ERT

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

Verizon partners with U.S. military in extreme training to prepare for natural disaster response

Retrieved on: 
Thursday, February 29, 2024
VZ, PATRIOT, Senior, Emergency, Air force, Core, RSS, ERT, Military personnel, Exercise, DIRT, Government, National Guard, MEMA, Category, Camp Shelby, Wi-Fi, Verizon Communications, Mississippi National Guard, The, Partnership

The Verizon Response Teams participating in the drills included:

Key Points: 
  • The Verizon Response Teams participating in the drills included:
    Verizon’s Major Emergency Response Incident Team (MERIT), a specialized team of hazardous material-certified Verizon technicians and engineers.
  • Verizon’s Dedicated Impact Response Team (DIRT), technicians and engineers with specialty training in mobile satellite asset deployment, basic generator repair, and cable/connector making.
  • Verizon Frontline Crisis Response Team dedicated to providing on-demand, emergency communication assistance during crisis situations to government agencies and emergency responders, on a 24/7 basis.
  • Headquartered in New York City and with a presence around the world, Verizon generated revenues of $134.0 billion in 2023.

ILUS Signs Letter of Intent to Acquire a British Manufacturer of Autonomous Vehicles While Completing its Next Phase of Restructuring for Anticipated Growth

Retrieved on: 
Tuesday, February 20, 2024
RB, Acquisition, Majority, OTC, Mergers and acquisitions, Growth, ERT, Defense, Letter, Information, Probability, Negotiation, SAML, Water, Security (finance)

This and several additional factors have led to increased instability, placing heightened demand on the defense and security sectors leading to increased growth.

Key Points: 
  • This and several additional factors have led to increased instability, placing heightened demand on the defense and security sectors leading to increased growth.
  • “This acquisition and those in the pipeline are expected to add incredible value to the ILUS Group.
  • Furthermore, ILUS is in discussions with RB Capital regarding the consolidation of its existing promissory notes into one single promissory note.
  • I am currently in the USA with JP Backwell for the final stage of the business combination transaction with the NASDAQ company.

Medecision Appoints Mark Gee as Chief Revenue Officer to Spearhead Growth and Innovation

Retrieved on: 
Monday, February 26, 2024
Aerial, Emerging market, Innovation system, Utah State University, Growth, ERT, ED, Lean manufacturing, Cigna, Health care, Ecosystem, Patient, UnitedHealth Group, Management

DALLAS and KING OF PRUSSIA, Pa., Feb. 26, 2024 /PRNewswire/ -- Medecision , the leading clinical data and analytics platform company, has announced the addition of Mark Gee to its team as Chief Revenue Officer (CRO).

Key Points: 
  • DALLAS and KING OF PRUSSIA, Pa., Feb. 26, 2024 /PRNewswire/ -- Medecision , the leading clinical data and analytics platform company, has announced the addition of Mark Gee to its team as Chief Revenue Officer (CRO).
  • Gee brings a wealth of experience in healthcare leadership, with a deep focus on value-based care strategies, and enterprise sales.
  • Before joining Medecision, Gee was Chief Growth Officer of Payer and Emerging Markets at WellSky , driving strategy for payer, life sciences, and channel partners.
  • Kenneth Young, President and CEO of Medecision, said, "We are delighted to welcome Mark Gee to our team.

Equality by Design: A Model for Managing Discriminatory Risks of AI

Retrieved on: 
Thursday, February 15, 2024
Cosmetics, Retail, Consumer, Technology, Catalog, Women, Artificial Intelligence, Bias, Mary Kay, ERT, Woman, Intelligence, Multimedia, Artificial intelligence, Research, Principle, Partnership

has partnered with the Equal Rights Trust (ERT) , an organization whose mission is to advance equality through law around the world.

Key Points: 
  • has partnered with the Equal Rights Trust (ERT) , an organization whose mission is to advance equality through law around the world.
  • As part of this collaboration, Mary Kay Inc. supported new research focused on understanding and addressing the discriminatory impacts of artificial intelligence (AI) and its impacts on gender equality, and the development of a new “equality by design approach.”
    This press release features multimedia.
  • View the full release here: https://www.businesswire.com/news/home/20240215911706/en/
    “Our collaboration with Equal Rights Trust underscores our dedication to ensuring that technological advancements, especially in AI, champion gender equality.
  • Read Jim Fitzgerald’s article titled Equality by Design: A Model for Managing Discriminatory Risks of AI here .

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

Retrieved on: 
Wednesday, February 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

Retrieved on: 
Wednesday, February 14, 2024
Enzyme replacement therapy, Fabry disease, ICV, EMA, Intelligence, European, Glycolipid, ERT, Sanfilippo syndrome, Hunter syndrome, Death, MPS, Prevalence, ODD, Safety, Sanfilippo, IDS, Disorder, Patient, Hanmi Pharmaceutical, Fibrosis, Clinical trial, Knowledge, Kidney disease, Disease, Urine, Heart, Alpha-galactosidase, Phase, Male, Kidney, Glycogen storage disease, Brain, Orphan, Mouse, Pharmaceutical industry

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease

Retrieved on: 
Monday, February 12, 2024
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Fabry disease, EMA, Orphan drug, Type D, Investment, Female, ERT, BLA, STAAR, Therapy, PRIME, Sangamo Therapeutics, Patient, Male, Enzyme replacement therapy, Partnership, Pharmaceutical industry

The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.

Key Points: 
  • The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.
  • A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA.
  • This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.
  • Sangamo is deferring additional investments in planning for a registrational trial until a collaboration partnership is secured.

Chiesi Global Rare Diseases Presents Long-Term Data on Treatment with Velmanase Alfa in Alpha-Mannosidosis and Announces Additional Presentations at the 20th Annual WORLDSymposium™ Research Meeting

Retrieved on: 
Thursday, February 8, 2024
Conjunctivitis, Arthralgia, Anaphylaxis, Common, ADAS, Birth, Prevalence, Velmanase alfa, Fabry disease, Human, Diagnosis, Headache, Cough, Erythema, ERT, Glycogen storage disease, Alpha-mannosidosis, DSM-IV codes, Vomiting, Hospital, Incidence, Patient, Rash, Nursing, Data, Chills, Enzyme replacement therapy, Pharmaceutical industry

BOSTON, Feb. 8, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the presentation of long-term data from up to 12 years of treatment with velmanase alfa in patients with alpha-mannosidosis (AM). The company also announced the presentation of immunogenicity and tolerability data from its pegunigalsidase alfa clinical trial program in Fabry disease, as well as analyses of the prevalence, diagnosis, and progression of these rare lysosomal storage disorders. The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.

Key Points: 
  • The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.
  • Several poster presentations are highlighting immunogenicity and tolerability data from the pegunigalsidase alfa clinical trial program in Fabry disease.
  • The analysis includes 108 patients, 27 (25%) of whom were ADA+ before receiving their first dose of pegunigalsidase alfa.
  • There was a low incidence of de novo ADAs (n=17) occurring predominantly in the first year of treatment with pegunigalsidase alfa.

Amicus Therapeutics Receives the 2024 New Treatment Award for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) at the 20th Annual WORLDSymposium™

Retrieved on: 
Thursday, February 8, 2024
Partnership, Amicus Therapeutics, Cipaglucosidase alfa, Glycogen, Myopathy, ERT, B cell, FDA, GAA, Fabry disease, Food, Physician, Blood, Therapy, Lysosome, Enzyme replacement therapy, Medicine, Pompe, Pharmaceutical industry

Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen.

Key Points: 
  • Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen.
  • Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA).
  • This is the second WORLDSymposium™ New Treatment Award presented to Amicus.
  • Amicus received the award in 2017 for the first and only oral therapy approved for people living with Fabry disease.