CRISPR

MaxCyte Signs Strategic Platform License with Kamau Therapeutics to Accelerate the Development of Cell Therapies for Genetic Diseases

Retrieved on: 
Monday, September 16, 2024
SCD, HDR, Cell engineering, Research, SPL, DNA, Patient, Therapy, CRISPR, Doctor of Philosophy, Gene editing, Partnership, MD, Sickle cell disease, Pharmaceutical industry

Under the terms of the agreement, Kamau obtains non-exclusive research, clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform.

Key Points: 
  • Under the terms of the agreement, Kamau obtains non-exclusive research, clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform.
  • HDR now forms the basis for Kamau’s lead investigational program, nula-cel, which is in clinical development for SCD.
  • “By partnering with us, Kamau gains access to our commercially validated Flow Electroporation technology as well as technical, regulatory and scientific support.
  • Kamau Therapeutics is MaxCyte’s 29th clinical/commercial partnership overall; each partnership generates pre-commercial milestone revenue, the vast majority of which includes program-related revenue.

KFSHRC Pioneering Ethical Innovation in Global Healthcare

Retrieved on: 
Friday, September 13, 2024
Research, Hospital, Bias, Telehealth, AI, CRISPR, Genomics, Ethics committee, WHO, Heart, Organization, Health, Harvard University, ANFAL, KFSHRC, Developed country, World Health Organization, GAIN, Medical device

RIYADH, Saudi Arabia, Sept. 13, 2024 (GLOBE NEWSWIRE) -- King Faisal Specialist Hospital & Research Centre (KFSHRC) has reiterated its commitment to ethical innovation in its efforts to champion medical research and patient care. KFSHRC ensures that its integration of novel technologies, such as AI and CRISPR, is guided by rigorous ethical frameworks prioritizing transparency, accountability, and patient welfare.

Key Points: 
  • KFSHRC ensures that its integration of novel technologies, such as AI and CRISPR, is guided by rigorous ethical frameworks prioritizing transparency, accountability, and patient welfare.
  • At the heart of KFSHRC’s commitment to ethical healthcare is its Research Ethics Committee.
  • By safeguarding participants’ rights and welfare through informed consent and risk minimization, the committee reassures stakeholders about the hospital's unwavering commitment to ethical healthcare.
  • Additionally, in the same year, Newsweek magazine ranked it among the world's best 250 hospitals, further solidifying its position as a global leader in healthcare.

Scribe Therapeutics to Participate in Upcoming Conferences

Retrieved on: 
Thursday, September 12, 2024
Biotechnology, Health, Genetics, Health Technology, Cardiology, CRISPR, Conference, Arizona Biltmore Hotel, Mesa, MST, Pharmaceutical industry

Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced that the Company will participate in the following conferences in September and October:

Key Points: 
  • Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced that the Company will participate in the following conferences in September and October:
    Cantor Global Healthcare Conference: Members of management will be participating in the event on Thursday, September 19, 2024 in New York, NY.
  • They will also be available for one-on-one meetings.
  • BofA Healthcare Trailblazers Private Company Conference: Members of management will be participating and available for one-on-one meetings on Wednesday, September 25, 2024 in Boston, MA.
  • Svetlana Lucas, Ph.D., Chief Business Officer, and David Parrot, Chief Financial Officer, will also be available for one-on-one meetings.

Intellia Therapeutics to Present Data from the Phase 2 Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the 2024 ACAAI Annual Scientific Meeting

Retrieved on: 
Thursday, September 12, 2024
ACAAI, Therapy, Webcast, CRISPR, Randomness, NTLA, Intellia Therapeutics, HAE, Hereditary angioedema, Medicine, American College

NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks.

Key Points: 
  • NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks.
  • Title: Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema
    Date and Time: Saturday, October 26, 2024, from 4:30 – 6:30 p.m.
  • ET
    Intellia will host a live webcast on Monday, October 28, 2024, at 8:00 a.m.
  • A replay of the webcast will be available on Intellia’s website for at least 30 days following the call.

Halberd Partnership Secures Contract from Defense Atomics Corporation to Address Brain Injury and PTSD

Retrieved on: 
Wednesday, September 11, 2024
HALB, Suicidal ideation, Research, CKD, Hospital, Blood, Patient, Safety, B cell, Physician, Prevalence, FDA, TBI, Growth, Chronic kidney disease, CRISPR, ATP, Diagnosis, OTC, LDX, Depression, Market, Diabetes, Anxiety, Addiction, Brain, Traumatic brain injury, Partnership, Defense, Veteran, PTSD, Post-traumatic stress disorder, NRT, Pharmaceutical industry

Defense Atomics Corporation agreed to use its proprietary CRISPR/Nanotechnology Stem Cell treatment for PTSD/TBI in a minimum of 10 paid veterans in a government pilot study of this cutting-edge protocol.

Key Points: 
  • Defense Atomics Corporation agreed to use its proprietary CRISPR/Nanotechnology Stem Cell treatment for PTSD/TBI in a minimum of 10 paid veterans in a government pilot study of this cutting-edge protocol.
  • Based on Defense Atomics’ success using CRISPR methodology with overseas patients, this study is intended to substantiate the benefits of this technology in conjunction with Halberd/Athena’s LDX PTSD and TBI/Brain Injury protocol.
  • The WatchDawg monitoring allows us to acquire near real time (NRT) objective data with respect to the administration of CRISPR.
  • This product is useful for myriad disorders from PTSD, anxiety, depression, anger and addiction, to Chronic Covid, Alzheimer’s, Chronic Kidney Disease (CKD), diabetes, and others.

TFF Pharmaceuticals Partners with Emory University and BARDA to Develop a Dry Powder Inhaled mRNA-based Treatment for Influenza and COVID

Retrieved on: 
Wednesday, September 11, 2024
ASPR, CRISPR, Emory University, Biomedical engineering, HHS, Administration, Therapy, COVID, SARS-CoV-2, Public, TFF, Partnership, Temperature, Development, Biomedicine, Vaccine

FORT WORTH, Texas, Sept. 11, 2024 (GLOBE NEWSWIRE) -- TFF Pharmaceuticals, Inc (Nasdaq: TFFP) (the “Company” or “TFF Pharmaceuticals”), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative drug products based on its patented Thin Film Freezing (TFF) technology platform, today announces its new partnership with Emory University and the Biomedical Advanced Research and Development Authority (BARDA). Under the partnership, TFF Pharmaceuticals will test the feasibility of converting Emory’s mRNA-based Cas13a antiviral against influenza A and B and SARS-CoV-2 into a dry powder formulation for more precise inhalational delivery, improved stability and widespread distribution without the need for cold-chain storage.

Key Points: 
  • Under the partnership, TFF Pharmaceuticals will test the feasibility of converting Emory’s mRNA-based Cas13a antiviral against influenza A and B and SARS-CoV-2 into a dry powder formulation for more precise inhalational delivery, improved stability and widespread distribution without the need for cold-chain storage.
  • The competitively awarded Easy Broad Agency Announcement (EZ-BAA) contract supports early-stage therapeutic platform development under BARDA’s Flexible and Strategic Therapeutics (FASTx) program.
  • Our partnership with Emory University and BARDA is an important opportunity to deploy our TFF technology to combat rapidly changing respiratory viruses, including influenza and COVID,” said Dr. Harlan Weisman, TFF Pharmaceuticals’ Chief Executive Officer.
  • With ever-changing influenza and COVID strains emerging, flexibility of delivery without the need for cold chain storage and distribution is all the more important.

Excision BioTherapeutics Announces Oral Presentation at the 2024 International HBV Meeting

Retrieved on: 
Monday, September 9, 2024
International, RNA, SAN, Hepatitis, CRISPR, Hepatitis B virus, Vaccine

SAN FRANCISCO, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc. (“Excision”, the “Company”), a clinical-stage biotechnology company developing CRISPR-based therapies to cure serious latent viral infectious diseases, today announced that it will present data from its program for Hepatitis B virus, EBT-107, at the 2024 International HBV Meeting, taking place September 11-15, 2024, in Chicago, IL.

Key Points: 
  • SAN FRANCISCO, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Excision BioTherapeutics, Inc. (“Excision”, the “Company”), a clinical-stage biotechnology company developing CRISPR-based therapies to cure serious latent viral infectious diseases, today announced that it will present data from its program for Hepatitis B virus, EBT-107, at the 2024 International HBV Meeting, taking place September 11-15, 2024, in Chicago, IL.
  • EBT-107 is a CRISPR-based gene therapy that is being developed as a potential cure for Hepatitis B. EBT-107 uses dual guide RNAs to effectively deactivate the virus and prevent the emergence of escape variants.
  • The details of the presentations are below:

Gladstone Institutes Launches Capital Campaign to Accelerate a Biomedical Revolution

Retrieved on: 
Thursday, September 5, 2024
Diabetes, Acute coronary syndrome, Disease, Biotechnology, SAN, Science, FDA, AI, CRISPR, Trustee, MD, Data science, Srivastava, Alzheimer's disease, Gap, Regenerative medicine, Computational biology, Gladstone Institutes, Medicine, Intelligence, Research, NOW, Immunology, Patient, Cardiovascular disease, Parkinson's disease, Virology, Neuroscience, HIV, Heart failure, Cancer, Pharmaceutical industry

SAN FRANCISCO, Sept. 5, 2024 /PRNewswire/ -- Citing the arrival of a "revolutionary moment in science and medicine," Deepak Srivastava, MD, president of Gladstone Institutes, announced a five-year, $350 million campaign to add more than 300 scientists and 15 state-of-the-art laboratories, develop the next generation of biotechnologies, and advance discoveries to the clinic as rapidly as possible.

Key Points: 
  • The $350 million Gladstone NOW campaign will raise funds to expand Gladstone's footprint, hire hundreds of additional scientists, and seize emerging opportunities for high-impact biomedical research that can overcome disease.
  • "Because of breakthroughs made in the last decade, we no longer have to accept disease," Srivastava says.
  • "We are working on exceptionally complex and difficult problems," says Robert Wicks, CEO of the Gladstone Foundation.
  • "I want to thank our trustees and the members of the Gladstone Foundation Board for their advice and guidance as we launch the institution's first complex capital campaign.

Leapfrog Bio Announces Publication in Nature PJ Precision Oncology Validating its Proprietary Precision PGx Platform™

Retrieved on: 
Thursday, September 5, 2024
Neoplasm, Cancer, PARP, Gene, Survival, Leapfrog, SAN, Biology, CRISPR, Disease, Therapy, Precision, Cell, Mutation, NGS, FDA, CSO, Patient, Lof, ADP, Pharmaceutical industry

SAN MATEO, Calif., Sept. 05, 2024 (GLOBE NEWSWIRE) -- Leapfrog Bio, a clinical-stage precision oncology company, accelerating oncology drug development by identifying novel therapies for undruggable cancer-driving mutations, today announced a publication in Nature PJ Precision Oncology describing and validating its Precision PGx Platform™, a novel pharmacogenomic approach to screening and discovering drug-genotype combinations that are clinically actionable and demonstrate the potential to significantly impact overall survival for cancer patients. The platform evaluates cancers resulting from LoF mutations and leverages real driver biology to expose hidden vulnerabilities that accompany these mutations and finds the drugs that exploit those vulnerabilities to destroy cancer cells.

Key Points: 
  • SAN MATEO, Calif., Sept. 05, 2024 (GLOBE NEWSWIRE) -- Leapfrog Bio, a clinical-stage precision oncology company, accelerating oncology drug development by identifying novel therapies for undruggable cancer-driving mutations, today announced a publication in Nature PJ Precision Oncology describing and validating its Precision PGx Platform™, a novel pharmacogenomic approach to screening and discovering drug-genotype combinations that are clinically actionable and demonstrate the potential to significantly impact overall survival for cancer patients.
  • They then applied the platform to approved cancer drugs for which real-world data was available and showed that platform-discovered genetic sensitizers impacted patient survival.
  • “This was a pivotal moment for me,” says Tomas Babak, CSO of Leapfrog Bio and corresponding author.
  • After screening only a small portion of these molecules, Leapfrog has identified several compelling opportunities and has filed corresponding intellectual property applications around the discoveries.”

Global Coronavirus Therapeutics Market Size Projected to Exceed $16 Billion By the End of 2031

Retrieved on: 
Wednesday, September 4, 2024
Coronavirus, DNA, Biotechnology, COVID, Sunlight, Drug design, Therapeutic index, MHRA, Intuition, Mutation, MA10, TCE, Risk, Patient, Infection, PALM, IC50, Sanofi, SARS-CoV-2, Science, VOC, Vaccine, PFE, Plasma, Tumor microenvironment, University, Overalls, COVID-19, Medication, Moderna, Food, Journal, Immune system, BLA, BioNTech, MBA, Compound, News, BNTX, NHS, Vir, Health care, Immunodeficiency, Toxicity, CRISPR, Medicinal chemistry, Pfizer, Therapy, Hospital, FDA, Public, Cytokine, Trichloroethylene, MRNA, Prognosis, CDC, Research, Vaccination, Pharmacy, RRP, Cancer, Breakthrough therapy, PSMA, Disease, Messenger, Breakthrough, Formula

A report from Transparency Market Research projects that the global COVID-19 therapeutics market size is projected to reach more than US$ 16.2 Bn by the end of 2031.

Key Points: 
  • A report from Transparency Market Research projects that the global COVID-19 therapeutics market size is projected to reach more than US$ 16.2 Bn by the end of 2031.
  • In a relatively short amount of time, emerging technologies, such as CRISPR and mRNA, have made it possible to develop new medicines.
  • Transparency Market Research continued: “Demand for COVID-19 treatments may be influenced by the ongoing coronavirus mutational alterations that are currently affecting parts of the world, including China.
  • Governments across the globe have taken steps to guarantee that people have access to COVID-19 treatments, which has further fueled the COVID-19 therapeutics industry.