How do you see the future of cell and gene therapy?

drug development, , ,

Our concise outlook is the following:

Transformational Potential: Cell and gene therapies are revolutionizing how we treat diseases, promising to cure certain conditions.
They remain at the forefront of healthcare innovation.

Fast-Growing Field: These therapies are the fastest-growing therapeutics areas, with over 50 new in vivo and ex vivo gene therapy launches planned in the next few years.

Challenges and Innovation: While promising, cell and gene therapies require substantial innovation to unlock their full potential for patients. Scaling digital and analytics in discovery and R&D is part of the solution.

Market Resilience: Despite the disruptions caused by the pandemic, stakeholders, including CGT innovators, healthcare providers, and CDMOs, are optimistic that positive developments lie ahead.

Viral-Vector Therapies: Viral-vector gene therapies are here to stay, but addressing challenges and strategizing for patient access is critical for their success.

Overall, the outlook is positive, but ongoing research, regulatory advancements, and manufacturing innovations will shape the future of cell and gene therapy. 🌟

Cyclodextrin encapsulation enabling the anticancer repositioning of disulfiram: Preparation, analytical and in vitro biological characterization of the inclusion complexes

drug development

Drug repositioning is a high-priority and feasible strategy in the field of oncology research, where the unmet medical needs are continuously unbalanced. Disulfiram is a potential non-chemotherapeutic, adjuvant anticancer agent. However, the clinical translation is limited by the drug‘s poor bioavailability. Therefore, the molecular encapsulation of disulfiram with cyclodextrins is evaluated to enhance the solubility and stability of the drug.

This collaboration of Semmelweis University and Egis Pharmaceuticals PLC describes for the first time the complexation of disulfiram with randomly methylated-β-cyclodextrin.

Throughout the different cancer cell lines‘ characteristics and disulfiram unspecific antitumoral activity, the inhibitory efficacy of the cyclodextrin encapsulated drug on melanoma (IC50 about 100 nM) and on glioblastoma (IC50 about 7000 nM) cell lines differ by a magnitude. This pre-formulation screening experiment serves as proof of concept for using cyclodextrin encapsulation as a platform tool for further drug delivery development in repositioning areas.

Cyclodextrin encapsulation enabling the anticancer repositioning of disulfiram: Preparation, analytical and in vitro biological characterization of the inclusion complexes – ScienceDirect

2024’s New FDA Drug Approvals

drug development, ,


🌟 Discover the latest from the FDA’s CDER: 9 innovative drugs for Q1!
🧴Berdazimer (Zelsuvmi) tackles topical molluscum contagiosum with nitric oxide release.
💉Cefepime/enmetazobactam (Exblifep) combines forces for urinary tract infection relief.
💉LetibotulinumtoxinA (Letybo) smooths glabellar lines with botulinum neurotoxin.
💉Tislelizumab (Tivembra) fights esophageal squamous cell carcinoma with PD-1 mAb technology.
💊Resmetirom (Rezdiffra), a thyroid hormone receptor beta agonist, battles nonalcoholic steatohepatitis.
💊Aprocitentan (Tryvio) manages hypertension through endothelin receptor antagonism.
💊Givinostat (Duvyzat) aims at Duchenne muscular dystrophy with HDAC inhibition.
💉Sotatercept (Winrevair), an activin inhibitor, offers hope for pulmonary arterial hypertension patients.
💊Vadadustat (Vafseo) addresses CKD-related anemia in dialysis patients as an oral HIF-PH inhibitor.

Explore these groundbreaking treatments! Our appreciation to Chris De Savi for the great compilation!

Adenovirus formulation

drug development

This recent patent from our friend Tao Peng at Roquette on preserving an adenovirus vaccine with the help of cyclodextrins got us thinking about how CDs can be used in this area.


EP4338727 ADENOVIRUS FORMULATIONS (wipo.int)

In the context of vaccines, cyclodextrins can effectively encapsulate antigens, ensuring their protection from degradation and improving their immunogenicity. Cyclodextrins offer stability advantages to vaccines by preventing the degradation of labile vaccine components during storage and transportation. Furthermore, cyclodextrins can serve as adjuvants, potentiating the immune response triggered by vaccines. Their unique structure and interaction with the immune system enhance the recognition of antigens by immune cells, leading to an improved activation of both innate and adaptive immune responses. This adjuvant effect contributes to the development of robust and long-lasting immune protection against targeted pathogens. Owing to the distinctive attributes inherent to nanoparticles, their integration into vaccine formulations has assumed an imperative role. Through the encapsulation of vaccine antigens/adjuvants within cyclodextrin nanoparticles, the potency and stability of vaccines can be notably enhanced. In particular, the capacity of amphiphilic cyclodextrins to form nanoparticles through self-assembly without surfactants or co-solvents is a captivating prospect for their application as carrier systems for antigens. In conclusion, cyclodextrins present a promising platform for enhancing the efficacy and stability of vaccines. Their ability to encapsulate antigens, stabilize labile vaccine components and act as adjuvants demonstrates their potential to revolutionize vaccine formulation and delivery. Further research and development in this field will facilitate the translation of cyclodextrin-based vaccine technologies into practical and impactful immunization strategies, ultimately benefiting global health and disease prevention.

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease

drug development

Tay-Sachs disease, a progressive neurodegenerative disorder caused by mutations in the HEXA gene, lacks effective treatment. This study explores the therapeutic potential of δ-tocopherol and hydroxypropyl-β-cyclodextrin, targeting lysosomal exocytosis in Tay-Sachs models. Results indicate significant lysosomal GM2 reduction, offering hope for future treatments.

Induction of Exocytosis Rescues Lysosomal GM2 Accumulation in Tay-Sachs Disease (hindawi.com)

Methods for the treatment of chronic kidney disease

drug development, , ,

We are happy to announce the acceptance of a great patent from CarboHyde’s collaborator, Renatus, a pharmaceutical company focused on developing cyclodextrin-based cholesterol metabolism modulators. Renatus is a pharmaceutical company whose mission is to provide safe and effective treatment options for the treatment of numerous cholesterol-driven diseases such as chronic kidney disease, atherosclerosis, and Alzheimer’s diseases. The core technology of the company allows the normalization of cholesterol homeostasis by removal of excessive and toxic cholesterol within diseased cells without significant harmful effects on normal cells.

The lead asset RN-005 is a cholesterol metabolism modulator that has proven strong therapeutic efficacy in preclinical models of both diabetic kidney disease (DKD) and focal segmental glomerulosclerosis (FSGS) wherein dysregulated cholesterol metabolism causes progressive damage and loss of function in the kidney. The gamma-cyclodextrin oligomers are effective in cholesterol metabolism enhancement, cholesterol efflux, reducing inflammatory cytokine secretion, renal clearance of cholesterol, and/or reducing albuminuria. Therefore, the gamma-cyclodextrin oligomers can be used to treat or alleviate chronic kidney disease, symptoms thereof and/or complications related to chronic kidney disease.

Espacenet – METHODS FOR THE TREATMENT OF CHRONIC KIDNEY DISEASE

Hydrogel composition for use in the treatment of articular disorders

carbohydrates, drug development,

Fascinating patent by Bernard Martel, Nicolas Blanchemain and colleagues presenting hydrogels on anionic cyclodextrin polymers and chitosan for use in the treatment of articular disorders. The hydrogel combines an analgesic action with a visco-supplementation (a lubricating effect).

WO2024023350 HYDROGEL COMPOSITION FOR USE IN THE TREATMENT OF ARTICULAR DISORDERS (wipo.int)

Resveratrol eye drops as well as preparation method and application

drug development, ,

In some cases, the simplest, most straightforward ideas are the best. Why not develop an eye-drop based on cyclodextrin and resveratrol?  The resveratrol captured in an HPBCD inclusion complex has the advantages of simple preparation, short preparation period, no organic solvent residue, and no bacteriostatic agent. The lipoic acid added in the clathrate eye drops prepared by the invention cooperates with the resveratrol to play a better antioxidant effect so that the xerophthalmia is better relieved, and the treatment effect is achieved.

What to create similar solutions? Visit: www.carbohydesolutions.com – Carbohyde
Original paper: Espacenet – Resveratrol eye drops as well as preparation method and application thereof

Adamgammadex vs Sugammadex

drug development,

Check out this fascinatnig discussion on next future of cyclodextrin-based antidotes and scavengers by J Robert Sneyd adn Jennifer Hunter.

Spoiler alert: Phase 3 clinical trials suggest that Adamgammadex is slightly less potent than sugammadex and has a non-inferior speed of onset. In a multicentre trial of 310 patients, there was a suggestion of a lower incidence of allergic responses and recurarisation after adamgammadex compared with sugammadex.

If you are also thinking about creating a special cyclodextrin like that, this may be useful for you: Custom synthesis of cyclodextrins – www.carbohydesolutions.com

Is adamgammadex the brother of sugammadex or the next generation of reversal agent? – British Journal of Anaesthesia (bjanaesthesia.org)