Different therapeutic approaches based on the physiological mechanisms regulating lysosomal function are currently proposed for lysosomal storage disorders (LSDs), including hematopoietic stem cell transplantation, enzyme replacement therapy, substrate reduction and chaperone therapy, gene therapy and other therapies. As a lysosomal innovator, CD BioSciences is committed to developing comprehensive drug discovery services for lysosomal storage disorders. Our dedicated team will ensure that you receive the most satisfactory solution for drug discovery of LSDs.
Introduction of Drug Discovery for Lysosomal Storage Disorders
LSDs are a group of inherited metabolic disorders caused by mutations in genes encoding soluble lysosomal hydrolases, integral membrane proteins and transporter proteins. There are more than 40 known LSDs, which are traditionally classified according to the chemical nature of the accumulated substrates. Because of their cumulative prevalence, the debilitating impact of their clinical presentation, and the need for a multidisciplinary approach to patient care, LSDs are challenging for pediatricians and other physicians and represent a heavy burden in terms of public health and economic costs. Over the past two decades, research in the field of LSDs has made significant advances with the rapid development of understanding of the physiology of lysosomes and the development of a variety of innovative therapeutic approaches. Further impetus for the development of LSDs therapies comes from the availability of technologies that allow large-scale production, purification and manipulation of new drugs, recombinant proteins and viral vectors, as well as incentives for biotechnology companies to invest in rare disease therapies.
Fig. 1. Therapeutic approaches in LSDs. (Fernández-Pereira C, et al., 2021)
Our Drug Discovery Solutions for Lysosomal Storage Disorders
The pathologies of LSDs are characterized by the presence of membrane-encapsulated tissue deposits. Each event in the LSDs pathogenic cascade is a potential therapeutic target, and a variety of approaches based on different strategies and principles can be utilized to treat these diseases. As a leading global provider of lysosomal services, CD BioSciences has different LSDs therapeutic solutions available or being evaluated in preclinical studies. The primary goal of our treatment strategies is to preserve or restore organ function, which can be achieved by preventing or reducing the accumulation of tissue matrix.
Our team of experts propose or test many new approaches in preclinical studies. In addition, we have a wealth of disease animal models. The solutions we offer have a positive impact in terms of bioavailability of therapeutic agents, toxicity, immune response and impact on patient quality of life. CD BioSciences is committed to providing comprehensive drug discovery strategies for lysosomal storage disease to our global customers.
Hematopoietic Stem Cell Transplantation Solutions for LSDs
We offer hematopoietic stem cell transplantation (HSCT) technology as a therapeutic solution for LSDs. An important advantage of HSCT is the ability of donor-derived cells that produce functional enzymes to migrate to the brain, thereby delaying neurocognitive degeneration.
Enzyme Replacement Therapeutic Solutions for LSDs
We develop novel recombinant enzymes to compensate for the metabolic deficits in patients with lysosomal storage disease. These therapeutic enzymes can be produced by transgenic cells from different organisms such as animals, humans, plants or ova.
Oral Small Molecule Therapeutic Solutions for LSDs
Small molecules exhibit properties suitable as potential therapeutic options for LSD. We provide customized small molecule therapeutic solutions for LSD to develop antigen-free small molecules with antagonistic or agonistic effects on intracellular and extracellular targets.
Gene Therapeutic Solutions for LSDs
LSD is well characterized as a monogenic disease and enzyme expression is usually unaffected by complex regulatory mechanisms. We offer both in vivo and ex vivo gene therapy solutions for LSD.
Nanocarrier-Based Delivery Therapeutic Solutions for LSDs
Drug therapy is continually evolving towards more precise delivery of biomolecules and targeted drugs. We develop cell-produced polymeric nanoparticles, lipids, liposomes, and extracellular vesicles to deliver bioactive enzymes and other types of therapeutic molecules to LSDs.
Advantages of Our Solutions
- Our team of experts has a solid knowledge of the molecular basis of genetic diseases and the pathophysiology of LSD.
- Collaboration with international cytologists.
- A variety of novel and attractive therapeutic solutions.
- Extensive drug development for preclinical treatment of lysosomal storage disorders.
- Exploring combinations of different approaches to achieve maximum therapeutic efficacy.
CD BioSciences has the most advanced therapeutic strategies for lysosomal storage diseases. Our experienced team of experts can help solve drug discovery problems in lysosomal storage diseases. Please feel free to contact us for a quote.
Reference
- Fernández-Pereira C, et al. (2021) Therapeutic Approaches in Lysosomal Storage Diseases[J]. Biomolecules. 11(12): 1775.