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Research

Ocugen
Research

Ocugen Gains EMA Backing for Single Pivotal Trial to Support Stargardt Disease Gene Therapy Approval

August 16, 2025August 14, 2025 - by Timothy Alexander

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has received a key regulatory boost in its bid to bring a gene therapy for Stargardt disease to market, with the European Medicines …

Ocugen Gains EMA Backing for Single Pivotal Trial to Support Stargardt Disease Gene Therapy Approval Read More

US Department of Health and Human Services (HHS)
National / Research

HHS to Phase Out mRNA Vaccine Projects, Redirect Funding to Broader Platforms

August 10, 2025August 10, 2025 - by Maryann Pugh

WASHINGTON, D.C. — The U.S. Department of Health and Human Services (HHS) has announced a sweeping plan to wind down its mRNA vaccine development initiatives under the Biomedical Advanced Research …

HHS to Phase Out mRNA Vaccine Projects, Redirect Funding to Broader Platforms Read More
Aclaris Therapeutics
Research

Aclaris Reports Positive Phase 2a Results for ATI-2138 in Atopic Dermatitis Trial

August 1, 2025July 31, 2025 - by Timothy Alexander

WAYNE, PA — Aclaris Therapeutics, Inc. (NASDAQ: ACRS) has announced encouraging top-line results from a Phase 2a trial evaluating ATI-2138, its oral investigational treatment targeting moderate-to-severe atopic dermatitis (AD). The study …

Aclaris Reports Positive Phase 2a Results for ATI-2138 in Atopic Dermatitis Trial Read More

U.S. Food and Drug Administration
National / Research

FDA Shuts Down Sarepta Gene Therapy Trials After Three Deaths Linked to Liver Failure

July 21, 2025July 20, 2025 - by Maryann Pugh

WASHINGTON, D.C. — The U.S. Food and Drug Administration on Friday placed Sarepta Therapeutics’ gene therapy trials for limb girdle muscular dystrophy on clinical hold following the deaths of three patients …

FDA Shuts Down Sarepta Gene Therapy Trials After Three Deaths Linked to Liver Failure Read More
Ocugen
Research

Ocugen Doses First Patient in Pivotal Trial for Stargardt Gene Therapy

July 19, 2025July 18, 2025 - by Timothy Alexander

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial evaluating OCU410ST, a gene therapy candidate designed …

Ocugen Doses First Patient in Pivotal Trial for Stargardt Gene Therapy Read More

Progenra
Research

Progenra Breakthrough Offers New Hope for Parkinson’s Disease and Beyond

July 15, 2025July 14, 2025 - by Timothy Alexander

MALVERN, PA — In a major scientific advance, Progenra Inc. has announced the discovery of a novel class of small-molecule drugs designed to restore the function of mutant forms of …

Progenra Breakthrough Offers New Hope for Parkinson’s Disease and Beyond Read More
Palvella Therapeutics
Research

Palvella Therapeutics Announces Completion of Phase 3 SELVA Trial for QTORIN Rapamycin

June 24, 2025June 23, 2025 - by Timothy Alexander

WAYNE, PA — Palvella Therapeutics, Inc. (Nasdaq: PVLA) has successfully completed enrollment in the Phase 3 SELVA clinical trial of QTORINâ„¢ 3.9% rapamycin anhydrous gel, a potential breakthrough treatment for …

Palvella Therapeutics Announces Completion of Phase 3 SELVA Trial for QTORIN Rapamycin Read More

Aclaris Therapeutics
Research

Aclaris Therapeutics Begins Phase 1a/1b Trial for Investigational Bispecific Antibody ATI-052

June 23, 2025June 23, 2025 - by Timothy Alexander

WAYNE, PA — Aclaris Therapeutics, Inc. (NASDAQ: ACRS) has launched a Phase 1a/1b clinical program to evaluate the safety and efficacy of ATI-052, an investigational bispecific anti-TSLP/IL-4R antibody that could …

Aclaris Therapeutics Begins Phase 1a/1b Trial for Investigational Bispecific Antibody ATI-052 Read More
DNA
National / Research

FDA Investigates Clinical Trials Sending U.S. DNA Overseas

June 23, 2025June 22, 2025 - by Maryann Pugh

WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has initiated an immediate review of clinical trials involving the international transfer of Americans’ living cells for genetic engineering and subsequent …

FDA Investigates Clinical Trials Sending U.S. DNA Overseas Read More
PleoPharma
Research

PleoPharma Presents Promising Phase 2b Data on PP-01 for Cannabis Withdrawal

June 20, 2025June 19, 2025 - by Timothy Alexander

PHOENIXVILLE, PA — PleoPharma, Inc. shared encouraging findings from its Phase 2b clinical trial of PP-01, a novel treatment for Cannabis Withdrawal Syndrome (CWS), during the 2025 annual meeting of …

PleoPharma Presents Promising Phase 2b Data on PP-01 for Cannabis Withdrawal Read More

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