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News and Trends 28 Feb 2023

What’s happening on Rare Disease Day?

Rare Disease Day 2023 is on February 28. The day is dedicated to raising awareness for the approximately 300 million people around the world living with a rare disease. More than 6,000 rare diseases are characterized by a diversity of disorders and symptoms that vary not only from disease to disease, but also from patient […]

February 28, 2023 - 8 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

European Commission approves first gene therapy for hemophilia B

The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

News and Trends 28 Feb 2023

Peter MacCallum Cancer Centre and Cartherics to work on CAR-T cell therapy for ovarian cancer

Peter MacCallum Cancer Centre (Peter Mac) in Australia and Cartherics Pty Ltd have entered into a collaborative development program agreement (CDPA) to develop Cartherics’ proprietary autologous CAR-T cell therapy (CTH-004) for the treatment of ovarian cancer. The clinical trial to be conducted by Peter Mac will be the first time a CAR-T cell therapy product […]

February 28, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

GC Biopharma acquiring hemophilia rare disease pipeline

GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to acquire three programs related to the orphan hematology disorders. The agreement includes Marzeptacog alfa (MarzAA), an engineered factor VIIa, which is ready for phase 3 clinical stage development. In its previous clinical development trials, MarzAA […]

February 28, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 24 Feb 2023

Resalis Therapeutics receives €10M to develop obesity and NAFLD treatment

Resalis Therapeutics has completed a seed financing round of €10 million to establish a non-coding RNA (ncRNA)-based therapeutic approach for the treatment of metabolic diseases. The funding will support the finalization of IND-enabling studies for the Resalis Therapeutics’ lead program, RES-010, which Resalis will initially develop as a novel treatment for obesity and nonalcoholic fatty […]

February 24, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 24 Feb 2023

Beyond Biotech podcast 35: Rare Disease Day

As February 28 is Rare Disease Day, we have three guests on the podcast today all relevant to rare diseases. And they are Dan Mandell, CEO and co-founder of GRO Biosciences; Stefano Portolano, chief executive officer, Azafaros; and from Replay, Adrian Woolfson, executive chairman, president, and co-founder, and Lachlan MacKinnon, CEO and co-founder. Rare Disease […]

February 24, 2023

News and Trends 24 Feb 2023

AELIX announces ‘important step towards HIV eradication’

AELIX Therapeutics S.L., a clinical-stage biotechnology company specializing in the discovery and development of immunotherapies for HIV infection, has announced positive topline results from its AELIX-003 trial. The study evaluated the safety, tolerability, immunogenicity and efficacy of AELIX Therapeutics’ HTI T-cell therapeutic HIV vaccine in combination with Gilead’s investigational Toll-Like Receptor 7 (TLR7) agonist, vesatolimod […]

February 24, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 24 Feb 2023

Sanofi hemophilia drug gets FDA approval

The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […]

February 24, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 24 Feb 2023

Researchers discover how tumor cells become resistant to colorectal cancer chemotherapy

Platinum-based chemotherapy, which is used to treat advanced colorectal cancer, accumulates in the healthy cells surrounding the cancer cells and, as a result, can reduce tumor sensitivity to treatment. This is demonstrated by a study published in the journal Nature Communications by the Hospital del Mar Medical Research Institute (IMIM-Hospital del Mar), in collaboration with […]

February 24, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 23 Feb 2023

Depression trial gets green light

Beckley Psytech Ltd has received investigational new drug (IND) approval from the U.S. Food and Drug Administration (FDA) for a global multi-site phase IIb study exploring the safety, efficacy and tolerability of two doses of its lead compound, BPL–003, in patients with treatment resistant depression (TRD). BPL-003 is the Beckley Psytech’s novel synthetic formulation of […]

February 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 23 Feb 2023

Positive topline data from sepsis trial

Modus Therapeutics AB has published positive top-line data from its phase 1b lipopolysaccharide (LPS) provocation study, a key step in evaluating the potential of its lead asset, sevuparin, as a treatment for sepsis and other conditions with systemic inflammation. In the study, healthy volunteers received LPS to induce a transient systemic inflammation reaction together with […]

February 23, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 23 Feb 2023

New promising targeted drug for a rare leukemia

Targeted drugs have been developed to supplement chemotherapy in the treatment of cancer. These drugs only affect cancer cells, leaving healthy cells alone. Venetoclax is a new targeted therapy option for the treatment of acute myeloid leukemia (AML). It was recently granted marketing authorization in Finland. Venetoclax works by sensitizing cancer cells to programmed cell […]

February 23, 2023 - 3 minutesmins - By Jim Cornall

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News and Trends

What’s happening on Rare Disease Day?

European Commission approves first gene therapy for hemophilia B

Peter MacCallum Cancer Centre and Cartherics to work on CAR-T cell therapy for ovarian cancer

GC Biopharma acquiring hemophilia rare disease pipeline

Resalis Therapeutics receives €10M to develop obesity and NAFLD treatment

Beyond Biotech podcast 35: Rare Disease Day

AELIX announces ‘important step towards HIV eradication’

Sanofi hemophilia drug gets FDA approval

Researchers discover how tumor cells become resistant to colorectal cancer chemotherapy

Depression trial gets green light

Positive topline data from sepsis trial

New promising targeted drug for a rare leukemia

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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