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News and Trends 2 Mar 2023

Partnership formed to develop miRNA to treat pancreatic cancer

RNA delivery and therapeutics company SiSaf Ltd is to collaborate with the University of Leipzig in Germany to develop targeted micro interfering RNAs (miRNA) for the treatment of cancer, with an initial focus on pancreatic cancer. The collaboration will combine SiSaf’s work in RNA delivery using its Bio-Courier silicon-stabilized hybrid lipid nanoparticles (sshLNPs) and the […]

March 2, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 2 Mar 2023

Genomic medicine company Chroma raises $135M

Chroma Medicine, Inc., a genomic medicine company advancing single-dose epigenetic editing therapeutics, has completed a $135 million Series B financing. The funding was led by GV (Google Ventures), with participation from additional new investors ARCH Venture Partners, DCVC Bio, Mubadala Capital, Sixth Street, and all existing investors, including Alexandria Venture Investments, Atlas Venture, Casdin Capital, […]

March 2, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 2 Mar 2023

Ten new companies join BioInnovation Institute’s Venture Lab program

BioInnovation Institute (BII), an international non-profit foundation incubating and accelerating life science research, has added 10 new companies to its Venture Lab acceleration program for early-stage companies. The cohort is aligned with BioInnovation Institute’s focus on supporting innovative early-stage start-ups within human and planetary health. The 12-month program is designed to support start-up companies with […]

March 2, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 2 Mar 2023

New lipid nanoparticle CRISPR delivery system developed

Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein. Gene therapy is a potential mode of treatment for a wide variety of diseases caused by genetic mutations. While it has been an area of diverse and […]

March 2, 2023 - 3 minutesmins - By Jim Cornall

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

Opinion 28 Feb 2023

Life changing treatments for rare disease – a beacon of hope

By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […]

February 28, 2023 - 6 minutesmins - By External Contributor

Best in Biotech 28 Feb 2023

5 advancements in rare disease research over the past year

There are more than 7,000 rare diseases that affect hundreds of millions of people worldwide. As Rare Disease Day is observed on February 28, here are five recent advancements that have transformed research and boosted the scope for the development of novel therapies. FDA approves tissue-agnostic therapies for rare cancers Tissue-agnostic drug development has been […]

February 28, 2023 - 6 minutesmins - By Roohi Mariam Peter

Best in Biotech 28 Feb 2023

10 biotech companies making a difference in rare diseases

February 28 is Rare Disease Day, which looks to raise awareness of rare diseases and bring about change for patients and families living with them. The event also brings attention to the need for improved access to diagnosis, as well as the need to develop more treatments to tackle a broader range of diseases. There […]

February 28, 2023 - 11 minutesmins - By Willow Shah-Neville

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

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Partnership formed to develop miRNA to treat pancreatic cancer

Genomic medicine company Chroma raises $135M

Ten new companies join BioInnovation Institute’s Venture Lab program

New lipid nanoparticle CRISPR delivery system developed

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

Life changing treatments for rare disease – a beacon of hope

5 advancements in rare disease research over the past year

10 biotech companies making a difference in rare diseases

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

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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