Molecular Therapy

Cabaletta Bio Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 9, 2023
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PHILADELPHIA, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today reported financial results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • In November 2023, Cabaletta announced the Company’s fourth IND application for CABA-201 was cleared by the FDA for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG).
  • Cabaletta will present new preclinical data for CABA-201 in a poster presentation and Cabaletta Bio Scientific Advisory Board members Carl June, M.D., and Georg Schett, M.D.
  • Cabaletta plans to participate in the following upcoming investor conferences:
    Stifel 2023 Healthcare Conference, which is being held from November 14-15, 2023 in New York, NY.
  • As of September 30, 2023, Cabaletta had cash, cash equivalents and short-term investments of $164.4 million, compared to $106.5 million as of December 31, 2022.

Neu REFIX β-glucan reduces muscle fibrosis in Duchenne muscular dystrophy MDX mice, in joint research with Dr. Yoshitsugu Aoki, NCNP, Tokyo, Japan.

Retrieved on: 
Tuesday, October 17, 2023
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This research was undertaken jointly with the team of Dr Yoshitsugu Aoki , Head, Dept.

Key Points: 
  • This research was undertaken jointly with the team of Dr Yoshitsugu Aoki , Head, Dept.
  • of Molecular Therapy, National Center for Neurology and Psychiatry (NCNP), Tokyo, Japan, a pioneer in developing solutions to DMD such as exon skipping therapy .
  • The findings published in Scientific Reports, a publication by Nature portfolio also reported a reduction in muscle inflammation, which precedes fibrosis and muscle dysfunction, leading gradually to respiratory muscle failure followed by lung dysfunction, cardiac fibrosis, heart failure and premature death.
  • With additional experiments in MDX mice underway , as more data evolves, that may be value adding to recommend Neu REFIX β-glucan to DMD patients, said Dr Yoshitsugu Aoki.

HDT Bio Publishes Peer-Reviewed Study Demonstrating Advantages of repRNA and LION™ Technology

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Monday, July 24, 2023
LION, Molecule, Vaccination, COVID-19, Molecular Therapy, Cancer, Infection, Publication, RNA, Mouse, LNP, Safety, Vaccine

The data were published in the journal Molecular Therapy, in an article entitled, "A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation."

Key Points: 
  • The data were published in the journal Molecular Therapy, in an article entitled, "A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation."
  • "While RNA vaccines have greatly curtailed the COVID-19 pandemic, legitimate safety concerns have arisen that must be taken into consideration," said Steve Reed, Ph.D., Chief Executive Officer of HDT Bio.
  • This is a clear demonstration that AMPLIFY is a next-generation platform with the potential to transform immunizations worldwide."
  • The publication can be found on the Molecular Therapy website and on the Publications page of the HDT Bio website.

Autolus Therapeutics Announces Publication in Molecular Therapy

Retrieved on: 
Tuesday, April 4, 2023
Acute leukemia, Antigen, CD19, Lymphoid leukemia, Molecular Therapy, CD22, CAR, Patient, B-ALL, Vaccine

LONDON, April 04, 2023 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced the publication of a paper in Molecular Therapy titled ‘Dual targeting of CD19 and CD22 against B-ALL using a novel high-sensitivity aCD22 CAR.’ 1

Key Points: 
  • CD22 is expressed early in B-cell development up until plasma cell differentiation and is expressed broadly by B-cell malignancies.
  • In this paper, the Autolus research team first develop a highly sensitive CD22 CAR which can recognize target antigen even if CD22 is expressed at low density.
  • Secondly, they explore a co-transduction approach with the clinically proven Autolus CD19 CAR, Obecabtagene autoleucel (obe-cel).
  • “CD22 targeting and CD19/CD22 co-targeting are challenging technical problems in the field,” said Martin Pule, Chief Scientific Officer, and Founder of Autolus.

ClearPoint Neuro Announces Exclusive Multi-Year Licensing Agreement with UCSF for Innovative Intra-Cerebral Cellular Delivery Platform

Retrieved on: 
Tuesday, March 28, 2023
Neurosurgery, Anatomy, Paper, Molecular Therapy, University of California, San Francisco, DSM-IV codes, Neurology, Brain, Cell, Doctor of Philosophy, MRI, MD, UCSF, GM, Software, Medical imaging, Medical device

Under the terms of the agreement, the Company will develop, obtain regulatory clearance, and commercialize in key geographies the intracerebral cellular delivery device designed by Dr. Lim.

Key Points: 
  • Under the terms of the agreement, the Company will develop, obtain regulatory clearance, and commercialize in key geographies the intracerebral cellular delivery device designed by Dr. Lim.
  • “We are delighted to partner with UCSF to bring this innovative delivery platform to our pharmaceutical partners working on cellular therapy.
  • We believe that, when combined with our ClearPoint system and software, Dr. Lim’s exciting cell delivery platform could potentially empower predicable and precise delivery of cells into different anatomical targets using various image-guidance technologies.
  • This innovative approach will allow cellular delivery at facilities that do not have access to MRI and will also expedite therapeutic delivery and ultimately patient recovery.”

HDT Bio Publishes Peer-Reviewed Preclinical Study Demonstrating Robust Immunological Response in Mothers and Newborns Following repRNA/LION™ Vaccination

Retrieved on: 
Friday, March 24, 2023
Vaccine, LION, Duke University, Molecule, Vaccination, HIV-1, Fetus, Infection, Molecular Therapy, Seattle Children's, Publication, Zika virus, Messenger, Risk, Child, Research institute, NIAID, Immunization, Virus, Safety, ZIKV

"The transmission of certain viruses from mother to developing fetus poses a significant risk to newborns," said Steven Reed, Ph.D., Chief Executive Officer of HDT Bio.

Key Points: 
  • "The transmission of certain viruses from mother to developing fetus poses a significant risk to newborns," said Steven Reed, Ph.D., Chief Executive Officer of HDT Bio.
  • Two viruses, HIV-1 and ZIKV, were chosen for the study due to their significant role in causing infections in newborns following mother-to-child transmission.
  • Furthermore, immunization in rabbit kits also led to a robust immunogenic response, regardless of the maternal vaccination status.
  • The publication can be found on the Molecular Therapy website and on the Publications page of the HDT Bio website.

Clovis Oncology Highlights Updated LuMIERE Phase 1 Data of Targeted Radiotherapy Candidate FAP-2286 at the 35th Annual EANM Congress

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Monday, October 17, 2022
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Clovis Oncology, Inc. (NASDAQ: CLVS) today announced an oral presentation at the 35th Annual European Association of Nuclear Medicine Congress (EANM) detailing updated Phase 1 data from the Clovis Oncology-sponsored Phase 1/2 LuMIERE clinical study (NCT04939610) investigating the safety, pharmacokinetics, dosimetry, and preliminary anti-tumor activity of its targeted radiotherapy candidate, FAP-2286 labeled with lutetium-177 (177Lu-FAP-2286).

Key Points: 
  • Clovis Oncology, Inc. (NASDAQ: CLVS) today announced an oral presentation at the 35th Annual European Association of Nuclear Medicine Congress (EANM) detailing updated Phase 1 data from the Clovis Oncology-sponsored Phase 1/2 LuMIERE clinical study (NCT04939610) investigating the safety, pharmacokinetics, dosimetry, and preliminary anti-tumor activity of its targeted radiotherapy candidate, FAP-2286 labeled with lutetium-177 (177Lu-FAP-2286).
  • The Phase 1 portion of the LuMIERE study is evaluating the safety of the investigational therapeutic agent 177Lu-FAP-2286 to identify the recommended Phase 2 dose and schedule.
  • The LuMIERE trial is the first prospective trial of a FAP peptide targeted radionuclide therapy and is currently in the dose escalation phase.
  • Updated results from the Phase 1 portion of the ongoing Phase 1/2 LuMIERE study found treatment-emergent adverse events (TEAEs) were mostly Grade 1 and 2 across cohorts.

AVROBIO Reports Favorable Data on Use of Combined State-of-the-art In Vitro Cell-based Assays to Identify Potential Genotoxicity Risk of Integrating Vectors During Preclinical Development

Retrieved on: 
Tuesday, October 11, 2022
Research, Genetics, Clinical Trials, Stem Cells, Biotechnology, Other Health, Health, Science, Oncology, LinkedIn, Data collection, Observation, AVRO, HSPC, Machine learning, Institute, European Society of Gene and Cell Therapy, EF1, Health, EFS, Stem cell, Cystinosis, Gaucher, Phrase, SAGA, Gene, HSC, IVIM, Compte rendu, Cell, Twitter, Cancer, Private Securities Litigation Reform Act, Industry, Clinical trial, Central nervous system, MECOM, ESGCT, Oncogene, Risk, Glycogen storage disease type II, Hospital, Growth, Hannover Medical School, Research, Division, Security (finance), Hunter syndrome, Lists of diseases, U.S. Securities and Exchange Commission, Algorithm, Gene expression, Experimental Hematology, Patient, Harvard Medical School, Nasdaq, MND, Safety, COVID-19, Molecular Therapy, Pharmaceutical industry, Vaccine

In collaboration with Professor Axel Schambach, Ph.D., Institute of Experimental Hematology, Hannover Medical School, Germany, AVROBIO is pioneering the use of two advanced preclinical cell-based assays -- in vitro immortalization (IVIM) assay and the novel surrogate assay for genotoxicity assessment (SAGA) -- to evaluate viral vectors before they move into clinical programs. Together these assays are designed to assess which vectors are less likely to exhibit genotoxic behavior and monitor if these vectors activate proto-oncogenes (genes that may lead to cancer).

Key Points: 
  • These assays enable preclinical risk assessment of gene therapy vectors, potentially paving the way for safer gene therapy vectors used in clinical trials.
  • Combining these assays helps assess vector genotoxicity risk early in preclinical development and further reaffirms that not all lentiviral vectors are designed the same.
  • In its research, AVROBIO used the two assays in combination to determine the potential genotoxicity risk of six lentiviral vectors.
  • Machine learning algorithms developed from transcriptional data of known genotoxic vectors are used to estimate the transformational potential of candidate vectors.

CARsgen Appoints Dr. Hua Jiang as Executive Director

Retrieved on: 
Tuesday, August 2, 2022
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SHANGHAI, Aug. 2, 2022 /PRNewswire/ --CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that Dr. Hua Jiang ("Dr. Jiang") has been appointed as Executive Director of the Company.

Key Points: 
  • SHANGHAI, Aug. 2, 2022 /PRNewswire/ --CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that Dr. Hua Jiang ("Dr. Jiang") has been appointed as Executive Director of the Company.
  • Dr. Jiang has contributed significantly to the early research, technology platform development, and innovative products development in CARsgen.
  • Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen, added, "On behalf of the Board, I would like to congratulate Dr. Jiang to be appointed as the Executive Director.
  • Dr. Hua Jiang, Executive Director, Vice President of Early Discovery of CARsgen, said, "I am pleased to become a member of the Board and would like to thank the Board for the recognition and trust.

Precision BioSciences Announces Publication in Molecular Therapy of ARCUS® In Vivo Gene Editing as a Promising Therapeutic Approach to Cure Chronic Hepatitis B Infection

Retrieved on: 
Wednesday, May 18, 2022
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Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced preclinical research from its ongoing in vivo gene editing program targeting hepatitis B virus (HBV) has been published online in Molecular Therapy.

Key Points: 
  • Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced preclinical research from its ongoing in vivo gene editing program targeting hepatitis B virus (HBV) has been published online in Molecular Therapy.
  • Our data suggest that LNP-delivered ARCUS mRNA is worth further exploration as a possible functional cure for chronic hepatitis B, said Derek Jantz, Chief Scientific Officer and Co-founder of Precision BioSciences.
  • Precisions gene editing program for chronic hepatitis B is designed to apply ARCUS to knock out persistent cccDNA and inactivate integrated hepatitis B genomes, potentially achieving durable HBsAg loss and functional cure.
  • ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery and control in mind.