Pharmaceutical Technology Editors

Orchard Therapeutics announced on Feb. 27, 2023 that it has reached an agreement with the New Therapies (NT) Council which will result in reimbursed access to Libmeldy (atidarsagene autotemcel) for all metachromatic leukodystrophy (MLD) patients who fall within the scope of the European marketing authorization in Sweden.

The agreement with NT Council is a key component of the company’s commercial expansion plans in Europe and follows similar arrangements made with reimbursement authorities in the United Kingdom, Italy, and Germany. Additionally, the company is qualifying a sixth treatment center in Europe at the University Hospital Skåne Lund in Sweden, intended to serve as the primary site for eligible patients referred from the country and the Nordic region.

Libmeldy received the strongest possible recommendation for use by the NT Council following the completion of a health economic evaluation by FINOSE, a health technology assessment consortium between Finland, Norway, and Sweden.

“Libmeldy has the potential to transform the lives of eligible children with a single administration and was approved by the European Commission for the treatment of early-onset MLD based on data now encompassing more than 10 years of follow-up in the earliest treated clinical trial patients,” said Robin Kenselaar, senior vice president, EMEA at Orchard Therapeutics, in a company press release. “We are encouraged that the reimbursement agreements secured in multiple European countries to date continue to recognize the value of Libmeldy commensurate with its clinical impact. The agreement with NT Council is a positive step toward enabling access for MLD patients in other Nordic countries. We look forward to continuing to work with reimbursement authorities in the Nordic region and throughout Europe to make Libmeldy available to patients who may benefit from treatment.”
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Orchard Therapeutics has announced an agreement which will enable reimbursed access to Libmeldy for all eligible MLD patients in Sweden.

Orchard Therapeutics to Reimburse Access to Libmeldy for MLD Patients in Sweden

Bio-Rad Laboratories introduced its new StarBright Blue and StarBright Yellow dyes in February 2023. The dyes are designed to promote flexibility in conventional and full spectrum flow cytometry applications. Dyes in the Starbright series are conjugated to flow validated antibodies, supporting flow cytometry panel design in immunology design.

The two new variations in the Starbright series are intended to expand research capabilities across blue (488 nanometers) and yellow (561 nanometers) lasers. According to a Feb. 13, 2023 press release, the new dyes offer exceptional brightness in conjunction with narrow excitation and emission profiles, allowing for precise resolution.

StarBright dyes are compatible with most experimental protocols and flow cytometers without the need for special buffers. Additionally, minimal lot-to-lot variation helps to ensure reproducible and consistent staining, and the dyes are resistant to photobleaching with no loss of signal in fixation, according to the release.

"The continued development of the Bio-Rad StarBright Dye portfolio, now totaling 28 dyes and spanning multiple lasers, gives researchers greater choice and flexibility across multiplex flow cytometry applications, offering superior alternatives to existing dyes," said Mike Blundell, product manager, Flow Cytometry, Life Science Group, Bio-Rad, in the release. "With the launch of the StarBright Blue and StarBright Yellow Dyes, Bio-Rad now has the most comprehensive series of new dyes specifically designed for flow cytometry."

Bio-Rad’s newly launched StarBright Blue and StarBright Yellow dyes are intended for use in various flow cytometry applications.

Bio-Rad’s New StarBright Blue and Yellow Dyes Enhance Multiplex Flow Cytometry

Eurofins Viracor, a Eurofins company specializing in testing for infectious diseases, immunology, and allergy, launched in February 2023 its Viracor ExPeCT chimeric antigen receptor T-cell (CAR-T) multiplexed quantitative real-time polymerase chain reaction (qPCR) assay. The tool is designed to monitor and optimize CAR-T therapy involving patients with pre-B cell acute lymphoblastic leukemia and B cell lymphomas.

According to a press release from Feb. 16, 2023, the test is designed to provide clinicians with an accurate and comprehensive assessment of the CAR-T cell population in the patient’s body, in turn offering potential insights into therapy effectiveness and what future treatment options may be advisable.

Eurofins Viracor ExPeCT CAR-T qPCR assay is a tool designed for monitoring and optimizating CAR-T therapies.

Eurofins Viracor Launches Test for Assessing Expansion and Persistence of CAR-T Therapy in Cancer Patients

Moderna and Life Edit Therapeutics, an ElevateBio company specializing in next-generation gene editing technologies and therapeutics, announced a strategic research and development collaboration on Feb. 22, 2023. The partnership, which will combine Life Edit’s suite of gene editing technologies with Moderna’s messenger RNA (mRNA) platform, will seek to advance 

gene editing therapies against a set of various therapeutic targets.

Under the agreement, Life Edit and Moderna will collaborate on research and preclinical studies funded by Moderna. Upon exercising an option for a target, Moderna will assume responsibility for further development, manufacturing, and commercialization. According to a company press release, Life Edit will receive an upfront payment and is eligible to receive potential development, regulatory, and commercial milestone payments for each target for which Moderna exercises an option, as well as tiered royalties on global net sales of products from the collaboration. The amount of financial compensation was not disclosed in the release.

Life Edit’s gene editing platform offers a library of base editors and RNA-guided nucleases (RGNs). According to the release, the RGNs are smaller in size when compared to conventional nucleases, which the company hopes can enable greater versatility for delivery. Additionally, the company’s nuclease collection features a range of protospacer adjacent motifs, short sequences that determine the DNA segments in the genome to which a nuclease can bind. The diversity of this range is intended to enable base editing at more sites than a single nuclease could achieve. This collaboration will apply Life Edit's RGNs and base editors together with Moderna's mRNA platform with the goal to develop curative therapies for challenging genetic diseases.

"This collaboration between Life Edit and Moderna demonstrates the strength of our respective technologies to advance programmable medicines to more specifically target disease," said Mitchell Finer, CEO, Life Edit Therapeutics, and president, R&D, ElevateBio, in the release. "Our novel editing systems have the potential to precisely modify gene targets for both 

 therapeutic development. We are excited that partners, such as Moderna, are recognizing the potential of our technology."

"At Moderna Genomics, we are constantly working to accelerate new therapeutic targets that may one day lead to the next generation of transformative mRNA medicines for patients," said Eric Huang, general manager and chief scientific officer, Moderna Genomics, in the release. "Through our collaboration with Life Edit, we hope to harness the power of gene editing technologies as part of our broader research and development engine, helping to advance our mission and deliver on the promise of mRNA."

Moderna will leverage Life Edit’s suite of gene editing technologies to advance development of in vivo gene editing therapies.

Moderna and Life Edit Therapeutics Enter Gene Editing Collaboration

FDA granted approval to Sanofi’s Altuviiio (Antihemophilic Factor [Recombinant] Fc-VWF-XTEN Fusion Protein-ehtl), a factor VIII replacement therapy, on Feb. 23, 2023. The treatment is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management, for adults and children with hemophilia A.

FDA based its approval in part on data from the pivotal XTEND-1 Phase III study, which found that once-weekly prophylaxis met the primary endpoint of providing significant bleed protection for people with severe hemophilia A. The treatment had a mean annualized bleeding rate (ABR) of 0.70 and a median ABR of 0.0. Additionally, it met the key secondary endpoint with a significant reduction of 77% in ABR versus prior factor prophylaxis based on an intra-patient comparison.

"Today’s approval of [Antihemophilic Factor (Recombinant) Fc-VWF-XTEN Fusion Protein-ehtl] allows patients and physicians to reimagine living with hemophilia,” said Paul Hudson, CEO, Sanofi, in a company press release. “The high sustained factor activity levels that can be achieved with [Antihemophilic Factor (Recombinant) Fc-VWF-XTEN Fusion Protein-ehtl] have the potential to change the hemophilia landscape. For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients.”

This is Antihemophilic Factor (Recombinant) Fc-VWF-XTEN Fusion Protein-eht’s first approval, which FDA granted after also granting priority review, breakthrough therapy, fast track, and orphan drug designations. Regulatory submission to the European Union is expected to occur in the second half of 2023.

Sanofi’s once-weekly replacement therapy is FDA-approved for prevention and treatment of bleeding episodes in people with hemophilia A.

FDA Approves Sanofi Hemophilia Treatment

AbbVie and Capsida Biotherapeutics, a biopharma company specializing in next-generation gene therapy platforms, announced an expanded strategic collaboration on Feb. 23, 2023. Building on a neurodegenerative disease partnership announced in 2021, the expanded partnership will see AbbVie work with Capsida’s novel adeno-associated virus (AAV) platform to identify and advance three programs.

According to a company press release, Capsida will lead capsid discovery efforts for all programs using its AAV engineering platform and will be responsible for process development and early clinical manufacturing. AbbVie will lead innovative therapeutic cargo approaches and be responsible for development and commercialization.

Per the terms of the agreement, Capsida will receive $79 million upfront. In addition to the upfront payment, Capsida is eligible for an additional $595 contingent on option fees and achievement of various research and development milestones. Should any therapies reach commercialization, Capsida is also eligible for further payments and low double-digit royalties.

“This expanded collaboration with Capsida has the potential to develop transformative therapies for patients with serious eye diseases,” said Jonathon Sedgwick, vice-president and global head of discovery research, AbbVie. “In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner.”

“AbbVie has been an excellent partner, and we are excited to expand our collaboration into ophthalmology with the world leader in this therapeutic area,” said Peter Anastasiou, CEO, Capsida, in the release. “Combining AbbVie’s expertise in eye disease drug development and commercialization with Capsida’s fully integrated next-generation AAV engineering platform and manufacturing capabilities offers the potential to provide novel therapies enabling unprecedented benefit to patients with serious eye diseases.”

AbbVie and Capsida have expanded their existing collaboration to advance three new gene therapy programs.