Inserm

Genethon Announces Publication in The Lancet Neurology of Clinical Trial Results of a Gene Therapy for Myotubular Myopathy, a Severe Muscle Disease

Retrieved on: 
Friday, November 17, 2023

Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.

Key Points: 
  • Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.
  • Characterized by extreme muscle weakness and severe respiratory distress, 50% of affected children die before age 18 months and 75% die before age 10.
  • The gene therapy uses an adeno-associated viral vector (AAV8) to deliver a copy of the MTM1 gene.
  • It took years of research to imagine, design and demonstrate the efficacy of the gene therapy for this very severe and complex disease.

Cassava Sciences Announces Science Publication That Confirms Mechanism of Action of Simufilam, a Novel Drug Candidate for People with Alzheimer’s Disease

Retrieved on: 
Monday, September 11, 2023

AUSTIN, Texas, Sept. 11, 2023 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today announced the publication of new research that confirms the biological activity of simufilam.

Key Points: 
  • AUSTIN, Texas, Sept. 11, 2023 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today announced the publication of new research that confirms the biological activity of simufilam.
  • Simufilam is Cassava Sciences’ novel drug candidate for people with Alzheimer’s disease dementia and is currently under evaluation in a pair of global Phase 3 clinical trials.
  • Cassava Sciences believes this protein interaction underlies simufilam’s mechanism of action in Alzheimer’s disease.
  • Cassava Sciences is evaluating simufilam oral tablets for Alzheimer’s disease dementia in two global Phase 3 clinical studies.

ILiAD Biotechnologies Announces $42.8M Financing Round to Advance BPZE1 Pertussis Vaccine

Retrieved on: 
Tuesday, September 6, 2022

ILiAD Biotechnologies, LLC (ILiAD), a clinical stage biotech company developing the worlds most advanced next generation pertussis vaccine, today announced the closing of a $42.8 million Class D round of financing.

Key Points: 
  • ILiAD Biotechnologies, LLC (ILiAD), a clinical stage biotech company developing the worlds most advanced next generation pertussis vaccine, today announced the closing of a $42.8 million Class D round of financing.
  • In addition to funding continued operations, R&D and manufacturing development of BPZE1, the financing will support further clinical studies of the vaccine.
  • The Phase 2b pertussis Human Challenge clinical study is currently enrolling volunteers and will directly measure whether ILiADs candidate vaccine, BPZE1, can prevent nasopharyngeal B. pertussis colonization after direct challenge with wild-type B. pertussis bacteria.
  • In preclinical studies, BPZE1 was able to prevent B. pertussis colonization after wild-type B. pertussis challenge, while acellular pertussis vaccines were not.

New Data in Nature Medicine Suggest Pre-Treatment Tumor Microenvironment Can Impact Response for CAR T-Cell Therapy in Patients with Large B-Cell Lymphoma

Retrieved on: 
Monday, August 29, 2022

Veracyte, Inc. (Nasdaq: VCYT) announced that new data published today in Nature Medicine provide the first evidence that the pre-treatment tumor microenvironment (TME) can impact response to chimeric-antigen-receptor (CAR) T-cell therapy among patients with large B-cell lymphoma (LBCL).

Key Points: 
  • Veracyte, Inc. (Nasdaq: VCYT) announced that new data published today in Nature Medicine provide the first evidence that the pre-treatment tumor microenvironment (TME) can impact response to chimeric-antigen-receptor (CAR) T-cell therapy among patients with large B-cell lymphoma (LBCL).
  • The study findings demonstrated for the first time the prognostic and predictive capabilities of Veracytes proprietary biomarkers among LBCL patients treated with CAR T-cell therapy.
  • This study aimed to identify biomarkers associated with CAR T-cell therapy outcomes in patients treated with Kites axicabtagene ciloleucel (axi-cel), a first-in-class anti-CD19 CAR T-cell therapy.
  • Results published today suggest that the pre-treatment tumor immune contexture was associated with, and potentially a major determinant of, clinical outcomes in ZUMA-1 patients.

SATT[1] Conectus and Find Therapeutics Sign Licensing Agreement to Develop a Promising New Therapy for Multiple Sclerosis and other demyelinating diseases

Retrieved on: 
Tuesday, June 7, 2022

Promising preclinical data show that this innovative therapy, by overcoming the molecular barriers of myelin regeneration, could restore myelin function.

Key Points: 
  • Promising preclinical data show that this innovative therapy, by overcoming the molecular barriers of myelin regeneration, could restore myelin function.
  • According to the Multiple Sclerosis Society of Canada, Canada has one of the highest MS rates in the world with 1 in every 400 people affected.
  • Initial preclinical pharmacology studies, funded by SATT Conectus, have demonstrated very encouraging results, delivering increased myelin and better function after treatment.
  • Find Therapeutics, is dedicated to the development of next generation trans-membrane allosteric modulators to treat rare and inflammatory diseases.

NovoBiome and Inserm form new collaboration to simulate human intestinal physiology

Retrieved on: 
Tuesday, February 22, 2022

The collaborative agreement has been negotiated and signed by Inserm Transfert, the private subsidiary of Inserm, on behalf of Inserm and Nantes University.

Key Points: 
  • The collaborative agreement has been negotiated and signed by Inserm Transfert, the private subsidiary of Inserm, on behalf of Inserm and Nantes University.
  • Pierre-Yves Mousset, Chief Executive Officer of NovoBiome, said: The implications of understanding the host-microbiota relationships are considerable in human health.
  • However, this major part of physiology is currently a black box that the exceptional cooperation between NovoBiome and two French academic structures of excellence, Inserm and INRAE, will seek to decipher.
  • In 2009, Inserm Transfert and Inserm established an investment fund to finance proofs of concept.

LinKinVax and Inserm Transfert Enter Into an Exclusive Worldwide Licensing Agreement Covering Use of an Innovative Vaccine Platform

Retrieved on: 
Thursday, February 3, 2022

LinKinVax, a clinical-stage biotech company, is announcing the signing of an exclusive worldwide licensing agreement with Inserm Transfert to develop a vaccine strategy with an innovative mechanism of action.

Key Points: 
  • LinKinVax, a clinical-stage biotech company, is announcing the signing of an exclusive worldwide licensing agreement with Inserm Transfert to develop a vaccine strategy with an innovative mechanism of action.
  • LinKinVax is developing a vaccine platform that directly targets dendritic cells.
  • This research has accelerated adaptation of this vaccine platform to several infectious agents, including SARS-CoV-2 with a universal approach to cover multiple variants.
  • LinKinVax is developing an innovative protein-based vaccine platform that can accelerate availability of vaccines by leveraging the research conducted at the Vaccine Research Institute (VRI).

InnoSkel Appoints Dr Sophie Amsellem-Bosq as Chief Technical Officer

Retrieved on: 
Tuesday, December 14, 2021

Nice, France, 14 December 2021 InnoSkel (the Company), a pioneering platform biotechnology company developing transformative therapies for the unmet needs of individuals with rare bone disorders, today announces the appointment of Sophie Amsellem-Bosq, PharmD, PhD as Chief Technical Officer (CTO).

Key Points: 
  • Nice, France, 14 December 2021 InnoSkel (the Company), a pioneering platform biotechnology company developing transformative therapies for the unmet needs of individuals with rare bone disorders, today announces the appointment of Sophie Amsellem-Bosq, PharmD, PhD as Chief Technical Officer (CTO).
  • Sophie has more than 20 years experience in cell and gene therapy across the entire value chain from discovery through clinical development and manufacturing, up to bedside.
  • She joins InnoSkel from advising and sharing her expertise with cell and gene therapy biotech companies and venture capital funds.
  • Sophie Amsellem-Bosq, PharmD, PhD, Chief Technology Officer at InnoSkel, added: InnoSkel is a pioneer in the development of innovative therapies for rare bone disorders.

TNFα-supplemented DL-4 culture system supports first-in-clinic Smart Immune ProTcell™ technology for immuno-deficient patients fighting cancers and infections

Retrieved on: 
Wednesday, September 29, 2021

T-cells are key to providing immunity to foreign pathogens and are indispensable in fighting life-threatening blood cancers, infections, and primary immunodeficiency diseases.

Key Points: 
  • T-cells are key to providing immunity to foreign pathogens and are indispensable in fighting life-threatening blood cancers, infections, and primary immunodeficiency diseases.
  • ProTcell, developed by Smart Immune SAS, is a new generation of allogenic cell and gene therapy designed to reset the patients immune system following allogeneic hematopoietic stem cell transplantation (HSCT) while reducing serious adverse events, morbidity, and mortality, thereby improving quality of life.
  • ProTcell is currently being evaluated in patients receiving an allogeneic HSCT for acute leukemia in an FDA-approved clinical trial.
  • ProTcell is currently being studies in two clinical trials in Europe, with two in the U.S. expected to start in Q4 2021.

Quantum Genomics Presents Results from Phase IIb QUORUM Study of Firibastat in Heart Failure at the European Society of Cardiology Meeting

Retrieved on: 
Friday, August 27, 2021

The QUORUM study demonstrates the efficacy and good safety profile of firibastat.

Key Points: 
  • The QUORUM study demonstrates the efficacy and good safety profile of firibastat.
  • In severe patients with an ejection fraction less than 50%, firibastat appears to be more effective than ramipril.
  • For this reason, QUORUMs phase IIb results pave the way for a phase III clinical study in severe patients whose protocol will be finalized with a selected partner pharmaceutical company.
  • Highlights from the phase IIb QUORUM study are included below:
    295 patients were enrolled in the study within 24 hours of a first myocardial infarction.