In a noteworthy development in the pharmaceutical industry, SiSaf Ltd, a renowned RNA delivery and therapeutics firm, recently announced that the U.S. FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation to its novel siRNA therapeutic, SIS-101-ADO, for the treatment of Autosomal Dominant Osteopetrosis Type 2 (ADO2).

The pharmaceutical industry is undergoing a significant shift as digital transformation in pharma continues to reshape the landscape. Bruker Corporation, a leading provider of high-performance scientific instruments and solutions, recently announced its acquisition of ZONTAL Inc., a pioneering platform for the digital transformation of analytical laboratories and integrated biopharma data solutions. This strategic move strengthens Bruker's Integrated Data Solutions (IDS) software division, which includes Mestrelab Research, Arxspan, and Optimal.

Myricx Pharma, an oncology drug discovery company specializing in precision medicines based on its N-myristoyltransferase (NMT) platform, has recently presented positive pre-clinical proof of concept data at the American Association for Cancer Research's Annual Meeting (AACR 2023). The company unveiled its novel antibody drug conjugate (ADC) programme, demonstrating the potential of Myricx's NMT inhibitors (NMTi) as a unique ADC payload.

Ultima Genomics and Genome Insight have forged a partnership with the objective of providing cost-effective whole genome sequencing solutions to individuals with cancer. As part of this collaboration, Genome Insight will join the early access program for Ultima's high-throughput next-generation sequencing (NGS) instrument, the UG 100™. Additionally, Genome Insight will develop an optimized version of its artificial intelligence-powered whole genome bioinformatics solution, tailored to Ultima's sequencing technology. The integration of Genome Insight's proprietary AI-driven bioinformatics with Ultima's affordable sequencing aims to make whole genome sequencing more accessible for cancer patients.

Portal Innovations, headquartered in Chicago, is a biotech-focused venture capital company seeking to bolster the expansion of biotech startups in up-and-coming life sciences centers like Chicago and Atlanta. John Flavin, the CEO of Portal, founded the company to cater to life sciences firms in ecosystems that lack the infrastructure and supportive community found in well-established hubs such as Boston and the Bay Area. Institutions like Northwestern, the University of Chicago, and the University of Illinois in Chicago have emphasized the recruitment of forward-thinking faculty in life sciences, resulting in an abundance of promising ideas. Portal Innovations provides specialized wet lab facilities to startups, equipping them with the vital infrastructure and support needed to thrive.

Biotechnology has revolutionized the healthcare industry, and with the increasing demand for advanced therapies, the focus has shifted towards innovative therapeutic modalities. In this blog article, we will explore some of the cutting-edge therapeutic modalities that have the potential to transform patient care.

Over 2020-2021 we have seen a remarkable parade of artificial intelligence (AI), focused on discovering novel therapeutics, going public. This reflects a continuing interest in AI as a transformative technology for the pharmaceutical industry and increasing belief in the wave of technology-driven “neo-biotechs”.

Here we list some notable companies which became public recently. For more information, please, check our unique interactive report “The Landscape of Artificial Intelligence (AI) In Pharmaceutical R&D”.

image credit: Chunumunu

Gene therapy is still an investigational technique. Viruses are commonly used as vectors to deliver a needed gene to a host (defective) cell. An ideal viral vector is supposed to be genetically stable, non-toxic, and non-immunogenic, to have a high packaging capacity along with other important features. 

What is a Mesenchymal Stem Cell? What Can it Do?

Mesenchymal stem cells (MSCs) are multipotent stem/progenitor cells that are present in different tissues, including the umbilical cord, bone marrow, and fat tissue. MSCs can self-renew by dividing and differentiate into cells of bone, cartilage, muscle and fat cells, and connective tissue. They are also famous for their ability to produce useful growth factors and cytokines. MSCs secrete multiple factors, including prostaglandin E2, interleukin-10, interleukin-1 receptor antagonist, interleukin (IL)-6, leukocyte inhibitory factor, nitric oxide, TNFα stimulated gene 6 (TSG-6), and many others, which limit immune response. In addition, MSCs skew maturing immune cell populations, i.e. populations of regulatory and anti-inflammatory T cells and dendritic cells become more abundant, while pro-inflammatory T cells, dendritic cells, and natural killers (NKs) decrease in numbers.  

Due to these unique features, MSCs are considered a promising approach to treat autoimmune diseases and to manage the rejection of grafts. For over 10 years, they have been successfully used in the therapy of autoimmune diseases (rheumatoid arthritis, ulcerative colitis, type 1 diabetes, multiple sclerosis) and for the inhibition of transplanted organs rejection.

Antisense oligonucleotides (ASOs) are short (about 12 to 25 nucleotides long), synthetic single strands of DNA or RNA that are complementary to a chosen sequence. They can alter RNA and reduce, restore, or modify protein expression. ASOs interact with proteins on the surface of cells and enter the cytoplasm. Then they can work either in right in the cytoplasm or enter the nucleus.

In their naked form, ASOs cannot permeate the plasma membrane and are highly sensitive to degradation by endonucleases and exonucleases. To overcome these problems, ASOs have been chemically modified. On the basis of these modifications, ASOs can be broadly classified into three generations.