Regenxbio

AbbVie Inc. (ABBV) stock at $173.90, down 2.58%, with a market cap of $307.306B. Specializes in pharmaceuticals, including Humira, Skyrizi, and Rinvoq, for various diseases. Offers products for eye care, cancer, and more. Collaborates with several biotech firms. Fiscal year ends December 31, headquartered in North Chicago, Illinois.

Ultragenyx Pharmaceutical Inc. (RARE) focuses on novel treatments for rare genetic diseases, with products like Crysvita and Mepsevii. It collaborates globally and reported a market cap of $4.031B, with a YTD return of 3.26% as of January 8, 2025.

Viking Therapeutics (VKTX) received a Buy rating from Stifel Nicolaus analyst Annabel Samimy, citing promising advancements in its VK2735 programs. The Phase 2 VENTURE study for oral VK2735 targets obesity with potential for success, while the injectable version is set for Phase 3 trials in 2025. Viking's dual-format strategy enhances market appeal. Morgan Stanley also maintains a Buy rating with a $105 target.

The Retinal Gene Therapy Market is projected to exceed $110 million in 2024, driven by the increasing prevalence of inherited retinal diseases, clinical trial successes, and advancements in gene therapy technologies. Despite growth, high costs and limited treatment reach pose challenges. The market is expected to evolve significantly by 2034, with opportunities in diversification, combination therapies, and precision medicine.

AbbVie partners with Regenxbio, paying $370M upfront for RGX-314, a gene therapy targeting wet AMD and other eye diseases. RGX-314, in phase 2/3 trials, offers a one-off treatment alternative to frequent eye injections. The deal includes $1.4B in milestones, with profit-sharing and royalties for Regenxbio.

Stifel Nicolaus analyst Annabel Samimy initiated a Buy rating for Oculis Holding (OCS), citing promising clinical results of OCS-05 for acute optic neuritis treatment. The drug showed significant improvements in visual outcomes and safety, with FDA clearance for further trials. H.C. Wainwright also reiterated a Buy rating with a $30 target.

The global mucopolysaccharidosis (MPS) treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth drivers include advancements in biotechnology, enhanced diagnostic methods, increased awareness of rare diseases, and the availability of enzyme replacement therapy (ERT). Clinical trials and new drug approvals, such as Lysogene's LYS-SAF302 and JCR Pharmaceuticals' JR-141, are pivotal. Despite high treatment costs and reimbursement challenges, the market is set to expand, with ERT remaining the preferred treatment.

Novartis's phase III STEER study showed OAV101 IT improved motor function in SMA Type 2 patients, with a favorable safety profile. Results support OAV101 IT's potential as a one-time gene therapy for SMA, aiming for regulatory approval by 2025.

The Age-related Vision Dysfunction Market report by DelveInsight provides insights into treatment trends, epidemiology, and market forecasts from 2020 to 2034. It highlights key companies and therapies, including bevacizumab and Rocklatan, and discusses the prevalence of conditions like AMD, glaucoma, and cataracts in the US, EU4, UK, and Japan.