Nusinersen

Cell Line Development Market Research Report 2023-2030 Featuring Key Players - Thermo Fisher Scientific, Corning, Lonza, Creative BioLabs., WuXi PharmaTech, Sartorius, Merck, Berkeley Lights, Danaher - ResearchAndMarkets.com

Retrieved on: 
Thursday, December 7, 2023
Health, Pharmaceutical, CLD, Research, Immortalised cell line, GMP, Thermo Fisher Scientific, Fujifilm, Type, Coronavirus, Application, Fujifilm Diosynth Biotechnologies, ATCC (company), Novartis, Sigma-Aldrich, Human, Growth, ATCC, Fine chemical, Pharmaceutical industry, Mobile phone, Vaccine, Nusinersen

Rising use of new healthcare technology and rising cancer incidences, create more attractive and lucrative growth prospects for the CLD market.

Key Points: 
  • Rising use of new healthcare technology and rising cancer incidences, create more attractive and lucrative growth prospects for the CLD market.
  • The expansion and growth of the biologics sector will also be a major CLD market development determinant.
  • For example, CYTENA, a BICO subsidiary, introduced a new platform for entirely automated stable cell line development in October 2021.
  • However, the high capital expenses and expenditure needed to construct research and development capabilities would be a barrier to the cell line development industry.

Royalty Pharma Reports Third Quarter 2023 Results

Retrieved on: 
Wednesday, November 8, 2023
Novartis, Aplastic anemia, Vertex, Food, N/A, Pressure, PFS, Cabozantinib, Schizophrenia, New Drug Application, Clinical trial, OS, Calendar, Sale, Exelixis, GAAP, Acquisition, Civil service commission, Apalutamide, Roche, NDA, Papillary tumors of the pineal region, Therapy, Patient, Food and Drug Administration, KarXT, European Commission, Risdiplam, Growth, Cystic fibrosis, Growth hormone, Ipsen, FDA, Progression-free survival, EBITDA, Bladder cancer, Ferring Pharmaceuticals, PTC, Multiple sclerosis, Carcinoma, PTC Therapeutics, Pharmaceutical industry, Video game, Music, Bank, Generic drug, Cryptocurrency, Adjustment, Lonapegsomatropin, Ibrutinib, Regadenoson, Cytokinetics, Nadofaragene firadenovec, Nusinersen, Elexacaftor/tezacaftor/ivacaftor

Drivers of total royalty receipts in the third quarter of 2023 are discussed below, based on commentary from the marketers of the products underlying the royalties in the preceding quarter (as royalty receipts generally lag product performance by one calendar quarter).

Key Points: 
  • Drivers of total royalty receipts in the third quarter of 2023 are discussed below, based on commentary from the marketers of the products underlying the royalties in the preceding quarter (as royalty receipts generally lag product performance by one calendar quarter).
  • Royalty Pharma acquired the Spinraza royalty in the first quarter of 2023.
  • In September 2023, Royalty Pharma acquired a royalty interest in Skytrofa from Ascendis Pharma A/S ( press release ) for an upfront payment of $150 million.
  • During the third quarter of 2023, Royalty Pharma repurchased approximately five million shares for $144 million.

Alltrna Expands Leadership Team with Appointments of Chief Scientific Officer and Chief Technology Officer

Retrieved on: 
Tuesday, August 15, 2023
Molecule, Columbia University, Medicinal chemistry, University, Pasteur Institute, Duke University, Dimethyl fumarate, Therapy, Spinal muscular atrophy, Small RNA, Disease, Development, Biogen, University of Cambridge, CMC, Coronavirus Scientific Advisory Board, Research, RNA, Multiple sclerosis, Movement disorder, Chemistry, DSM-IV codes, Publication, Flagship Pioneering, Pasteur Institute of Iran, Ophthalmology, Biology, Entrepreneurship, SVP, Nusinersen, Rest, ALS, Management, Pharmaceutical industry, Trophos

Dr. Henderson was formerly Senior Vice President, Head of Research at Biogen, and Dr. Kiesman was Senior Vice President, CMC and Medicinal Chemistry at Alltrna.

Key Points: 
  • Dr. Henderson was formerly Senior Vice President, Head of Research at Biogen, and Dr. Kiesman was Senior Vice President, CMC and Medicinal Chemistry at Alltrna.
  • "Chris' deep scientific expertise and experience advancing programs into the clinic across a wide range of disease areas will be invaluable.
  • Will has significantly advanced our internal medicinal chemistry programs and developed a flexible manufacturing strategy to scale and deliver engineered tRNAs.
  • Dr. Henderson has more than 90 scientific research publications and been an editor and reviewer for top-tier scientific journals.

Royalty Pharma Reports Second Quarter 2023 Results

Retrieved on: 
Tuesday, August 8, 2023
Acceleration, Net, Pressure, University, Civil service commission, Astellas Pharma, Chronic lymphocytic leukemia, Vertex, Calendar, Aplastic anemia, DPP, GAAP, Leuprorelin, Sale, Ulcerative colitis, Psoriasis, Gilead, EBITDA, Acquisition, European Commission, Patient, Risk, Metastasis, Cystic fibrosis, Growth, Johnson & Johnson, California Miramar University, FDA, Sacituzumab govitecan, Regulation of tobacco by the U.S. Food and Drug Administration, Death, Food, Ivacaftor, Pfizer, Talazoparib, Bank, Video game, Music, Vaccine, Pharmaceutical industry, Real estate, Cryptocurrency, Berotralstat, Apalutamide, Nusinersen, Elexacaftor/tezacaftor/ivacaftor, Adjustment, Guselkumab, Ibrutinib

Drivers of total royalty receipts in the second quarter of 2023 are discussed below, based on commentary from the marketers of the products underlying the royalties in the preceding quarter (as royalty receipts generally lag product performance by one calendar quarter).

Key Points: 
  • Drivers of total royalty receipts in the second quarter of 2023 are discussed below, based on commentary from the marketers of the products underlying the royalties in the preceding quarter (as royalty receipts generally lag product performance by one calendar quarter).
  • Royalty Pharma acquired a royalty interest in Trelegy in July 2022 and began receiving royalty receipts in the third quarter of 2022.
  • During the second quarter of 2023, Royalty Pharma began repurchasing its Class A ordinary shares and repurchased approximately four million shares for $134 million.
  • The weighted-average diluted Class A ordinary shares outstanding for the second quarter of 2023 was 606 million as compared to 607 million for the first quarter of 2023.

New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

Retrieved on: 
Friday, June 30, 2023
Biogen, SMA, Interim, Investigational device exemption, SMN2, Alcyone and Ceyx, Aes, BIIB, Research, Meta-analysis, Brain cell, Trial of the century, HIV disease progression rates, Patient, Therapy, RESPOND, Nusinersen, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Caregiver, Safety, Medical device, Pharmaceutical industry

(Nasdaq: BIIB) announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community.

Key Points: 
  • (Nasdaq: BIIB) announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community.
  • No new emerging safety concerns have been identified in enrolled participants who received SPINRAZA after Zolgensma.
  • “The RESPOND study has begun to characterize remaining unmet need in some SMA patients treated with Zolgensma whose outcomes have not met clinical expectations.
  • This summer, Alcyone plans to begin initial enrollment of the PIERRE study ( clinicaltrials.gov ), which will evaluate the safety and performance of ThecaFlex for the delivery of SPINRAZA in SMA patients.

Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRxTM System for Administration of SPINRAZA® (nusinersen)

Retrieved on: 
Tuesday, June 27, 2023
SMA, Investigational device exemption, Biogen, Biotechnology, Boston Children's Hospital, BIIB, DSM-IV codes, Cerebrospinal fluid, Harvard Medical School, Patient, ASO, CSF, Hospital, IDE, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Nusinersen, Memory, Food, Safety, Scoliosis, Pharmaceutical industry, Alcyone and Ceyx

The PIERRE pivotal study ( https://clinicaltrials.gov/ct2/show/NCT05866419 ) will enroll up to 90 patients and will be conducted in two stages.

Key Points: 
  • The PIERRE pivotal study ( https://clinicaltrials.gov/ct2/show/NCT05866419 ) will enroll up to 90 patients and will be conducted in two stages.
  • "Alcyone is excited to begin the process of evaluating the safety and performance of ThecaFlex in SMA patients being treated with SPINRAZA.
  • ThecaFlex represents the culmination of a deliberate effort to design a delivery system specifically for repeat bolus intrathecal drug delivery," said PJ Anand, Chief Executive Officer of Alcyone.
  • The results of the PIERRE study to evaluate ThecaFlex with SPINRAZA in SMA may inform pathways for Biogen's broader portfolio of investigational ASO therapies.

Ionis Chief Scientific Officer C. Frank Bennett, Ph.D. to receive Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research

Retrieved on: 
Thursday, January 26, 2023
PDUFA, Miller, Tauopathy, University, ALS, Ionis Pharmaceuticals, Neurology, Lewy body dementias, Hope, Pharmacokinetics, Nusinersen, Breakthrough Prize, Huntington's disease, Rain, IONS, Palsy, DRS, Breakthrough Prize in Life Sciences, Washington University School of Medicine, Prescription Drug User Fee Act, Alzheimer's disease, Molecular medicine, Hereditary Disease Foundation, Brain, Oligonucleotide, Patient, University of California, Research, BPS Barbara Wilson Lifetime Achievement Award, Neurodegenerative disease, Pharmaceutical industry, Ionis, Science

CARLSBAD, Calif., Jan. 26, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Chief Scientific Officer C. Frank Bennett, Ph.D., has been named a co-recipient of the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research.

Key Points: 
  • CARLSBAD, Calif., Jan. 26, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Chief Scientific Officer C. Frank Bennett, Ph.D., has been named a co-recipient of the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research.
  • The prizes will be presented during the Eurotau 2023 conference on April 27, 2023, in Lille, France.
  • Bennett, Cleveland and Miller are being recognized for their work advancing antisense technology for the treatment of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Huntington's disease, Alzheimer's disease and tauopathies.
  • A founding member of Ionis, Dr. Bennett is responsible for continuing to advance Ionis' technology and expand its discovery platform.

Rainwater Charitable Foundation Announces Fourth-Annual Rainwater Prize Winners for Brain Research

Retrieved on: 
Thursday, January 26, 2023
Miller, Conference, Presidential Early Career Award for Scientists and Engineers, Research group, Neurology, Washington University School of Medicine, FDA, Huntington's disease, Brain, Molecular medicine, Disease, Engineering, Innovation, DSM-IV codes, Tauopathy, DZNE, Biophysics, ASO, Nervous system, DRS, SMA, Biology, Neurodegenerative disease, Progressive supranuclear palsy, Neuron, Messenger, DNA, University of California, San Diego, UCSD, Alzheimer's disease, Washington University in St. Louis, Drug discovery, Cell, Tau, Parkinson's disease, University, ALS, German Center for Neurodegenerative Diseases, Distinguishing, Nusinersen, Rain, SOD1, Wegmann, Pharmaceutical industry, Research, Ionis

FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist. This year's Outstanding Innovation Prize will be awarded to a team: Dr. C. Frank Bennett, Ionis; Dr. Don W. Cleveland, University of California San Diego (UCSD); and Dr. Timothy M. Miller, Washington University. Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist. The prizes will be presented during the Eurotau 2023 Conference on April 27, 2023 in Lille, France.

Key Points: 
  • FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist.
  • Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist.
  • Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research will be shared by:
    Collaborators Drs.
  • "I could not be more excited about this year's winners of the Rainwater Prize," said Todd Rainwater, Trustee of the Rainwater Charitable Foundation.

Rainwater Charitable Foundation Announces Fourth-Annual Rainwater Prize Winners for Brain Research

Retrieved on: 
Thursday, January 26, 2023
Miller, Conference, Presidential Early Career Award for Scientists and Engineers, Research group, Neurology, Washington University School of Medicine, FDA, Huntington's disease, Brain, Molecular medicine, Disease, Engineering, Innovation, DSM-IV codes, Tauopathy, DZNE, Biophysics, ASO, Nervous system, DRS, SMA, Biology, Neurodegenerative disease, Progressive supranuclear palsy, Neuron, Messenger, DNA, University of California, San Diego, UCSD, Alzheimer's disease, Washington University in St. Louis, Drug discovery, Cell, Tau, Parkinson's disease, University, ALS, German Center for Neurodegenerative Diseases, Distinguishing, Nusinersen, Rain, SOD1, Wegmann, Pharmaceutical industry, Research, Ionis

FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist. This year's Outstanding Innovation Prize will be awarded to a team: Dr. C. Frank Bennett, Ionis; Dr. Don W. Cleveland, University of California San Diego (UCSD); and Dr. Timothy M. Miller, Washington University. Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist. The prizes will be presented during the Eurotau 2023 Conference on April 27, 2023 in Lille, France.

Key Points: 
  • FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist.
  • Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist.
  • Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research will be shared by:
    Collaborators Drs.
  • "I could not be more excited about this year's winners of the Rainwater Prize," said Todd Rainwater, Trustee of the Rainwater Charitable Foundation.

Spinal Muscular Atrophy Treatment Market Worth $18.0 Billion by 2030: Grand View Research, Inc.

Retrieved on: 
Wednesday, January 11, 2023
Clinical trial, Ageing, SMA, Gene therapy, EC, UNC Medical Center, Food, Therapy, Research, Spinal muscular atrophy, Genentech, Royal commission, USD, SAN, Disease, Growth, Patient, Novartis, MAP, Revenue, Treatment, Route of administration, European Commission, Food and Drug Administration Amendments Act of 2007, EMA, Prevalence, Diagnosis, PTC Therapeutics, Pharmaceutical industry, Medical device, Medical imaging, Nusinersen, Onasemnogene abeparvovec, Risdiplam

SAN FRANCISCO, Jan. 11, 2023 /PRNewswire/ -- The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030, according to a new study by Grand View Research Inc.

Key Points: 
  • SAN FRANCISCO, Jan. 11, 2023 /PRNewswire/ -- The global spinal muscular atrophy treatment market size is expected to reach USD 18.0 billion by 2030, according to a new study by Grand View Research Inc.
  • For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India.
  • Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.
  • Grand View Research has segmented the global spinal muscular atrophy treatment market based on type, application, technology, end-use, and region:
    Spinal Muscular Atrophy Treatment Market - Type Outlook (Revenue, USD Million, 2018 - 2030)