Retinitis Pigmentosa - Pipeline Insight, 2024

This “Retinitis Pigmentosa- Pipeline Insight, 2024” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Retinitis Pigmentosa 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

Retinitis Pigmentosa: Understanding

Retinitis Pigmentosa: Overview

Retinitis pigmentosa (RP) is an umbrella term for a group of hereditary progressive retinal disorders that cause progressive degeneration of the retina. The disease is inherited as autosomal recessive, autosomal dominant or X-linked recessive traits. RP is a genetic disease that people are born with. Symptoms usually start in childhood, and most people eventually lose most of their sight. There’s no cure for RP. But vision aids and rehabilitation (training) programs can help people with RP make the most of their vision.

Signs of retinitis pigmentosa usually appear during childhood or adolescence. Common symptoms include difficulty seeing at night and a loss of side (peripheral) vision. RP also causes loss of side (peripheral) vision - so have trouble seeing things out of the corners of the eyes. Over time, field of vision narrows until only have some central vision (also called tunnel vision). Some people with RP lose their vision more quickly than others. Eventually, most people with RP lose their side vision and their central vision. Other symptoms of RP also include sensitivity to bright light and loss of color vision. Most of the time, RP is caused by changes in genes that control cells in the retina. These changed genes are passed down from parents to children. RP is linked to many different genes and can be inherited in different ways. If have RP, can talk with doctor or a specialist called a genetic counselor to learn more about risk of passing RP to children. Sometimes RP happens as part of other genetic conditions, like Usher syndrome. Usher syndrome causes both vision and hearing loss.

RP is diagnosed by electroretinography (ERG), visual field testing, and retinal imaging, fundus auto-fluorescence (FAF) and genetic testing. Other tests for RP include: Optical coherence tomography (OCT), Genetic testing. Currently, there is no therapy that stops the evolution of pigmentary retinopathies or restores the vision.

Management of retinitis pigmentosa includes dietary supplements, ozone therapy, surgery etc. Gene therapy based treatments are under research focus for the treatment of Retinitis pigmentosa. The other ways to manage RP include: Using low vision aids and assistive devices. There are a range of magnifiers and technology that can identify things or people that the wearer points to, using sunglasses and other methods to avoid exposure to too much light. Light may make RP worse, treating associated conditions, such as cystoid macular edema (CME), which may happen with RP. CME refers to collections of fluid in the middle of retina, treating cataracts, which happens when the lens of eyes becomes cloudy, with surgery to remove them.

Retinitis Pigmentosa- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Retinitis Pigmentosa pipeline landscape is provided which includes the disease overview and Retinitis Pigmentosa treatment guidelines. The assessment part of the report embraces, in depth Retinitis Pigmentosa 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, Retinitis Pigmentosa 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 Retinitis Pigmentosa R&D. The therapies under development are focused on novel approaches to treat/improve Retinitis Pigmentosa.

Retinitis Pigmentosa Emerging Drugs Chapters

This segment of the Retinitis Pigmentosa 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.

Retinitis Pigmentosa Emerging Drugs

Botaretigene sparoparvovec : Meira GTx Botaretigene sparoparvovec (bota-vec) is designed to treat the most common form of X-linked retinitis pigmentosa (XLRP) caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function. AAV-RPGR has received Fast Track and Orphan Drug designations from the FDA, as well as PRIME, ATMP and Orphan Medicinal Product designations from the EMA. Currently, the drug is in Phase III stage of its development for the treatment ofrenitispigmentosa.

ADX-2191: Aldeyra Therapeutics, Inc.ADX-2191 is a novel drug candidate being developed by Aldeyra Therapeutics, Inc. The drug candidate is tetrahydrofolate dehydrogenase inhibitors and thymidylate synthase inhibitors. ADX-2191 received orphan drug designation in 2018. Currently, the drug is in Phase II stage of its development for the treatment of renitis pigmentosa.

SPVN-06 : Sparing Vision SPVN06, is a novel product candidate that acts independently of the causative gene mutation. SPVN06 counteracts the degeneration of cone photoreceptors by restoring Rd CVF, a neurotrophic factor naturally produced by functioning rods in the retina; and by promoting RdCVFL, a potent antioxidant which protects cones against oxidative stress. The DNA of the two distinct isoforms (RdCVF and RdCVFL) of the NXNL1 gene are supplied via an Adeno-associated virus (AAV), a viral vector classically used in gene therapy. SPVN06 is delivered via the subretinal route to the cones and retinal pigmented epithelium (RPE). Upon delivery of SPVN06, the transgenes are mainly expressed in the RPE cells for RdCVF and in the cones for RdCVFL. SPVN06 is expected to provide a long-lasting neuroprotective effect to prevent the progression of RP in patients with moderate to severe pathology. Currently, the drug is in Phase I/II stage of its development for the treatmentofrenitispigmentosa.

FT-002: Frontera Therapeutics FT-002 is a recombinant adeno-associated virus gene therapy drug intended to treat patients with X-linked retinitis pigmentosa (XLRP) caused by RPGR gene mutation. There is no treatment for this disease globally. FT-002 is an intraocular injection of recombinant AAV virus carrying the gene that expresses active functional proteins and repairs damaged retinal cell structure and function. One injection can effectively delay disease progression or restore the patient’s visual function. FT-002 is the first AAV gene therapy drug to be tested in XLRP patients in China and is also a potential first-in-class drug. Currently, the drug is in Phase I stage of its development for the treatment of renitis pigmentosa.

Retinitis Pigmentosa: Therapeutic Assessment

This segment of the report provides insights about the different Retinitis Pigmentosa drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Retinitis Pigmentosa

• There are approx. 40+ key companies which are developing the therapies for Retinitis Pigmentosa. The companies which have their Retinitis Pigmentosa drug candidates in the most advanced stage, i.e. phase III include, MeiraGTx

Phases

This report covers around 40+ products under different phases of clinical development like

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

Route of Administration

Retinitis Pigmentosa 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
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Retinitis Pigmentosa: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Retinitis Pigmentosa 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 Retinitis Pigmentosa drugs.

Retinitis Pigmentosa Report Insights

• Retinitis Pigmentosa Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Retinitis Pigmentosa 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 Retinitis Pigmentosa drugs?
• How many Retinitis Pigmentosa drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Retinitis Pigmentosa?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Retinitis Pigmentosa 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 Retinitis Pigmentosa and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• MeiraGTx
• Aldeyra Therapeutics, Inc.
• SparingVision
• Frontera Therapeutics
• Pro QRTherapeutics
• Bionic Sight
• Retro SenseTherapeutics
• Dompe Farmaceutici
• Endogena Therapeutics
• Nacuity Pharmaceuticals
• Kiora Pharmaceuticals
• jCyte
• Ocugen
• Neurotech USA
• Nanoscope Therapeutics

Key Products

• Botaretigene sparoparvovec
• ADX-2191
• SPVN-06
• FT-002
• Ultevursen
• BS 01
• RST-001
• Cenegermin
• EA-2353
• NPI-001
• KIO-301
• Retinal stem cell therapy
• OCU400
• NT-501
• MCO-010

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