Gene

Domain Therapeutics to Present Latest Data on DT-9045, a First-in-class Negative Allosteric Modulator of PAR2 for Immuno-oncology, at AACR 2024 Annual Meeting, Highlighting Clear Competitive Advantages of This Candidate

Retrieved on: 
Tuesday, March 26, 2024
Oncology, Health, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, GPCR, Gene, Immunotherapy, Patient, Immunosuppression, PAR2, ICB, Research, Tumor microenvironment, Cancer, G protein-coupled receptor, IND, AACR, Gq alpha subunit, Therapy, Drug resistance, Module (mathematics)

PAR2 is one of the genes most significantly linked to resistance against immune checkpoint blockage (ICB) and T cells dysfunction in cancer patients.

Key Points: 
  • PAR2 is one of the genes most significantly linked to resistance against immune checkpoint blockage (ICB) and T cells dysfunction in cancer patients.
  • Its upregulation across a variety of cancer types and expression on diverse cells within the tumor microenvironment underscore PAR2’s critical role in cancer development.
  • Remarkably, when compared to its most advanced competitors positioned in other therapeutic areas, this candidate has demonstrated clear differentiated features.
  • Stephan Schann, Chief Scientific Officer of Domain Therapeutics, commented: “We are excited to present our newest findings on DT-9045 at AACR.

bluebird bio Reports Fourth Quarter and 2023 Annual Results and Highlights Operational Progress and 2024 Guidance

Retrieved on: 
Tuesday, March 26, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, PIN, Sickle cell disease, MSA, Bluebird bio, Telephone, Webcast, Patient, Gene, CMMI, QTC, Bank statement, Thalassemia, Annual report, Medicaid, Cell, Medicare, Bluebird, FDA, Pharmaceutical industry

bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported fourth quarter and annual financial results and business highlights for the year ended December 31, 2023, including recent commercial and operational progress.

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported fourth quarter and annual financial results and business highlights for the year ended December 31, 2023, including recent commercial and operational progress.
  • “In 2023, bluebird established a validated, commercial gene therapy strategy that brought ZYNTEGLO and SKYSONA to individuals living with beta-thalassemia and cerebral adrenoleukodystrophy.
  • Completed 2 patient starts for SKYSONA since the beginning of 2024, in addition to 6 patient starts completed for SKYSONA in 2023.
  • In the first quarter of 2024, bluebird signed its first Medicaid outcomes-based agreement for LYFGENIA with the state of Michigan.

eGenesis Announces World’s First Successful Transplant of Genetically Engineered Porcine Kidney in a Living Patient

Retrieved on: 
Thursday, March 21, 2024
Research, Other Health, General Health, Oncology, Hospitals, Genetics, Surgery, Clinical Trials, Science, Biotechnology, FDA, Health, Other Science, Pelvic floor dysfunction, MGH, Kidney disease, Gene, Organization, Survival, Massachusetts General Hospital, Transplant, Inflammation, Immunosuppression, Kidney failure, Mortality, Coagulation, Hope, Doctor of Philosophy, Expanded access, Quality of life, Genome, Transgene, Hospital, Kidney, Dialysis, CD40L, Courage, Kidney transplantation, Medicine, Dentistry

eGenesis , a biotechnology company developing human-compatible engineered organs to address the global organ shortage, today announced the first ever transplantation of a genetically engineered porcine kidney into a living human recipient.

Key Points: 
  • eGenesis , a biotechnology company developing human-compatible engineered organs to address the global organ shortage, today announced the first ever transplantation of a genetically engineered porcine kidney into a living human recipient.
  • Following the procedure, the patient is in good condition and recovering well at MGH.
  • Yet the demand for organs far outpaces supply, with more than 90,000 individuals on the kidney waitlist and approximately 25,000 kidney transplants performed each year.
  • Decades of progress in cross-species transplantation, accelerated by the advancement of modern genome editing tools and next-generation sequencing, have enabled eGenesis to progress genetically engineered organs to the clinical setting.

Profluent Secures $35M in Additional Funding and Key Industry Experts to Scale Foundational AI Models for Biomedicine and Tackle First Vertical in Gene Editing

Retrieved on: 
Thursday, March 21, 2024
Other Health, Software, Research, Pharmaceutical, Technology, Artificial Intelligence, Genetics, Health Technology, Science, Biotechnology, Other Science, Health, AIX, Cas9, OpenAI, Gene editing, Insight Partners, Growth, Spotlight, General partnership, Salesforce, Nature Biotechnology, Gene, Spark Capital, AI, University, Tome, Nature, Protein engineering, Biology, Microbiology, CRISPR, De novo, ChatGPT, Therapy, Machine learning, Probability, Management

Profluent, an AI-first protein design company, today announced the close of additional funding to bring the total raised to $44M.

Key Points: 
  • Profluent, an AI-first protein design company, today announced the close of additional funding to bring the total raised to $44M.
  • “Our research at the forefront of AI has enabled Profluent to create large language models that begin to learn the blueprint of nature,” said Ali Madani, Profluent co-founder and Chief Executive Officer.
  • By first applying its platform to gene editing, the company showcases AI as the ideal interface for engineering complex biological systems.
  • Just as ChatGPT can write compelling essays and emails, Profluent has developed AI that designs entirely new and functional proteins.

Demeetra and Indiana University Biopesticide Platform Combining Gene Editing and Synthetic Biology Selected as a MDPI Journal Editor’s Choice Article

Retrieved on: 
Wednesday, March 20, 2024
Science, Other Science, Biotechnology, Research, Health, University, Genetics, Education, MDPI, Generation, Gene editing, Medicine, Saccharomyces cerevisiae, Transposase, Synthetic biology, Sterilization, RNA, Gene, Female, Indiana University, Engineering, Disease, Male, Mosquito control, Plant, Publication, Transgene, SIT, RNA interference, Therapy, Multimedia, Cas9

A pioneering study, authored by scientists from Demeetra and Indiana University, has been selected for the MDPI Journals Editor's Choice Articles , highlighting the impact of advanced gene editing in synthetic biology to produce biopesticides.

Key Points: 
  • A pioneering study, authored by scientists from Demeetra and Indiana University, has been selected for the MDPI Journals Editor's Choice Articles , highlighting the impact of advanced gene editing in synthetic biology to produce biopesticides.
  • View the full release here: https://www.businesswire.com/news/home/20240320823585/en/
    Demeetra and Indiana University research on gene edited yeast biopesticide was featured on the front cover of Fermentation.
  • According to MDPI “Editor’s Choice articles are based on recommendations by the scientific editors of MDPI journals from around the world.
  • “We wanted to engineer the ideal yeast platform for stable RNAi expression,” said Dr. Corey Brizzee, Director of Gene Editing at Demeetra.

World-Renowned Ancient DNA Expert, HHMI Investigator, and MacArthur Award Winner Beth Shapiro, Ph.D., Joins Colossal as Chief Science Officer

Retrieved on: 
Tuesday, March 19, 2024
Environment, Research, Genetics, Environmental Issues, Environmental Health, Biotechnology, Health, Other Science, Science, Genomics, Gene, Executive Committee, National Geographic Explorer, Woolly mammoth, Cell, University, Pennsylvania State University, Television, Life, DNA, Stanford University, CRISPR, Ice, Vertebrate Genomes Project, Hologenomics, Genome, HHMI, Colossal Biosciences, Balliol College, Oxford, Howard Hughes Medical Institute, Ecology and Evolutionary Biology, CSO, Domestication, Natural history, National academy, American Academy, Biology, Searle, George Church, Multimedia, MacArthur Fellows Program, Hunting

Colossal Biosciences, the world’s first de-extinction company, today announced that Beth Shapiro, Ph.D. , internationally renowned evolutionary molecular biologist, leader in paleogenomics, and ancient DNA expert, has joined as Chief Science Officer.

Key Points: 
  • Colossal Biosciences, the world’s first de-extinction company, today announced that Beth Shapiro, Ph.D. , internationally renowned evolutionary molecular biologist, leader in paleogenomics, and ancient DNA expert, has joined as Chief Science Officer.
  • In this role, she will oversee the continued expansion of the company’s de-extinction and conservation science teams.
  • A MacArthur “Genius Grant” Award winner and National Geographic Explorer, Beth Shapiro is a pioneer in the scientific fields of ancient DNA and paleogenomics.
  • “She’s the author of inspiring books 'How to Clone a Mammoth' and 'Life as We Made It,' plus an HHMI and MacArthur Fellow.

Revvity Introduces New Workflow to Accelerate Newborn Sequencing Research

Retrieved on: 
Friday, March 15, 2024
Research, Medical Devices, Medical Supplies, Baby, Maternity, Genetics, Health Technology, Health, Consumer, Science, Quality control, SVP, Workflow, FACMG, NGS, Element, Database, Gene, Collection, RUO, Doctor of Philosophy

Revvity, Inc. today announced the introduction of a flexible end-to-end workflow solution for newborn research, enabling users to utilize different instruments, reagents and databases based on a lab’s needs.

Key Points: 
  • Revvity, Inc. today announced the introduction of a flexible end-to-end workflow solution for newborn research, enabling users to utilize different instruments, reagents and databases based on a lab’s needs.
  • “This new workflow from Revvity seeks to elevate the customer experience by leveraging a rich variant database for newborn sequencing research and providing critical components necessary to be able to go from sample to result,” said Madhuri Hegde, PhD, FACMG, SVP and chief scientific officer, Revvity.
  • “We are accelerating the democratization of genomic sequencing by addressing the typical challenges faced by labs.”
    The research-use only (RUO) offering from Revvity culminates in analysis and report, covering essential steps in the sequencing process.
  • The solution enables identification of variants in more than 350 genes, complemented by a large database of carefully pre-curated variants.

New Study Shows that the Drug-Drug Interactions and Lifestyle Factors Provided by Castle Biosciences’ IDgenetix® Test Significantly Improve Medication Recommendations Over Drug-Gene Interactions Alone for Patients 65 and Older

Retrieved on: 
Friday, March 15, 2024
Science, Other Science, Biotechnology, Pharmaceutical, Oncology, Health, Mental Health, Genetics, Diagnosis, Depression, Gene, Schizophrenia, Post-traumatic stress disorder, Patient, Research, Bipolar disorder, Anxiety, Personality disorder, Polypharmacy, Pain, Geriatric psychiatry, Annual general meeting, Pharmaceutical industry

The data will be shared via a poster presentation at the 2024 American Association for Geriatric Psychiatry (AAGP) Annual Meeting, taking place March 15-18 in Atlanta.

Key Points: 
  • The data will be shared via a poster presentation at the 2024 American Association for Geriatric Psychiatry (AAGP) Annual Meeting, taking place March 15-18 in Atlanta.
  • “This study found that, despite all patients carrying potentially clinically significant genetic variants, one-third of medication recommendations were due to drug-drug interactions and lifestyle factors provided on the IDgenetix report.
  • Additionally, 58% of the patients in the study were on five or more medications, with an average of seven, at the time of testing.
  • All patients in the study had genetic polymorphisms that could impact medication selection in at least three of the 15 genes on the IDgenetix report.

Sensorion Announces its Participation in the Annual Bioprocessing Summit Europe

Retrieved on: 
Friday, March 15, 2024
Research, Genetics, Clinical Trials, Health Technology, Biotechnology, Other Health, Health, Other Science, Science, CMC, Gene, EDT, Doctor of Philosophy

Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announces its participation in the Bioprocessing Summit Europe, taking place on March 19-21, 2024, in Barcelona, Spain.

Key Points: 
  • Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, today announces its participation in the Bioprocessing Summit Europe, taking place on March 19-21, 2024, in Barcelona, Spain.
  • Christine Le Bec, PhD, Head of CMC Gene Therapy, will chair a session entitled “Advancing Technical Development of Gene Therapies”, on March 19, 2024, at 8.25am CET (3.25am EDT).
  • Christine will also make the following presentation “Manufacturing Challenges and Control Strategies for Dual AAV Vectors”, on March 19, 2024, at 2.50pm CET (9.50am EDT).

Charles River and Navega Therapeutics Announce Comprehensive Gene Therapy Manufacturing Collaboration

Retrieved on: 
Thursday, March 14, 2024
Research, Medical Devices, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Erythromelalgia, AAV, Diabetic neuropathy, CDMO, Patient, CRL, Good manufacturing practice, Gene, DNA, CAP, Chronic pain, Doctor of Philosophy, GMP, Navega, Therapy, Peripheral neuropathy, CGT, Pharmaceutical industry

Charles River Laboratories International, Inc. (NYSE: CRL) and Navega Therapeutics , Inc., a biotechnology company developing epigenetic gene therapies, today announced an AAV9 production program agreement.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) and Navega Therapeutics , Inc., a biotechnology company developing epigenetic gene therapies, today announced an AAV9 production program agreement.
  • As part of Charles River’s Cell and Gene Therapy (CGT) Accelerator Program (CAP), Navega will have access to established contract development and manufacturing (CDMO) capabilities and advisory services to produce an adeno-associated virus (AAV)-based gene therapy, NT-Z001, for Phase I clinical trials.
  • To bring NT-Z001 to clinic, Navega will leverage Charles River’s off-the-shelf plasmid products, custom plasmid capabilities, and Good Manufacturing Practice (GMP)-grade AAV production.
  • Join Ana Moreno, CEO, Navega Therapeutics on March 19 in San Francisco – see the full agenda and save your seat: https://bit.ly/3SW3VOV
    “The collaboration with Navega will tap into our premier gene therapy CDMO capabilities and robust AAV offerings.