Gene therapy

Gene therapy Agency Consulting firm Experts Specialists Consultancy

Develop new therapeutic approaches and market access strategies

The Alcimed Healthcare team has been exploring the field of gene therapy for more than 25 years, and supports its clients in their innovation and development projects around gene therapy drugs: from research to pharmaceutical development, up to market launch.

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    The challenges related to gene therapy and gene therapy drugs

    Initially designed in the 1970s and 1980s with the aim of treating diseases linked to the dysfunction of a single gene (known as monogenic diseases), gene therapy has proved over the years to be promising in fields much broader than genetic diseases: oncology, neurology, ophthalmology, cardiovascular, etc.

    The field is therefore experiencing a strong growth and since 1989, over 2,000 clinical trials have been carried out or are in progress, and more than 20 gene therapies have been approved to date.

    Even though this market is developing, the pharmaceutical industry, biotechs and research centers are facing many scientific and business challenges:

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      How we support you in your projects related to gene therapy and gene therapy drugs

      For more than 25 years, Alcimed has supported its clients on many issues related to gene therapies and gene therapy drugs, in many therapeutic areas such as neurology, oncology, cardiology and rare diseases.

      We have thus carried out dozens of projects on gene therapy for different players such as national and European institutions and research centers (for example AFM / Genethon), pharmaceutical companies (for example Bayer, Novartis or Pfizer) biotechs (for example Vertex or Spark Therapeutics (Roche)) or patient associations.

      The diversity of our clients, the topics we cover, the geographic areas and the therapeutic areas we explore, and the types of projects we develop give us comprehensive and in-depth insight into issues related to gene therapy.

      Our projects cover areas as diverse as new technologies in the field of gene therapies, the development of portfolio strategies, the assessment of market opportunities, the search for partners, the development of business cases, and many more!

      Examples of recent projects carried out for our clients in gene therapy

      • Construction of a portfolio strategy in gene therapy

        One of our clients, a major player in gene therapy, wanted to understand how to change its portfolio strategy by engaging in new therapeutic areas.

        To this end, Alcimed carried out 7 opportunity assessments for different diseases by identifying their potential (disease severity, impact on the healthcare system, opportunity for gene therapy, patient population, etc.) and the associated risk (current treatments, risks for gene therapy linked to the healthcare structure already in place, feasibility of a clinical trial, etc.).

        With the business cases built, the new therapeutic areas considered were compared and prioritized, the synergies evaluated, in order to orient our client’s developments in a portfolio logic.

      • Identification and evaluation of relevant technologies to maintain a platform of excellence in gene therapy

        Alcimed has supported a major player in gene therapy in the identification and analysis of 24 families of emerging technologies that could potentially disrupt their activity, for example in the field of immunotolerance or capsid engineering.

        The technologies were prioritized by our team according to their level of maturity (proofs, first tests, etc.) and the opportunities or risks they represented for this player.

        Then, during a working session with all the members of the management committee, the technologies allowing to maintain a leadership position were selected to lead to the definition of an innovation strategy.

      • Creation of an innovative access model for "first-in-class" gene therapy

        We supported a European pharmaceutical company in the pre-launch phase of the first gene therapy for a given pathology. The question was how to best prepare the acceptance by the healthcare authorities of this innovative therapy, delivered punctually and a priori with a high cost compared to the reference drugs.

        We were able to assess the perception of public institutions and prescribers of the value provided by this gene therapy drug, thanks to discussions and a benchmark of already approved gene therapies, then tested the potential of different funding models for this biotherapy (ex: “Pay-for-Performance”, subscription model, etc.).

        An innovative access model emerged, supported by a pan-European action plan to ensure the successful marketing of our client’s gene therapy.

      • Positioning of a CMO’s gene therapy offer

        A European CMO (Contract Manufacturing Organization) wanted to assess the opportunity that the development of a gene therapy offerrepresented for it and to understand how to position its offer.

        To do this, Alcimed analyzed the competing offers offered by CMO players already present in gene therapy, to achieve a fine segmentation of the offer. Then, a field investigation of the demand made it possible to define the typology of target customers, their criteria for choosing partners, and the necessary associated services.

        The result for our client: a clear marketing strategy, and its associated technical and commercial roadmap.

      • Selecting the best development opportunities for a gene therapy in rare diseases

        We supported a biotech specializing in gene therapy for rare diseases in one of its strategic development projects. Our client’s goal was to build a franchise in the field of neuro-ophthalmology over the next 5 years.

        As the main challenge was linked to the nature of rare diseases (little or no information, highly dispersed patient populations, poorly optimized care pathways, strong disparities between geographies, etc.), we helped our client with a commercial pre-assessment of 3 rare ophthalmic diseases: disease description, epidemiological analysis, diagnosis and treatment practices, competitive landscape, and unmet needs, in order to guide our client towards commercial opportunities, indicate how to address them, and recommend which opportunity to work on first.

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