gene therapy Archives - Medical Design Sourcing

One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients

[Zolgensma image from Novartis]

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations and demonstrated a positive overall benefit-risk profile. LT-001 is an ongoing 15-year long-term follow-up study of patients who completed the phase 1 START study. More than 3,000 children have been treated with Zolgensma in clinical trials, managed access programs and commercial settings.

Novartis expects Zolgensma to eventually generate $1.5–2 billion, according to BioPharma Dive. NVS shares were up 1 percent to $83.01 today after announcing the new data.

FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for chi…

Prominent cell and gene therapy vendors in 2023

[Image courtesy of catalin/Adobe Stock]

In recent years, the cell and gene therapy market has grown at a rapid clip. The momentum is likely to continue. According to Vision Research Reports, the global cell and gene therapy market size could hit around $42.56 billion by 2030, growing at a CAGR of 39% from 2022 to 2030.

The burgeoning demand in the industry, along with an increasing number of FDA-approved therapies, is also fueling the growth of cell and gene therapy vendors offering tools and services for product development and manufacturing.

The following table includes notable vendors and suppliers active in the call and gene therapy marketplace. This curated selection of companies includes a brief overview of their focus areas.

We have a separate feature focused on companies with prominent cell and gene therapy development efforts.

Company Name Description Notable partnerships Founded He…

‘Freeze-Lock’ cryo label supports the needs of frozen medications

The Freeze-Lock cryo label from Schreiner MediPharm (Oberschleissheim, Germany) is suited for mRNA-based vaccines. The labels also support many cell and gene therapy medications and clinical trials, given their ability to withstand subzero storage and transport temperatures required by pharmaceutical and biopharmaceutical manufacturers and clinical researchers.

Schreiner MediPharm notes that conventional labels fail to offer consistent adhesion. The company’s Freeze-Lock cryo label comprises two interlocking label layers to optimize for adhesive strength and product information readability.

The label includes two components: a bottom and a top label.

The bottom label layer has a microfine surface texture that bonds to a container at room temperature. After being filled with the active ingredient, the label is ready to be stored at subzero temperatures requiring dry ice (–78 °C/–108.4 °F) or liquid nitrogen (–196 °C/–320.8 °F).

The top label layer …

Gene therapy specialist Viralgen opens new facility in Spain’s Basque Country

San Sebastián, Spain–headquartered Viralgen, has opened a new factory in Basque Country in northern Spain.

The company focuses on manufacturing adeno-associated virus (AAV) vectors, which are used in gene therapy and some vaccines.

Since 2018, Viralgen has offered AAV manufacturing for customers working in preclinical through Phase 2 development programs. The company has licensed its Pro10 AAV platform from AskBio.

The first phase of investment in the facility represents a €70 million investment (approximately $83 million). The company plans on spending a total of €120 million (roughly $142 million) after the construction of two additional buildings is completed.

A total of 250 people will work in the facility in 2022. Of that total, some 130 will be new hires.

The inauguration of Viralgen’s new facility occurred on June 30, 2021.

Formed in 2017, Viralgen was a joint venture…