Drug Discovery World

JUNE 25, 2024

Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, President and Chief Executive Officer, Sarepta. Diana Spencer, Senior Digital Content Editor, DDW The post FDA expands gene therapy use in Duchenne muscular dystrophy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).

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Drug Discovery World

FEBRUARY 20, 2024

Gilead Sciences has opened a new research site at the Business Park in Oxford, UK. Flavius Martin, Executive Vice President, Research, Gilead Sciences, said: “Our new Oxford site will enable us to continue our pivotal research in autoimmune conditions such as inflammatory bowel disease, rheumatoid arthritis and lupus.

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Drug Discovery World

FEBRUARY 8, 2024

In 2023, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. How is the titer determined?

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Drug Discovery World

JULY 29, 2024

Furthermore, when cell competition takes place between cancer cells, it can lead to some cancer cells developing resistance to chemotherapy or other targeted therapies. Diana Spencer, Senior Digital Content Editor, DDW The post Science reveals how cancer cells outsmart normal cells appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 4, 2023

AviadoBio, a gene therapy company developing medicines for neurodegenerative disorders, will establish laboratory and office presence at the Innovation Centre. Kadans and Canary Wharf Group are also progressing their joint venture to develop an GIA commercial health and life sciences building, the largest in Europe, on the 3.3-hectare

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Drug Discovery World

JANUARY 23, 2023

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

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Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. JMB: Since the early days of CAMP4, we have always followed the science.

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Drug Discovery World

JANUARY 31, 2024

Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs. Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor.

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Drug Discovery World

FEBRUARY 5, 2024

Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs. Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor.

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Drug Discovery World

OCTOBER 3, 2023

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Therapies 130

Therapies Bioinformatics Packaging Molecular Biology 130

Drug Discovery World

FEBRUARY 15, 2024

Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor.

Therapies 130

Therapies RNA Science Marketing 130

Drug Discovery World

FEBRUARY 22, 2024

Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs. Mark Treherne will talk about advanced therapies and the development of RNA gene therapy for aggressive cancers which is being carried out at Spliceor.

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Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

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Drug Discovery World

NOVEMBER 24, 2022

With European life sciences R&D investment at a 20-year low, biopharmaceutical associations and MEPs are calling on the EU to modernise its policies. DDW’s Diana Spencer asks what will be needed for Europe to compete in the growing global market for advanced therapies. . What will improve Europe’s attractiveness for life sciences?

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Drug Discovery World

MARCH 14, 2023

Life sciences membership organisation OBN (UK) will be holding in-person event BioTrinity 2023 in London on April 25-26. The conference will feature two tracks: ‘Scientific’, showcasing innovation across eight core therapy areas, and ‘Life Sciences Business & Investment’.

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Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. It is imperative for the industry to communicate the precise composition and attributes of these therapies to regulatory bodies.

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Drug Discovery World

AUGUST 4, 2023

CRADL’s first Seattle facility opened in 2021, in response to the city’s growing position as a key life sciences hub with significant start-up and entrepreneurial growth. In October, CRADL Philadelphia will open in uCity Square to support Philadelphia’s growth as a life sciences biohub.

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Drug Discovery World

JANUARY 17, 2024

ERS Genomics, a CRISPR/Cas9 licensing company, and StemSight, a biotechnology company developing stem cell based therapies for corneal blindness, have entered a non-exclusive CRISPR/Cas9 license agreement. StemSight is a preclinical biotechnology company developing off-the-shelf cell therapies for unmet medical needs in corneal blindness.

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Drug Discovery World

FEBRUARY 13, 2024

In this webinar, hosted by DDW and sponsored by Astrea Bioseparations, you will learn about how market growth can be sustained to maximise on opportunities in cell and gene therapy (CGT). Doug Danison will discuss transitioning innovative science into commercial success in the areas of CGTs.

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Drug Discovery World

AUGUST 4, 2023

Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world. Sven identified the two most prominent barriers as financing, and how to get ground-breaking therapies to the patients that most need them around the world.

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Drug Discovery World

MARCH 6, 2024

We are grateful for the support of a syndicate of leading life science investors and a team of industry veterans, including scientific co-founders Dr Howard Federoff and Dr Jeffrey Kordower, who see the promise in Kenai’s approach to treating central nervous system disorders,” said Nick Manusos, Chief Executive Officer of Kenai Therapeutics.

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Drug Discovery World

APRIL 23, 2024

Given the increased awareness of concussion and the associated long-term sequela from concussive injury it is imperative that we accelerate drug development efforts to find an effective therapy. Tremendous strides have been made with other diseases, yet there are still no FDA-approved drug therapies for TBI.

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Drug Discovery World

DECEMBER 12, 2023

Bristol-Myers Squibb has received manufacturing and marketing approval in Japan for Abecma (idecabtagene vicleucel) as a third-line therapy for relapsed or refractory multiple myeloma (RRMM). We remain committed to researching and developing innovative therapies to transform patient lives with serious diseases through science.”

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Drug Discovery World

MARCH 16, 2023

The cell and gene therapy (CGT) market is rapidly expanding, and projections suggest it will reach over $90B by 2023. Challenges of cell and gene therapy manufacturing Compared to traditional biologics, CGTs are living drugs, making their development and manufacturing substantially more complex.

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Drug Discovery World

JULY 5, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

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Drug Discovery World

JUNE 29, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Virus 130

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Drug Discovery World

JUNE 22, 2023

Join DDW for this exclusive free event , A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences. AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers.

Virus 130

Virus Therapies Bioinformatics Packaging 130

Science Daily: Pharmacology News

SEPTEMBER 6, 2024

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

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Drug Discovery World

MAY 22, 2023

Ryan Leahy , a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector. The fact remains that the science behind the drugs is sound, there is direct patient need, and the medical benefits are pertinent.

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Drug Discovery World

OCTOBER 12, 2023

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business. In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio.

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Drug Discovery World

APRIL 3, 2023

As life science operates globally, the vision for the business was always to bring aptamers as an antibody alternative to researchers worldwide. What advice would you offer to similar start-ups?    AT: Integral to Aptamer Group’s offering is an in-depth understanding of molecular science. To what do you attribute your success?

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Drug Discovery World

JUNE 12, 2023

It is the UK’s first specifically designed accessible laboratory for the cell and gene therapy industry. This investment in Scotland builds upon CGT Catapult’s existing work supporting emerging advanced therapy clusters in the UK, with its Manufacturing Innovation Centres in Stevenage, Hertfordshire and Braintree, Essex.

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Drug Discovery World

OCTOBER 25, 2023

Gamida Cell has revealed early data in 10 patients with CD20 positive non-Hodgkin lymphoma as part of an ongoing study of natural killer (NK) cell therapy candidate GDA-201. The post Natural killer cell therapy shows powerful anti-tumour activity appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JULY 17, 2023

The collaboration brings together IRBM’s capabilities in drug discovery and development, RCF’s medical research resources via the Tau Consortium, and the expertise of Weill Cornell Medicine neuroscientist Dr Li Gan to pursue innovative therapies for neurodegenerative disorders.

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Drug Discovery World

JUNE 19, 2023

Anoat Therapeutics’ goal is to bring a transformative and mutation-agnostic treatment option to CF patients for whom existing therapies are not effective – for example, patients not eligible or responsive to CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapies.

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