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Mustang Bio Reports Full-Year 2023 Financial Results and Recent Corporate Highlights

Retrieved on: 
Monday, March 11, 2024

WORCESTER, Mass., March 11, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced financial results and recent corporate highlights for the full-year ended December 31, 2023.

Key Points: 
  • WORCESTER, Mass., March 11, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced financial results and recent corporate highlights for the full-year ended December 31, 2023.
  • Research and development expenses were $40.5 million for the year ended December 31, 2023, compared to $62.5 million for 2022.
  • 2023 and Recent Corporate Highlights:
    In July 2023, Mustang announced that it amended its previously announced asset purchase agreement with uBriGene (Boston) Biosciences Inc. (“uBriGene”) and closed the transaction.
  • In October 2023, Mustang completed a registered direct offering priced at-the-market for approximately $4.4 million in gross proceeds.

Mustang Bio Announces Publication in Nature Medicine of Data from Phase 1 Trial Evaluating MB-101 IL13Rα2-targeted CAR T-Cells in High-Grade Glioma

Retrieved on: 
Thursday, March 7, 2024

WORCESTER, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced Phase 1 clinical data were published in Nature Medicine that demonstrated the promising safety and clinical activity of Mustang’s MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of patients with recurrent and refractory malignant glioma, including glioblastoma.

Key Points: 
  • MB-101 was developed by City of Hope, one of the largest cancer research and treatment organizations in the United States, and exclusively licensed to Mustang.
  • Central nervous system (CNS) increases in inflammatory cytokines, including IFNγ, CXCL9, and CXCL10, were associated with CAR T-cell administration and bioactivity.
  • Primary endpoints were safety and feasibility, with secondary endpoints measuring therapy-related cytokine dynamics, CAR T-cell persistence and clinical outcomes.
  • Dr. Brown has a financial interest in Mustang and has previously been a paid consultant for the company.

Neurogene Announces Expansion and Plans for More Rapid Patient Enrollment of Rett Syndrome Gene Therapy Clinical Trial

Retrieved on: 
Monday, March 4, 2024

Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the expansion of its ongoing Phase 1/2 gene therapy clinical trial for NGN-401 for female pediatric patients with Rett syndrome and updates to enable more rapid enrollment in the trial.

Key Points: 
  • Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the expansion of its ongoing Phase 1/2 gene therapy clinical trial for NGN-401 for female pediatric patients with Rett syndrome and updates to enable more rapid enrollment in the trial.
  • We expect that expansion of the clinical trial and the removal of staggered dosing in Cohort 1 will enable us to treat more patients in a shorter period of time.
  • Based on this update, we expect to complete enrollment of Cohort 1 in the second half of 2024.”
    The U.S.
  • The dosing stagger has been removed from Cohort 1, enabling the remaining patients to be dosed in parallel.

Grace Science, LLC Announces First Patient Treated in Phase 1/2/3 Trial of GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency

Retrieved on: 
Tuesday, March 5, 2024

Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency.

Key Points: 
  • Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency.
  • Part 1/2 of the study will investigate dose-escalation and the safety and efficacy of a single intracerebroventricular (ICV) infusion of GS-100 in NGLY1 Deficiency patients aged 2-18 years.
  • Matt Wilsey, CEO & Co-Founder of Grace Science, LLC, stated that "Treating the first patient is a tremendous milestone for our company and the NGLY1 Deficiency patients we strive to cure.
  • Dr. Bertozzi went on to say “The team has worked incredibly hard to reach the goal of treating the first NGLY1 Deficiency patient with this potentially life-changing therapy.

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

Retrieved on: 
Wednesday, February 14, 2024

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.

Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome

Retrieved on: 
Wednesday, January 31, 2024

Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).

Key Points: 
  • Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).
  • JAG201 aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.
  • “We are pleased to receive FDA clearance to bring our investigational SHANK3 gene therapy to the clinic.
  • The pre-clinical data indicate that JAG201 may have the potential to be transformative for those suffering with the disorder,” said Joe Nolan, chief executive officer of Jaguar Gene Therapy.

European Medicines Agency (EMA) Grants Orphan Drug Designation (ODD) to GC Biopharma's Treatment for Sanfilippo Syndrome (Type A)

Retrieved on: 
Tuesday, January 23, 2024

GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023.

Key Points: 
  • GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023.
  • Sanfilippo Syndrome (type A) is a genetic disorder that triggers central nervous system damage by accumulating Heparan sulfate, leading to a progressive neurodegeneration in pediatric population.
  • "This EMA designation further underlines the potentials of our collaborative pipeline in addressing the disease pathology in upcoming clinical trials.
  • ", said GC Biopharma and added that it will be "committing to expeditiously advancing into clinical trials, acknowledging the urgent, unmet medical needs of Sanfilippo Syndrome patients."

Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome

Retrieved on: 
Thursday, November 30, 2023

NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.

Key Points: 
  • NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.
  • NGN-401 delivers the full-length human methyl cytosine binding protein 2 (MECP2) gene, providing an optimal gene replacement approach.
  • In non-clinical studies with NGN-401 at clinically relevant doses, cardinal features of Rett syndrome were ameliorated, and no overexpression toxicity was observed.
  • Your resilience, courage, and support not only contribute to the progress of this research, but also inspire hope within the entire Rett syndrome community.

Grace Science Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Phase 1/2/3 Trial for the Treatment of NGLY1 Deficiency with GS-100, an AAV9 NGLY1 Gene Therapy

Retrieved on: 
Tuesday, November 7, 2023

Grace Science, LLC announced today that it has received FDA clearance of its Investigational New Drug (IND) application for GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency.

Key Points: 
  • Grace Science, LLC announced today that it has received FDA clearance of its Investigational New Drug (IND) application for GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency.
  • Bertozzi and I started Grace Science to save lives across the rare disease community — and beyond.
  • “This is an important milestone for NGLY1 Deficiency families, as well as for our company.
  • This is the first Grace Science program to receive FDA clearance to enter the clinic and the first gene therapy clinical trial for NGLY1 Deficiency.”

WiMi's Research on Human-Computer Interaction System for Intelligent Connected Vehicles Based on Big Data and Hybrid Algorithm

Retrieved on: 
Thursday, July 20, 2023

Human-computer interaction refers to the data exchange and mutual understanding between humans and machines through specific sensors and interfaces with the support of certain interaction technologies.

Key Points: 
  • Human-computer interaction refers to the data exchange and mutual understanding between humans and machines through specific sensors and interfaces with the support of certain interaction technologies.
  • Applying artificial intelligence to the key technologies of human-computer interaction is an important way to solve the bottleneck of current human-computer interaction technologies.
  • Using big data technology to process real-time data, algorithms are used to help intelligent vehicles find the best driving path and pass it to the networked vehicles through the human-computer interaction system, which realizes human-computer interaction and parallel processing and calculation of data in networked vehicles and saves various resource costs.
  • WiMi's human-computer interaction system for intelligent connected vehicles based on big data and hybrid algorithm can accumulate many derived big data, including state monitoring, interaction control, personal preference and web service data, which can be used for active learning and self-adaptation of in-vehicle systems.