Editing Technology to Identify and Advance Genetic Dysfunction Therapies

BlueAllele | July 27, 2020

BlueAllele, a biotechnology company focused on enhancing the quality and precision of gene editing systems for patients with genetic diseases, announced the launch of PALIDON™, an innovative and proprietary repair template technology. Many rare genetic diseases are caused by mutations within a single gene. However, the location of the mutation often varies for different individuals with the same disease. Conventional gene editing approaches are primarily limited to correcting small regions or even single locations. Additionally, repair templates have failed to achieve sufficient integration efficiency in vivo needed to sustain therapeutic thresholds and durable efficacy. BlueAllele's PALIDON repair templates were designed to specifically overcome both of these challenges. PALIDON's "bi-directional" templates use a reverse-complementary structure that is designed to leverage the high rate of non-homologous end joining (NHEJ) repair observed in cells while preserving the ability to achieve precise correction of damaged mRNA and protein. PALIDON templates are designed to be compatible with existing gene editing tools (e.g., CRISPR, zinc-finger nucleases) and delivery systems, making them particularly suitable for targeting a variety of genetic diseases.

Spotlight

The reason for the rapid expansion and development of the therapeutic antibody sector is the capacity of monoclonal antibodies to precisely bind a target antigen and neutralise or promote its activity. The efficacy and safety.

Spotlight

The reason for the rapid expansion and development of the therapeutic antibody sector is the capacity of monoclonal antibodies to precisely bind a target antigen and neutralise or promote its activity. The efficacy and safety.

Related News

INDUSTRIAL IMPACT, MEDICAL

Celularity and CH Trading Group Declares Territory Distribution Agreement

Celularity | January 17, 2023

Celularity Inc., a biotechnology firm based in the United States that develops placental-derived allogenic cell treatments and biomaterials, and CH Trading Group LLC, an international import, export, and trading corporation, have agreed to an exclusive territorial distribution agreement. CH Trading Group will serve as the exclusive territorial distributor for halal-certified products previously announced by Celularity in over 100 countries. The product distribution agreement for halal-certified products by Celularity provides a five-year minimum accumulated purchase commitment of US $225 million. CEO of CH Trading Group, Sayed Zayan, said, “Based on market demand for Celularity’s commercial biomaterial products, we believe there is a billion-dollar commercial opportunity across the Middle East and North Africa Islamic markets.” He also added, “We are excited to be in a position to expand access to these important medicines and wellness products to more people across the region through our exclusive territories distributor agreement with Celularity.” (Source – Business Wire) “According to Emergent Research's most recent analysis, the global regenerative medicine market was valued at $9.80 billion in 2021 and is predicted to expand at a CAGR of 15.9%, reaching $37.10 billion in 2030.” Under the agreement, CH Trading Group will distribute products to more than 100 countries that are members of or affiliated with the following intergovernmental organizations (‘Islamic Markets’) The Gulf Cooperation Council: Consists of Bahrain, Oman, Kuwait, Qatar, Saudi Arabia, and the UAE. The Organization of Islamic Cooperation: It spans four continents and includes 57 member nations The African Union: Includes 55 member countries Robert J. Hariri, M.D., Ph.D., Celularity’s CEO, Chairman and Founder, said, “Our partnership with the CH Group represents another critical milestone towards bringing our innovative halal-certified—under globally recognized Circle H International Inc. standards – regenerative biomaterial products to treat degenerative disease across Islamic Markets.” (Source – Business Wire) About Celularity Headquartered in Florham Park, N.J., Celularity is a clinical-stage biotechnology firm paving the way for the future of cellular medicine by offering off-the-shelf allogeneic placenta-derived cellular therapeutics with unmatched scale, quality, and affordability. With its novel approach to cell therapy, the company can unlock the unique therapeutic potential of the postpartum placenta. In addition, through the placenta, nature’s immunotherapy engine, the firm is driving the next generation of cellular medicine using placenta-derived T cells, NK cells, and pluripotent stem cells to address unmet and underserved clinical needs in cancer, infectious, and degenerative diseases.

Read More

MEDTECH, INDUSTRIAL IMPACT

Janux Therapeutics Submits IND Application for JANX008

Janux | December 29, 2022

Janux Therapeutics, Inc. is a clinical-stage biopharmaceutical company that uses its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms to develop a diverse pipeline of novel immunotherapies. On December 27, 2022, the company announced that it had submitted an investigational new drug (IND) application for JANX008 to U.S. Food and Drug Administration, an EGFR-TRACTr in (SCCHN). The company is pleased to announce its second IND. It considers this a big step forward for its TRACTr platform, which has also helped many next-generation immunotherapies move forward. JANX008 is made in a particular way to avoid cytokine release syndrome and the known harmful effects of EGFR in on-target healthy tissue. As a result, the company thinks it could be easier to treat EGFR-expressing cancers in many ways. JANX008 is a double-masked TRACTr that conceals both the EGFR-binding and T-cell-specific binding domains (CD3). JANX008 exhibited minor cytokine release syndrome and healthy tissue toxicity in preclinical studies. Besides, it demonstrated superior safety and pharmacokinetic (PK) qualities compared to unmasked TCEs. These data and JANX008's strong manufacturability qualities justify its continued development as a potential therapy for solid tumors. About theJanux Janux Therapeutics is a clinical-stage business developing immunotherapies that target cancers without affecting healthy tissue. The company uses platform masking to generate innovative therapeutic candidates that address T-cell engager toxicity and efficacy limitations. It develops next-generation cancer therapeutics using TRACTR platform. Janux Therapeutics focuses on developing medically-validated T cell engagers (TCEs). Its pipeline includes PSMA, EGFR, and TOP2 efforts. All of its programs are IND-enabling or discovery.

Read More

CELL AND GENE THERAPY, INDUSTRIAL IMPACT

CBM, Stoic Bio and Nucleus Biologics Announce Strategic Alliance

The Center for Breakthrough Medicines | March 13, 2023

Nucleus Biologics and Stoic Bio have teamed up to form a supply agreement with the Center for Breakthrough Medicines (CBM), a contract development and manufacturing organization that aims to accelerate the development and manufacture of advanced therapies. Under the agreement, Nucleus Biologics will become CBM's selected supplier of cell culture media and other biological solutions, ensuring a consistent supply of this critical material with tighter quality control measures. Nucleus Biologics' approach to cell culture media, which sees it as a critical raw material that can drive desired attributes of cells crucial to the success of cell and gene therapy research, is complementary to CBM's focus on transparency, technology, and end-to-end solutions. By working together, they hope to expedite cell and gene therapy breakthroughs. In addition, CBM will be an early adopter of Stoic Bio's innovative family of point-of-use media makers, Krakatoa™, which allows scientists to manufacture their own media with 65% fewer CO2 emissions than conventional media. By demonstrating that developing and manufacturing lifesaving therapies doesn't have to come with a high environmental cost, CBM hopes to set an example for the industry. With over 2,200 cell and gene therapies in development, collaborations like this one are needed to speed up the process of getting these therapies to the clinic and ultimately approved. The CBM, Stoic Bio and Nucleus Biologics collaboration aim to create a unique service offering that introduces a paradigm shift in development and manufacturing, showing how technology and sustainability can converge to make better therapies sustainably. About The Center for Breakthrough Medicines The Center for Breakthrough Medicines (CBM) is a CDMO focused on accelerating the development and manufacture of advanced therapies to save lives. It offers end-to-end solutions such as process development, manufacturing, and quality control for cell and gene therapies, gene editing, and immunotherapies. In addition, CBM collaborates with pharmaceutical companies, academic institutions, and healthcare providers to make advanced therapies more accessible while ensuring compliance with regulatory standards. The company also prioritizes sustainability by implementing green initiatives and sustainable technology and strives to positively impact the communities it operates in.

Read More