the success of CGT depends on a robust and reliable supply chain” Cell and gene therapies (CGT) offer a revolutionary approach to the treatment, prevention and potential cure of diseases. These advanced medical treatments provide hope for patients suffering from conditions that have historically been poorly understood and widely considered incurable, including genetic diseases and cancers. A recent survey1 of healthcare industry professionals showed that CGT is expected to have the most significant impact on the pharmaceutical industry in 2024. However, the success of CGT depends on a ..read more

A study has reported the application of light-assisted drying (LAD) to large-volume samples (250 μL) that are closer to those used for doses of vaccines and therapeutics. According to the authors of the paper, this is “a five-fold increase in volume compared to previous studies”. While lyophilisation (freeze drying) is most widely adopted method for storing proteins, temperature-controlled storage can be time-consuming and costly, especially considering the thermal sensitivity of some biologics, the authors noted. While previous studies have demonstrated that LAD can prepare small-volume ..read more

Withdrawal of the marketing authorisation for the COVID-19 vaccine (ChAdOx1-S [recombinant]) Vaxzevria (SRD) by the European Medicines Agency on 7 May, follows a request by AstraZeneca. “Global demand for all COVID vaccines is now much lower and overall supply exceeds demand. This is in marked contrast to the early part of the pandemic when supply was limited and distribution very limited, especially in poorer countries. Case for withdrawing the AstraZeneca vaccine there is probably no commercial case for continuing to manufacture and distribute the vaccine and I think this is like ..read more

Regulatory agencies in the US across three sectors: agriculture and environmental, have produced a joint plan to “update, streamline, and clarify” regulations and oversight mechanisms for biotechnology products. The plan, developed by the US Environmental Protection Agency (EPA), the US Food and Drug Administration (FDA), and the US Department of Agriculture (USDA), helps to address US President Biden’s related goals, including and improving the “transparency, predictability, coordination, and efficiency” of the biotechnology regulatory system, as detailed in the Executive Order 14081 ..read more

New data from a Phase IIa trial in moderate-to-severe atopic dermatitis has demonstrated that a novel monoclonal antibody (mAb) could facilitate dosing every 12 weeks for induction therapy. This is due to a 31-day half-life at anticipated therapeutic dose levels, Inmagene Biopharmaceuticals confirmed. Humanised anti-OX40 IgG1 mAb IMG-007 works via a silenced antibody-dependent cellular cytotoxicity (ADCC) function, Inmagene explained. What did the interim data for IMG-007 demonstrate? The clinical trial is evaluating the mAb IMG-007 in adults with moderate-to-severe atopic dermatitis who have ..read more

Two new formulations cabotegravir (cabotegravir extended-release injectable suspension and tablets) have been approved by the Medicines and Healthcare products Regulatory Agency (MHRA). The treatment consists of Apretude 30mg film-coated tablets and Apretude 600mg prolonged-release suspension for injection. These treatments are indicated for the prevention of sexually transmitted HIV-1 infection — pre-exposure prophylaxis (PrEP) in adults and adolescents weighing at least 35kg with a higher infection risk, MHRA confirmed. About cabotegravir Cabotegravir long-acting (LA) for PrEP is ..read more

A new report by MarketsandMarkets has predicted that the injectable drug delivery market will reach an 8.6 percent compound annual growth rate (CAGR) between 2024 to 2029. The market is anticipated to value $1139.4 billion by 2029. Injectable drugs are adopted widely as treatments for chronic infections such as HIV/AIDS and tuberculosis (TB). Treatment approaches for the latter condition is often a combination of oral antibiotics plus injectable drugs such as rifampin, isoniazid, and streptomycin, the report noted. Injectable vaccines activate the immune system to help to prevent infectious c ..read more

New results from a Phase I/II clinical trial of a CRISPR/Cas9 gene editing therapy show that around 79 percent of patients with a rare form of inherited blindness experienced “measurable improvement”. The BRILLIANCE trial included 14 participants (12 adults and two children) who received an injection of EDIT-101 in one eye. These individuals had a form of Leber Congenital Amaurosis (LCA) caused by mutations in the centrosomal protein 290 (CEP290) gene. “This trial represents a landmark [for the treatment of] genetic blindness, by offering an important alternative treatment, when traditio ..read more

Following a review on the suitability of different technologies, researchers have determined that “[nucleic acid amplification techniques] NATs combined with an efficient extraction method” is the fitting alternative for compendial mycobacterial testing in biological products. The current challenge in mycobacterial testing information on using mycobacterial testing for the quality control of biologicals is limited, the paper noted. Mycobacteria can be difficult to detect on media cultures, partly due to the “opacity of the cell wall [resulting in] a slow bacterial metabolism” the authors expl ..read more

An interim analysis of a Phase III trial of Calquence (acalabrutinib) plus chemoimmunotherapy has revealed positive progression-free survival outcomes for adults with untreated mantle cell lymphoma. AstraZeneca’s ECHO Phase III trial evaluated acalabrutinib in combination with standard-of-care chemoimmunotherapy, bendamustine and rituximab. Calquence (acalabrutinib) is a next-generation, Bruton’s tyrosine kinase (BTK) inhibitor.  Based on the high-level data, using Calquence in the first-line setting “significantly delays disease progression and, for the first time, shows poten ..read more