Lou Gehrig's disease - Pipeline Insight, 2024

This “Lou Gehrig's disease - Pipeline Insight, 2024,” report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in Lou Gehrig's disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Lou Gehrig's disease Understanding

Lou Gehrig's disease: Overview

Lou Gehrig’s disease is often called Amyotrophic Lateral Sclerosis (ALS). ALS is a progressive, degenerative disease that destroys the nerve cells that control voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking.It affects nerves in the brain and spinal cord that control muscles. The result is progressive muscle weakness and incoordination, causing difficulty with mobility, speaking, swallowing and breathing. Gradually the body becomes paralyzed, which means that the muscles no longer work. The exact cause is unknown, but environmental and genetic factors may be involved. ALS tends to strike in mid-life between the ages of 40 and 60, but others can develop the disease. ALS can be classified as either sporadic or familial. Sporadic ALS occurs randomly, there is no clear factor or cause. Familial ALS is inherited due to change in gene.

Lou Gehrig's disease - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Lou Gehrig's disease pipeline landscape is provided which includes the disease overview and Lou Gehrig's disease treatment guidelines. The assessment part of the report embraces, in depth Lou Gehrig's disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Lou Gehrig's disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Lou Gehrig's disease R&D. The therapies under development are focused on novel approaches to treat/improve Lou Gehrig's disease.

Lou Gehrig's disease Emerging Drugs Chapters

This segment of the Lou Gehrig's disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Lou Gehrig's disease Emerging Drugs

BIIB067: Ionis Pharmaceuticals, IncBIIB067 (Tofersen) is a second generation antisense oligonucleotide designed to reduce the production of superoxide dismutase 1 (SOD1), the cause of a genetic form of ALS, referred to as SOD1-ALS, that results from mutations in the SOD1 gene. Tofersen is currently in a Phase 3 clinical trial in SOD1-ALS patients led by Ionis Pharmaceuticals, Inc in collaboration with Biogen.

Arimoclomol: OrphazymeOrphazyme is developing arimoclomol in four indications: Niemann-Pick disease Type C (NPC), Gaucher disease, Amyotrophic Lateral Sclerosis (ALS), and Inclusion Body Myositis (IBM). Arimoclomol has been shown to increase the production of cell protective HSPs. This increase in the production of naturally occurring HSPs inside the cells, reduces protein misfolding and aggregation and improves lysosomal function. Arimoclomol is currently in its phase III of development.

ALZT-OP1a: AZTherapies, Inc.

AZTherapies, Inc. a biopharmaceutical company leading the advanced clinical trials to treat neuroinflammatory diseases, ALZT-OP1a is currently in Phase 2a stage of development examining the safety and efficacy in patients with mild- to moderate-stage Amyotrophic Lateral Sclerosis (ALS). ALZT-OP1a is the company’s proprietary inhaled formulation of cromolyn, engineered to improve blood brain barrier permeability and bioavailability.

RNS60: Revalesio Corporation.

RNS60 is a leading clinical drug candidate to treat amyotrophic lateral sclerosis (ALS) and other neurological diseases. It can be administered by IV infusion or inhalation (nebulization).RNS60 is currently being tested in a placebo-controlled, multicenter ALS Phase 2 study. RNS60 acts on mitochondria and activates intracellular signaling pathways that have anti-inflammatory effects and promote cellular survival and differentiation. Thereby, RNS60 protects neurons and oligodendrocytes, and helps the immune system restore homeostasis through modulation of immune cell activity in the central nervous system and throughout the body.

Lou Gehrig's disease: Therapeutic Assessment

This segment of the report provides insights about the different Lou Gehrig's disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Lou Gehrig's disease

There are approx. 30+ key companies which are developing the therapies for Lou Gehrig's disease. The companies which have their Lou Gehrig's disease drug candidates in the most advanced stage, i.e. phase III include, Ionis Pharmaceuticals Inc.

Phases

This report covers around 30+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II and phase I/II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Lou Gehrig's disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Inhalation
• Molecule Type

Products have been categorized under various Molecule types such as

• Peptides
• Small molecule
• Oligonucleotides
• Gene Therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Lou Gehrig's disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Lou Gehrig's disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Lou Gehrig's disease drugs.

Lou Gehrig's disease Report Insights

• Lou Gehrig's disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Lou Gehrig's disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Lou Gehrig's disease drugs?
• How many Lou Gehrig's disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Lou Gehrig's disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to Lou Gehrig's disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Lou Gehrig's disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Ionis Pharmaceuticals Inc.
• AZTherapies Inc.
• Revalesio Corporation.
• AB science
• Biogen
• Hoffmann-La Roche
• Mitsubishi Tanabe Pharma Development America Inc.
• Orphazyme
• Q Therapeutics, Inc.
• Genuv Inc.
• Orion Corporation
• AL-S Pharma
• Immunity Pharma Ltd.
• Everfront Biotech Co., Ltd.

Key Products

• IPL344
• BIIB078
• MT-1186
• Levosimendan
• HK-001
• IPL344
• Arimoclomol
• Dexpramipexole
• BIIB078
• Olesoxime
• ALZT-OP1a
• Masitinib

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