Étiquette : Rare diseases

INFLECTIS BIOSCIENCE

by jbertholdin Biotech, Pharma

INFLECTIS BIOSCIENCE

About the company

InFlectis is a French-based drug discovery company, incoporated in 2013, developing first-in-class therapies for neurological rare diseases. They are in clinical development with an orally available small molecule that has the potential to extend lives or improve the quality-of-life for people suffering from ALS. They are also ready to enter in phase 2 clinical trial in Charcot-Marie-Tooth disease and preclinical proof of efficacy has been obtained in rare cerebellar ataxias.

About the solution

IFB-088 is a first-in-class orally available small molecule drug candidate with a validated mechanism of action and a promising pharmacokinetic profile capable of crossing the blood brain barrier to target the central and peripheral nervous system. IFB-088 is a multifunctional drug that modulates the PPP1R15A/PP1c phosphatase complex and inhibits NR2B-containing NMDAR. The potential of IFB-088 lies in its ability to target different major cellular pathophysiological mechanisms to manage disease progression: protein aggregation, oxidative stress, and glutamate excitotoxicity.

Website
Key information

–  Therapeutic areas: Neurological rare diseases

–  Based in: Nantes (FRANCE)

–  Employees: 1-10

–  Created in: 2013

MABSILICO

by jbertholdin Biotech, Pharma

MABSILICO

About the company

MAbSilico is the TechBio pionner company specializing in computational solutions for discovering and designing therapeutic antibodies. Their solutions leverages artificial intelligence (AI) and machine learning to characterize, optimize and design therapeutic antibodies.

MAbSilico’s platform aims to accelerate the antibody discovery process by structural design with epitope-driven and multiparametric approaches. This allows to design highly effective antibodies, saving time and resources compared to traditional methods. We provide fully characterized antibodies against a target within 21 days that are internally validated with wet lab assays, leading to faster development of life-saving antibody therapies.

About the solution

MAbSilico uses a combination of 3D modeling, interaction simulation, and sequence analysis to identify promising antibody candidates after assessing for various factors, including their binding target (epitope), affinity, potential off-target effects, and developability.

While the solutions can be applied to antibody characterization, optimization and design, MAbSilico does not need to generate biological data to apply their solutions.

Integrating multi-parameters for the antibody design and validating the newly sequence with state of the art biological assays, MAbSilico is pioneering a game-changing approach to antibody discovery with internal discovery pipeline.

Website
Key information

–  Therapeutic areas: Computational antibody design & discovery applied in oncology, Immuno-oncology, Infectious diseases, Rare diseases

–  Based in: Tours & Paris (FRANCE)

–  Employees: 11 – 50

–  Created in: 2017

CLINSEARCH

by jbertholdin Institutions, Service Providers & others

CLINSEARCH

About the company

ClinSearch is a full service European CRO, established in 1999, offering clinical research and medical evaluation services throughout Europe, as well as the Middle East and the United States.

They have conducted hundreds of studies, from phase I to IV studies, early access programs, post-marketing studies, RWE, PASS, PAES, database analyses and medico-economic studies, involving 1 patient up to 220,000 subjects.
Their customers include global and local medical device, biotechnology and pharmaceutical companies, as well as public health institutions.

About the solution

Full service offering from A to Z: methodological input to study design through to peer-reviewed publications, passing via in-house development of sophisticated healthcare tools (EDC, ePRO, …).

The in-house team of over 70 people work from our headquarters in Malakoff (France), but hail from ~15 different countries and speak over 10 languages, and are trained to a high standard (MD, PhD, PharmD, MS, MPH …).

Website
Key information

–  Therapeutic areas: Addiction, Aesthetics, Algorithm, Software, Cardiovascular, Dermatology, Fertility, Immunology, Infectiology, Hematology, Neurology, Neuromuscular, Neurovascular, Nursing care, Orthopedic, Oncology, Rheumatology, Rare diseases, Respiratory, Surgery, Urology

–  Based in: Boston (USA)

–  Employees: 51-200

–  Created in: 1999

B2G LIFE SCIENCES

by jbertholdin Institutions, Service Providers & others

B2G LIFE SCIENCES

About the company

B2G Life Sciences is a science-driven, international, medical consulting company, dedicated to clinical development.
They serve as acting CMO, yet also provide extended stand-alone as well as fully-integrated services, to the biotechnology and pharmaceutical industry, with a particular focus on rare and orphan diseases, oncology, as well as biotherapies and advanced therapies.

They have operational capacities across Europe and the Mediterranean region, as we endeavor to build bridges and gateways between operators in different markets.

About the solution

They serve as acting CMO and provide extended stand-alone as well as fully-integrated consulting services to the biotechnology and pharmaceutical industry, with a particular focus on rare and orphan diseases, oncology,
as well as biotherapeutics and advances therapies.

From study design to execution, they provide services in the following focus areas:
– Clinical development strategies and protocol development
– Management of scientific boards
– Study conduct and patient enrollment
– Medical review and medical writing
– Trainings in the busnisses of biotech and pharma industries.

Website
Key information

–  Therapeutic areas: Clinical Development Consulting services, Rare diseases, Oncology, Biotherapies, Advanced therapies

–  Based in: Sèvres (FRANCE)

–  Employees: 11 – 50

–  Created in: 2011

SATT AxLR

by jbertholdin Institutions, Service Providers & others

SATT AxLR

About the company

SATT AxLR is a TTO based in south of France with a focus on innovative health projects from academic teams. SATT AxLR is funding early stages to allow project reaching a stage for technical transfer or company creation.

About the solution

SATT AxLR provide advice, contacts, funding to academic researchers (Universities, Hospitals, Public Instittutes,..) and generate intellectual property that is available for licensing.

Website
Key information

–  Therapeutic areas: Oncology, Immunology, CNS, Cardiology, Rare diseases, Obesity, Cell therapy, Medical Device

–  Based in: Montpellier (FRANCE)

–  Employees: 11 – 50

–  Created in: 2012

CEVIDRA

by jbertholdin Biotech, Pharma

CEVIDRA

About the company

Founded 17 years ago, Cevidra is an independent pharmaceutical company with a strong expertise in hospital affairs, particularly in tenders, and a vast network of experts. As a rapidly growing entity in the early access domain, Cevidra acts as a gateway to the French market for foreign companies. Rooted in innovation, agility, and commitment, Cevidra offers comprehensive services from its headquarters in Grasse, with additional offices in Paris and Lyon.

Holding GDP/GMP certifications, it excels as an « Exploitant » for pharmaceutical products, specializing in distribution through its logistical platform. Cevidra provides services ranging from regulatory affairs to market access and real-world data collection. It has a strong track record in managed access programs across various areas, including ATMPs.

About the solution

As an Exploitant with an in-house GDP platform, Cevidra offers flexible support to foreign companies launching medicines in France. They handle compassionate/early access programs, market approval, and distribution, including market access and promotional activities in hospitals and pharmacies. Their tailored approach ensures a seamless entry into the French market.

Website
Key information

–  Therapeutic areas: All therapeutic areas including Oncology, Rare diseases and Addictive medicine.

–  Based in: Grasse, Lyon and Paris (FRANCE)

–  Employees: 11 – 50

–  Created in: 2007

THERAVIA (ADDMEDICA)

by jbertholdin Biotech, Pharma

THERAVIA (ADDMEDICA)

About the company


THERAVIA is a European-based company dedicated to the development, marketing, and distribution of innovative and original products dedicated to serious conditions, rare diseases, and unmet medical needs. Formed in 2023 through the merger of two French companies (ADDMEDICA and CTRS), they benefit from their track records in drug development, registration, and commercialization, as well as from a strong hospital presence and a solid KOL network, especially for rare disorders. They also benefit from a robust commercial network in all countries of Western Europe.

They are supported by MERIEUX EQUITY PARTNERS as a reference shareholder to accelerate their growth in France and internationally. To expand their portfolio, they are now looking for new products with the following features:

– Designed to treat a limited number of patients

– And/or highly innovative products

– And/or addressing emerging markets with few competitors and requiring specialized sales & distribution processes

About the solution

Theravia is a leader in some rare diseases’ treatment, having, for example, marketed a safe and easy-to-dose form of hydroxyurea called « Siklos » in Europe, the US (https://siklosusa.com/), and other territories to treat sickle cell disease. They invest in large clinical trials to advance the knowledge of the disease and its treatment. For instance, they are conducting ESCORT-HU, a Phase IV observational cohort study that includes more than 2,000 patients.

Website
Key information

–  Therapeutic areas: Rare diseases, Neglected disorders, Paediatric diseases

–  Based in: Ile de France (FRANCE)

–  Employees: 11 -50

– Created in: 2005

IKTOS

by jbertholdin Biotech, Digital Health, Pharma

IKTOS

About the company

Incorporated in October 2016, Iktos is a French start-up company specialized in the development of artificial intelligence solutions applied to chemical research, more specifically medicinal chemistry and new drug design.

Iktos is developing a proprietary and innovative solution based on deep generative models, which enables, using existing data, to design molecules that are optimized in silico to meet all the success criteria of a small molecule discovery project.

The use of Iktos technology enables major productivity gains in upstream pharmaceutical R&D. Iktos offers its technology through the SaaS software platforms Makya™ for generative drug design and Spaya™ for retrosynthesis, and through strategic collaborations with pharma companies where Iktos mobilizes its unique platform and leading-edge capabilities to expedite small molecule drug discovery for the benefit of its partners. Iktos is also developing Iktos Robotics, a unique AI-driven synthesis automation platform which dramatically accelerates the Design-Make-Test cycle in drug discovery.

About the solution

Iktos offers its technology through the SaaS software platforms Makya™ for generative drug design and Spaya™ for retrosynthesis, and through strategic collaborations with pharma companies where Iktos mobilizes its unique platform and leading-edge capabilities to expedite small molecule drug discovery for the benefit of its partners. Iktos is also developing Iktos Robotics, a unique AI-driven synthesis automation platform which dramatically accelerates the Design-Make-Test cycle in drug discovery.

Iktos offers the possiblity to its partners to use it unique technology via proprietary SaaS software platforms: Makya for de novo generative design and Spaya for retrosynthesis and synthetic access. Its partners can also rely on Iktos expertise to run integrated drug discovery projects from hit discovery, hit-to-lead, lead optimization, all the way down to preclinical drug candidate.

Website
Key information

–  Therapeutic areas: Any therapeutic areas including oncology, CNS / neurology, Immunology, Infectious diseases, Rare diseases

–  Based in: Headquarters and lab: Paris (FRANCE)
Additional offices: US and JAPAN

–  Employees: 51 – 200

– Created in: 2016

CONTACT
Bose AvirupChief Business Officer

avirup.bose@iktos.com