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Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics is making significant strides in developing gene therapies for inherited retinal diseases (IRDs), specifically OPGx-LCA5 for LCA5-associated IRD and OPGx-BEST1 for BEST1-associated IRD. According to Dr. George Magrath, CEO of Opus Genetics, the OPGx-LCA5 program, targeting an ultra-rare disease affecting approximately 200 U.S. children, has shown promising results. All six participants treated to date (three pediatric and three adult) demonstrated improvement across various measures, including full-field stimulus threshold (FST) testing, best-corrected visual acuity (BCVA), virtual-reality maze tests, and microperimetry. Retinal sensitivity improved by about 1.5 log units at both blue and red wavelengths. The company has also secured FDA alignment for an eight-participant Phase 3 registrational trial for OPGx-LCA5, utilizing a 6-month pretreatment period where each participant serves as their own control. The OPGx-BEST1 program aims to restore bestrophin-1 channel function in the retinal pigm...

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Vyriad Initiates Phase 1 Clinical Trial for In Vivo CAR T Cell Therapy VV169 in Multiple Myeloma

Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

Representatives Introduce Bipartisan Bill to Permanently Authorize Rare Pediatric Disease Priority Review Voucher Program

U.S. Representatives Gus Bilirakis (R-FL), Nanette Barragán (D-CA), Kat Cammack (R-FL), Jake Auchincloss (D-MA), Tom Kean, Jr. (R-NJ), and Kevin Mullin (D-CA) have introduced bipartisan legislation to permanently authorize the U.S. Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) Program. This program, established in 2012, incentivizes the development of treatments for children suffering from rare and devastating diseases. The PRV program awards a transferable priority review voucher to companies that successfully develop and receive FDA approval for qualifying treatments. Since its inception, the program has facilitated the market entry of treatments for 47 rare pediatric diseases, a significant increase from the four FDA-approved treatments that existed before the program. The current authorization for the program is set to expire in 2029. The proposed permanent authorization aims to provide long-term certainty for researchers and investors, encouraging continued ...

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

Wellness Influencers Promote Berberine as "Nature's Ozempic" in the U.S., Despite Traditional Use

Tenaya Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for TN-401

Tenaya Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for TN-401

Tenaya Therapeutics, a clinical-stage biotechnology company, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for its gene therapy candidate, TN-401. This designation is for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), a serious heart disease. The RMAT designation is an expedited program designed to facilitate the development and review of regenerative medicine therapies for severe conditions where preliminary clinical evidence suggests the potential to address unmet medical needs. Tenaya's RMAT application included data from the RIDGE-1 clinical trial, which showed improvements in electrical stability measures, such as reduced rates of premature ventricular contractions (PVCs) and non-sustained ventricular tachycardias (NSVTs), common forms of ventricular arrhythmias in ARVC. The company's CEO, Faraz Ali, highlighted that this designation underscores the recognition of the seriousness of PK...

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics, Inc., a clinical-stage biotechnology company, has announced the enrollment of its 100th participant in the Phase 2/3 ALSTARS Trial (NCT07161999) for COYA 302. This investigational drug, a combination of proprietary low-dose IL-2 and CTLA-4 Ig, is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The company anticipates completing the full enrollment of 120 participants in the coming weeks, with topline data expected in early Q2 2027. COYA 302 has received FDA Fast Track designation for ALS treatment. The ALSTARS Trial is a randomized, multi-center, double-blind, placebo-controlled study designed to assess the efficacy and safety of COYA 302. Participants are randomized to receive one of two dosing regimens or a placebo over a 24-week double-blind treatment phase, followed by a 24-week blinded active-treatment extension phase. The primary endpoint focuses on changes in disease severity as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised...

FDA Approves New Warnings for Kybella Injections Due to Adverse Reactions from Unapproved Use

FDA Approves New Warnings for Kybella Injections Due to Adverse Reactions from Unapproved Use

The U.S. Food and Drug Administration (FDA) has approved significant updates to the labeling for Kybella (deoxycholic acid) injection. These updates include new warnings regarding adverse reactions that can occur when the drug is used outside its approved indication, which is for submental fat (under the chin). The FDA now explicitly recommends against this unapproved use. This decision follows a comprehensive review of reports from the FDA Adverse Event Reporting System (FAERS) and medical literature, which revealed that administering Kybella outside the submental fat region can lead to serious neuromuscular and other adverse events. Additionally, the FDA has added language to the Warnings and Precautions section concerning the serious risks of injection site nodules (3 cm or smaller) and masses (larger than 3 cm). These nodules and masses may not resolve over time and could require medical intervention, occurring even when Kybella is used as indicated in the submental area, as well as in unapproved areas...

Pennsylvania Governor Josh Shapiro Calls for Federal AI Oversight Amid Industry Warnings

Pennsylvania Governor Josh Shapiro Calls for Federal AI Oversight Amid Industry Warnings

Pennsylvania Governor Josh Shapiro has publicly urged President Trump and Congress to implement federal regulations for artificial intelligence (AI) development. His call comes in response to warnings from AI industry leaders, including the CEO of Anthropic, Dario Amodei, who suggested that unchecked AI advancement poses significant risks, potentially leading to catastrophic outcomes. Governor Shapiro emphasized that AI development should not be solely controlled by private entities, especially when these companies themselves are advocating for restraint and regulation. He drew parallels between the need for AI regulation and the existing federal oversight in industries like pharmaceuticals and aircraft manufacturing, where products must meet strict standards set by agencies such as the FDA and FAA before reaching consumers. Shapiro's statements highlight a growing concern among some political figures and industry experts about the rapid progression of AI and the perceived lack of governmental action to es...

FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin

FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin

FDA Accepts Curi Bio's 3D Neuromuscular Junction Assay for ISTAND Program, Advancing Non-Animal Testing for Botulinum Toxin

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

TwoStep Therapeutics Secures $62.5 Million Series A Funding and FDA Clearance for Cancer Drug

FDA holds public hearing on psychedelic drugs as Trump-driven push gathers pace

FDA holds public hearing on psychedelic drugs as Trump-driven push gathers pace

By Puyaan Singh and Kamal Choudhury Sept 14 (Reuters) - The U.S.