Spinal muscular atrophy

Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Patient, Liver, Medical Subject Headings, Marketing, Survival, INN, Risk, Fever, Caregiver, Spinal muscular atrophy, ATC, SMN2, Platelet, International, Disease, Atrophy, Language, Troponin, SMN1, Thrombocytopenia, Ventilation, European Medicines Agency, Select, Hepatotoxicity, Facial muscles, Safety, Anatomy, Vomiting, IIA, SMA, Medical device

Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Zolgensma, onasemnogene abeparvovec, Date of authorisation: 18/05/2020, Revision: 14, Status: Authorised

Voyager Therapeutics Enters Capsid License Agreement and Strategic Collaboration with Novartis to Advance Novel Gene Therapies

Retrieved on: 
Tuesday, January 2, 2024
Spinal muscular atrophy, NVS, HD, Therapy, TRACER, DSM-IV codes, Biomedical Research, Biomedicine, Central nervous system, Patient, SMA, Pharmaceutical industry

LEXINGTON, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER™ capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for HD.

Key Points: 
  • LEXINGTON, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA).
  • Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER™ capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for HD.
  • “We are thrilled to expand our existing relationship with Novartis, a global leader in the gene therapy field,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager.
  • Novartis will also receive worldwide rights to Voyager’s AAV gene therapy for HD, leveraging Voyager’s TRACER capsids and proprietary payloads.

NMD Pharma Raises €75 Million (~$80 million) in a Series B Financing

Retrieved on: 
Wednesday, November 15, 2023
Spinal muscular atrophy, Acetylcholine receptor, Investment, Patient, Myasthenia gravis, Novo Holdings A/S, Therapy, Charcot–Marie–Tooth disease, MuSK protein, NMD, Growth, Pharma, Pharmaceutical industry, Fine chemical

The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.

Key Points: 
  • The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.
  • NMD Pharma is developing first-in-class small molecule inhibitors of the skeletal muscle specific chloride ion channel (ClC-1) to enhance neuromuscular transmission with the potential to restore muscle function in a range of rare neuromuscular diseases.
  • Proceeds from the financing will be used to complete three Phase 2 studies with NMD670, the Company’s lead ClC-1 inhibitor.
  • Furthermore, NMD Pharma will continue to expand its pipeline of ClC-1 inhibitor molecules and pursue undisclosed targets for the treatment of other neuromuscular diseases.

OXFORD PROPERTIES BREAKS GROUND ON 165,000 SQ FT EXPANSION OF IONIS PHARMACEUTICALS CARLSBAD CAMPUS

Retrieved on: 
Tuesday, December 5, 2023
Spinal muscular atrophy, Foot, Acquisition, Research, Scripps Research, Patient, Investment, Ecosystem, Science, ALS, First Ever, Therapy, Novartis, San Francisco Bay Area, Partnership, Oxford Properties, Growth, Ionis Pharmaceuticals, UC, San Diego State University, Thermo Fisher Scientific, RNA, Genentech, Grocery store, Fine chemical, Pharmaceutical industry, Ionis

Located in the San Diego life sciences submarket of Carlsbad, Ionis' existing campus features approximately 250,000 square feet of lab and office space.

Key Points: 
  • Located in the San Diego life sciences submarket of Carlsbad, Ionis' existing campus features approximately 250,000 square feet of lab and office space.
  • The expansion builds on Oxford and Ionis' established partnership, with Oxford completing a purchase and long-term lease back of Ionis' existing campus in October 2022.
  • Oxford entered the San Diego life sciences market in February 2022 via a US$464 million acquisition of a 13-building portfolio.
  • The campus expansion supports Ionis' strategic goals of prioritizing its wholly owned pipeline and advancing its drug discovery technology.

Editas Medicine Announces Third Quarter 2023 Results and Business Updates

Retrieved on: 
Friday, November 3, 2023
FDA, Research, Regenerative Medicine Advanced Therapy, Food, Spinal muscular atrophy, Biogen, SCD, Life, Public expenditure, TDT, Cas9, Sickle cell disease, Regulation of tobacco by the U.S. Food and Drug Administration, Poster, Society, ASH, Commercialization, HSC, File, Fetal hemoglobin, Collaboration, Lead, Beta thalassemia, Anemia, Multiple sclerosis, Bone marrow, Therapy, Patient, BLA, MS, Pharmaceutical industry, Cryptocurrency, Edith A. Perez, Editas Medicine, EU

CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.
  • Cash, cash equivalents, and marketable securities as of September 30, 2023, were $446.4 million compared to $480.0 million as of June 30, 2023.
  • The increase is primarily related to an upfront payment for the non-exclusive Cas9 license to Vor Bio in the third quarter of 2023.
  • Editas Medicine plans to participate in the following scientific and medical conference:
    Editas Medicine plans to participate in the following investor events:

Royalty Pharma Announces Agreement to Purchase Up to $1.5 Billion of PTC Therapeutics’ Royalty on Evrysdi

Retrieved on: 
Thursday, October 19, 2023
SMN2, FDA, Spinal muscular atrophy, Food, EDT, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Exercise, Growth, Medicine, Partnership, PTC, Roche, PTC Therapeutics, Therapy, SMA, Patient, CHF, Music, Pharmaceutical industry, Risdiplam

In 2022, Evrysdi generated sales of approximately CHF 1.1 billion ($1.2 billion), an increase of 87% at constant exchange rates versus the prior year.

Key Points: 
  • In 2022, Evrysdi generated sales of approximately CHF 1.1 billion ($1.2 billion), an increase of 87% at constant exchange rates versus the prior year.
  • Following the transaction announced today, in total, Royalty Pharma is increasing its ownership of the Evrysdi royalty from 43% to 81%, equating to a royalty of 6.5% to 13% (not including any exercise of future options by PTC or Royalty Pharma).
  • If PTC exercises fewer than three of these options, Royalty Pharma has the option to purchase 50% of the remaining PTC royalty for $250 million less royalties received until March 31, 2026.
  • Since 2020, Royalty Pharma has announced transactions of up to $12.8 billion, including up to $3.8 billion in 2023.

NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Tuesday, October 3, 2023
SMA, Spinal muscular atrophy, NMD, EVP, Clinical trial, Myasthenia gravis, EDT, Congress, MG, Patient, Type III, International Congress on Tuberculosis, Pharmaceutical industry

The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.

Key Points: 
  • The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.
  • Details of NMD Pharma’s poster presentation are below:
    Date and time: Thursday, 5 October from 3:30 pm to 4:30 pm EDT.
  • "The International Congress of the World Muscle Society is one of the most significant scientific gatherings in our field, globally.
  • We eagerly anticipate the opportunity to share data from our clinical trial involving patients with myasthenia gravis," remarked Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma.

NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Tuesday, October 3, 2023
SMA, Spinal muscular atrophy, NMD, EVP, Clinical trial, Myasthenia gravis, EDT, Congress, MG, Patient, Type III, International Congress on Tuberculosis, Pharmaceutical industry

The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.

Key Points: 
  • The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.
  • Details of NMD Pharma’s poster presentation are below:
    Date and time: Thursday, 5 October from 3:30 pm to 4:30 pm EDT.
  • "The International Congress of the World Muscle Society is one of the most significant scientific gatherings in our field, globally.
  • We eagerly anticipate the opportunity to share data from our clinical trial involving patients with myasthenia gravis," remarked Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma.

'I want to get bogged at a beach in my wheelchair and know people will help'. Micheline Lee on the way forward for the NDIS

Retrieved on: 
Tuesday, September 26, 2023
People, Schwartz Publishing, Heart, Spinal muscular atrophy, Life, Friends, Disability, Parent, Review, Policy, Survival, Eating, Dignity, Metaphor, Human, National Disability Insurance Scheme, Research, History, Movement, Government, Essay, Sibling, Time, Logic, Humanity, NDIS, Health, Health insurance, Renewable energy, Insurance, Hunting, Health care

The NDIS is the “lifeboat in the ocean”, “an oasis in the desert”, “a plane being built mid-flight” or a “limitless magic pudding”.

Key Points: 
  • The NDIS is the “lifeboat in the ocean”, “an oasis in the desert”, “a plane being built mid-flight” or a “limitless magic pudding”.
  • I research disability policies and services and confess I’ve used more than a few of these in my work.
  • They are a way of explaining complex concepts we might not be familiar with and helping others to make sense of the world.
  • The latest Quarterly Essay Lifeboat: Disability, Humanity and the NDIS written by author Micheline Lee weaves together personal testimony and detailed analysis of history and policy.

The deficit model of disability

    • Disability was firmly seen as being a problem or deficit within the body of an individual who needed to be cured.
    • Lee’s discovery of the social model of disability challenged how she had been raised to understand it.
    • The social model sees disability as produced by
      social and environmental barriers such as discriminatory attitudes and policies, inaccessible buildings and transport, and inflexible work arrangements.
    • Disability activists, such as Rhonda Galbally, have documented the rise of the disability rights movement in Australia, which fought for the establishment of the NDIS.

Citizens or consumers?

    • As Lee describes,
      Disabled people wanted more than just survival, getting out of bed, showering and eating, and maintaining basic health.
    • Disabled people wanted more than just survival, getting out of bed, showering and eating, and maintaining basic health.
    • Yet, inherent within the design of the NDIS are two competing logics: citizenship and consumer rights.
    • The former sees the role of the scheme, in collaboration with others, as enabling and empowering people with disability to engage with the community and broader society.
    • Disability is seen by many as something specialist professionals and services should handle, not a facet of the human condition.
    • I want to get bogged at a beach in my wheelchair and know people will help.

Draining the ocean

    • The market-based system is not operating as intended and changing attitudes outside the scheme have been neglected.
    • The current NDIS Review is hearing about all these issues, but none are surprising to disabled people.
    • It is incumbent upon those of us within the non-disabled community to listen and act to create a more inclusive society.
    • Making the NDIS work as intended, is a job for all of Australian society and not just disabled people.

CENTOGENE Reports First Half 2023 Financial Results

Retrieved on: 
Thursday, September 7, 2023
TWIST, Saskatchewan River Sturgeon Management Board, Spinal muscular atrophy, GCC, Tax, G&A, Cystic fibrosis transmembrane conductance regulator, Patient, R, Development, WES, Sequence, Disease, Annual general meeting, Genetics, Research, WGS, Growth, Diagnosis, Whole genome sequencing, Total, Gaucher's disease, Observational study, LRRK2, DNA sequencing, History, Warburg Micro syndrome, JV, DSM-IV codes, Supervisory board, Gulf Cooperation Council, Therapy, Cancer, Partnership, Public Investment Fund, Mox, Medication, Joint venture, CFTR, Twist Bioscience, EBITDA, Fine chemical, Cryptocurrency, Pharmaceutical industry, Clothing, Video game, Palladium, Pharma

and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.

Key Points: 
  • and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.
  • Cost of sales increased by €2,692 thousand, or 20.7%, to €15,728 thousand for the first half of 2023, from €13,036 thousand for the first half of 2022.
  • General administrative expenses decreased by €112 thousand, or 0.6%, to €17,172 thousand for the first half of 2023, from €17,284 thousand for the first half of 2022.
  • The decrease is mainly due to the increase in consumable expenses on approximately €1,601 thousand for the first half of 2023.