. Shah continued, Additionally, we continue to see encouraging evidence of efficacy for RP-L201 for the treatment of LAD-I.
  • Patients have shown sustained CD18 expression of 23% to 40%, far exceeding the 4-10% threshold associated with survival into adulthood.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals to Hold Webcast for Investors Highlighting Data Presented at the 62nd American Society of Hematology Annual Meeting

Retrieved on: 
Wednesday, December 2, 2020
Cardiology, Stem cells, Biotechnology, Pharmaceutical, General Health, Health, Genetics, Clinical trials, Blood disorders, Medical specialties, Blood, Hematopathology, Anemias, Pyruvate kinase deficiency, Fanconi anemia, Pyruvate kinase, Fanconi, Kinase, Leukocyte adhesion deficiency

The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs.

Key Points: 
  • The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs.
  • Following the discussion, Rocket management and key opinion leaders will be available for a brief Q&A session.
  • The webcast replay will be available on the Rocket website following the completion of the call.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Cerecor Announces FDA Acceptance of Investigational New Drug Application for CERC-803 to Treat Leukocyte Adhesion Deficiency Type II

Retrieved on: 
Tuesday, December 1, 2020
Proteins, Medical specialties, Health, Hematology, Rare diseases, Leukocyte adhesion deficiency, Food and Drug Administration, Selectin, Investigational New Drug, LAD

and CHESTERBROOK, Pa., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug Application (IND) to study the use of CERC-803 to treat Leukocyte Adhesion Deficiency Type II (LAD II).

Key Points: 
  • and CHESTERBROOK, Pa., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug Application (IND) to study the use of CERC-803 to treat Leukocyte Adhesion Deficiency Type II (LAD II).
  • There are currently no FDA-approved therapies for patients suffering from LAD-II said H. Jeffrey Wilkins, MD, Chief Medical Officer of Cerecor.
  • LAD-II is caused by genetic mutations that result in deficiency in expression of surface adhesion molecule selectin, leading to defective leukocyte functions.
  • Cerecor is a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for rare and orphan diseases.

Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd American Society of Hematology Annual Meeting

Retrieved on: 
Wednesday, November 4, 2020
Biotechnology, Health, Stem cells, Clinical trials, Medical specialties, Clinical medicine, Branches of biology, Hematology, Stem cells, Fanconi anemia, Hematopoietic stem cell transplantation, FANCA, Pyruvate kinase deficiency, Leukocyte adhesion deficiency, Bone marrow failure, Fanconi

There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs as well as a poster presentation highlighting preliminary clinical data from the Pyruvate Kinase Deficiency (PKD) program.

Key Points: 
  • There will be two oral presentations highlighting clinical data from the Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) programs as well as a poster presentation highlighting preliminary clinical data from the Pyruvate Kinase Deficiency (PKD) program.
  • The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life.
  • Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning.
  • Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair.