gene therapy Archives - Medical Design Sourcing

From Novartis to Pfizer: A closer look at novel cell and gene therapy pricing and reimbursement strategies

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Cell and gene therapies are upending the treatment of a growing number of diseases by addressing the underlying causes of genetic disorders. Yet the high costs associated with these therapies, sometimes costing multiple millions of dollars for a single treatment, pose significant challenges for patients, payers and healthcare systems. To address this matter, a growing number of companies are pioneering novel pricing and reimbursement strategies such as outcomes-based agreements and risk-sharing models to help ensure patient access while mitigating financial risks.

Spotlight on Beqvez: A promising hemophilia therapy

In that vein, Pfizer recently announced the FDA approval of Beqvez, a one-time gene therapy for adults with moderate to severe hemophilia B. In tandem with the launch, Pfizer revealed a novel warranty program “based on durability of patient response to treatment.” The firm n…

Unicorn gene-delivery firm VectorBuilder eyes future breakthroughs

This image from VectorBuilder showcases a comparative study of luciferase expression in various mouse organs, highlighting the targeted gene delivery capabilities of Anti-CD31 conjugated LNP-mRNA. Controls include IgG2a-conjugated FLuc LNP-mRNA and naked FLuc mRNA.

Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current financial climate poses challenges, “the future is very bright in the cloning world and gene delivery,” Mussar noted. “At VectorBuilder, we’re all about innovation and having our finger on the pulse. We’re always focusing on the next thing.”

Earlier this y…

50 of the best-funded biotechs of 2023

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As the year draws to a close, it is clear that molecular science and diagnostics is the hottest funding area in the biotech industry. In an analysis of 50 of the best-funded biotechs of 2023 focused on human health, molecular and science and diagnostics startups collectively attracting roughly $945 million, dwarfing the figures in other segments. The next popular two niches, gene therapies and oncology, had average funding levels of approximately $245 million and $170 million, respectively. While AI has received a significant amount of attention this year, biotechs specializing in that field garnered an average funding of only about $66 million. Outside of the life sciences, startups with a broader focus on AI raised a cumulative average of $202.47 million, based on an analysis of close to 1000 companies.

Caris Life Sciences has raised nearly $1.7B to date

In terms of best-funded companies overall,…

Prix Galien Awards: The most innovative biotech, pharma, and orphan drugs of 2023

Prix Galien

Prix Galien names 2023 winners in pharma and biotech

In the world of medical innovation, few accolades carry as much weight as the Prix Galien Awards, which highlight the advances in biotech, pharmaceuticals, and other domains. The 2023 winners include Bristol Myers Squibb’s Camzyos (mavacamten) as the best biotechnology product and Lilly’s Mounjaro (tirzepatide) and Novo Nordisk’s Ozempic (semaglutide) sharing the honor for best pharmaceutical product. Finally, Boehringer Ingelheim’s Spevigo (spesolimab) and CSL / uniQure’s Hemgenix were twin winners in the rare/orphan disease category.

Here, we take a closer look at the Prix Galien 2023 winners in pharma, biotech and rare/orphan disease.

Camzyos: A new chapter in cardiomyopathy therapy

First winning FDA approval in April 2022, Camzyos is a novel option for symptomatic NYHA class II-III obstructive hypertroph…

Regeneron agrees to spend up to $213M to acquire Decibel Therapeutics

To date, 2023 is shaping up to be a relatively brisk year for M&A deals. A recent case in point is Regeneron’s acquisition of Decibel Therapeutics. The centerpiece of the deal is DB-OTO, a gene therapy intended for patients with otoferlin-related hearing loss. The drug is in its first clinical trial. Regeneron agreed to pay $4.00 per share for Decibel stock with the potential to pay up to $3.50 per share in cash if Decibel hits agreed-upon regulatory milestones for DB-OTO in a specified time frame, bringing the total value of the deal to roughly $213 million.

Last year, Decibel touted DB-OTO’s ability to support otoferlin expression for several weeks before it plateaued. The company said the data for the drug were in line with earlier preclinical research in which mice achieved a functional recovery.

Involved in transmission of sound signals in the inner ear, the protein otoferlin is essential for hearing. Deficiency or mutations in the gene that produces o…

Deepcell’s REM-I platform marries AI with morphological analysis for drug discovery

Introducing Deepcell’s REM-I, which applies AI to cell morphology analysis.

From her early days as a research assistant at UCLA, to her work as a postdoctoral fellow at Stanford, Deepcell co-founder and CEO Maddison Mahdokht Masaeli has actively engaged in the field of biomedical engineering. Now, Masaeli and her team at Deepcell, a company she co-founded in 2017, are introducing a new strategy to drug discovery with the launch of REM-I. The platform combines AI and morphological analysis, a technique that studies the form and structure of organisms and their specific structural features.

“The REM-I platform offers several unique capabilities for deep assessment of single cell images and the ability to sort cells without bias,” Masaeli explained in an interview. “It is specifically designed to expose and analyze the heterogeneity within samples, as well as the heterogeneity in respons…

Capsida Biotherapeutics to present preliminary findings on engineered AAV capsids at ASGCT annual meeting

The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles.

In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained that the company employed directed evolution and a high-throughput screening platform to optimize capsid performance across key domains. “We start with a disease and aim to create the best possible therapy, as opposed to trying to mold a disease profile around the limitations of wildtype capsids,” Catalano said.

Preliminary results suggest these engineered AAV capsids can reach up to 68% of neurons across multiple brain regions when delivered intravenously. That finding could translate to new possibilities in gene therapies targeting common neurological diseases that have…

Development challenges and regulatory changes for cell and gene therapies

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Cell and gene therapies are the fastest growing area for drug development. The groundwork for this category was laid with the mapping of the human genome in 2003, and the field has developed rapidly in the intervening decades. These powerful therapies have significant potential for the treatment of cancer and other previously “undruggable” diseases. But cell and gene therapies also have unique characteristics that can lead to manufacturing challenges and extended regulatory timelines. As of February 2020, cell and gene therapies account for 12% of the preclinical pipeline and 16% of the clinical pipeline.

Given the scale of investments made to support these development efforts and advancements possible, drug sponsors have looked to the U.S. FDA to consider accelerating gene and cell therapy development. The regulatory body has responded by shifting its departmental structure to st…

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

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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…