Digital dilemma in UK’s life sciences job market
Pharmaceutical Technology
SEPTEMBER 8, 2023
In this issue: Digital jobs in UK life sciences, generative AI changes drug discovery, and how robotics is impacting gene therapy manufacturing.
Forge Biologics and Labcorp partner for gene therapies development
Pharmaceutical Technology
MAY 4, 2023
Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.
Gene Therapy 
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Forge Biologics joins BGTC for new AAV gene therapies development
Pharmaceutical Technology
MAY 24, 2023
Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Research consortium picks eight rare diseases to advance gene therapies
Pharmaceutical Technology
MAY 16, 2023
The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.
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In conversation with FNIH’s Dr. Courtney Silverthorn, Director of the AMP Bespoke Gene Therapy Consortium, About New Rare Disease Trial Portfolio – Xtalks Life Science Podcast Ep. 114
XTalks
JUNE 14, 2023
Courtney Silverthorn who is an Associate Vice President at the Foundation for the National Institutes of Health (FNIH) and the Director of the Accelerating Medicines Partnership (AMP) program including the Bespoke Gene Therapy Consortium. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.
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Life Science Trends for 2023 – Xtalks Life Science Podcast Ep. 91
XTalks
JANUARY 4, 2023
In this episode, Ayesha talked about some of the trends to look out for in the life sciences in 2023. Also hear about the issue of drug shortages and how pharma companies hope to tackle it in 2023. Read the full article here: 4 Life Sciences Trends for 2023.
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Life Science Trends for 2024 – Xtalks Life Science Podcast Ep. 143
XTalks
JANUARY 24, 2024
In this episode, Ayesha talked about some of the trends in the life sciences to watch out for in 2024. Hear about the latest technologies and approaches that will help drive innovations in drug discovery, clinical research and medical device design this year.
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GSK and Wave Life Sciences partner to develop oligonucleotide therapeutics
Pharmaceutical Technology
DECEMBER 14, 2022
GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. Wave will conduct all preclinical research for the programmes of both companies until investigational new drug (IND)-facilitating studies. . By Cytiva Thematic.
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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio
XTalks
MAY 17, 2023
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. As such, rare disease patients and their families often face little hope for effective treatments.
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Life sciences hub at Canary Wharf sees rapid growth
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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Guide to using AAV vectors in gene therapy
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.
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Can vertical AI advance cell and gene therapies?
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. Here’s what this means for pharmaceutical leaders and patients.
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Exclusive event on using adeno-associated virus (AAV) vectors in gene therapy
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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Learn how to use adeno-associated virus (AAV) vectors in gene therapy
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.
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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease
XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work? million after discounts.
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FDA Approval of First Rapid-Acting Oral Depression Drug + Approval of $2.8 Million Gene Therapy for Rare Blood Disorder – Xtalks Life Science Podcast Ep. 75
XTalks
AUGUST 31, 2022
Ayesha shared news about another significant FDA approval for a gene therapy to treat transfusion-dependent beta thalassemia. Bluebird bio was awarded the approval for its gene therapy Zynteglo (beti-cel), which is a one-time treatment for the rare blood disorder. Bluebird’s $2.8M
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A guide to using adeno-associated virus (AAV) vectors in gene therapy
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.
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4 Life Sciences Trends for 2023
XTalks
DECEMBER 29, 2022
The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.
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Charles River opens facilities within two US life science hubs
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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FDA Approves World’s Most Expensive Gene Therapy for Rare Disease + CDC Concerned About New COVID-19 Variant – Xtalks Life Science Podcast Ep. 79
XTalks
SEPTEMBER 29, 2022
The one-time administered gene therapy has also taken the title of being the world’s most expensive drug at $3 million. Hear more about the treatment and how the high prices of gene therapies can be alarming, but reimbursement programs are designed to pick up the costs. 7 that the CDC is closely watching.
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Guide to using AAV vectors in gene therapy
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.
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BioTrinity 2023 will combine life science innovation and business
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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Parkinson’s Disease: Link With Melanoma + Drug Development – Xtalks Life Science Podcast Ep. 6
XTalks
APRIL 14, 2021
In this episode of the Xtalks Life Science Podcast, Sarah revisits two Parkinson’s biotech companies — Prevail Therapeutics and Voyager Therapeutics — to see what progress they’ve made in their gene therapy clinical development programs since April 2019. Parkinson’s and Melanoma Share an Amyloid Link, Says New Research.
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Top Life Science News and Trends in 2023 at Xtalks
XTalks
DECEMBER 20, 2023
In 2023, there were significant advancements and notable trends in the life sciences. These advancements and trends addressed important industry challenges and offered opportunities to foster innovations and solutions in areas including drug development, healthcare and medical devices.
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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases
XTalks
APRIL 24, 2023
Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.
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First Topical Gene Therapy + Bausch + Lomb’s First Prescription Med for Dry Eye Ep. 112
XTalks
MAY 31, 2023
In this episode, Ayesha talked about the FDA approval of the first topical gene therapy for the treatment of a rare skin condition. Krystal Biotech’s Vyjuvek is a topical gene therapy that has been approved for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).
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Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod
XTalks
JUNE 30, 2023
It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).
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Can characterisation industrialise cell and gene therapy?
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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Accelerating gene therapy research with AAV detection kits
Drug Discovery World
DECEMBER 1, 2022
PerkinElmer has launched ready-to-use adeno-associated virus vectors ( AAV ) detection kits to support researchers working on gene therapies for a variety of serious diseases. . The post Accelerating gene therapy research with AAV detection kits appeared first on Drug Discovery World (DDW).
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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease
XTalks
MAY 26, 2023
Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).
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Can Europe become a major player in cell and gene therapy?
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. . Developing world class innovation hubs.
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Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy
XTalks
JUNE 28, 2023
Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). million price tag of Elevidys, a one-time gene therapy. Options for managing the symptoms of DMD have been limited.
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Cell and Gene Therapy Catapult opens new Scottish laboratories
Drug Discovery World
JUNE 12, 2023
CGT Catapult hopes this laboratory will help widen participation in the industry and increase opportunities for more scientists living with disabilities to take up a career in cell and gene therapies.
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BioMarin Gene Therapy Halted by FDA Over Safety Concerns
XTalks
SEPTEMBER 9, 2021
The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.
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Sarepta helps bankroll new gene therapy startup AavantiBio
pharmaphorum
OCTOBER 22, 2020
Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. Unlike a one-shot gene therapy, omaveloxolone would require continuous dosing to maintain its effects.
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Rare Disease Day 2024: Highlighting Patient Stories and Advancements in Rare Disease Research – Xtalks Life Science Podcast Ep. 148
XTalks
FEBRUARY 28, 2024
Hear more about new rare disease drugs approved by the FDA in the past couple of years. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.
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No sign of stopping: The rise of cell and gene therapy
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 cost 2022 has not been shy of approvals – Uniqure and CSL’s Haemophilia B drug even nabbed the title of “Most Expensive Drug” at $3.5
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Interview with Dr. Kristin Yarema, President of Cell Therapy at Poseida Therapeutics – Xtalks Life Science Podcast Ep. 129
XTalks
SEPTEMBER 27, 2023
Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. Yarema sharing her thoughts about the promise of allogeneic CAR-T cell therapy for patients.
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Understanding the future roadmaps of cell and gene therapy
Drug Discovery World
MAY 23, 2023
DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.
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Cell and gene therapies: Opportunities for innovation
Drug Discovery World
SEPTEMBER 6, 2022
In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.
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Transcending expectations for cell & gene therapy development
Drug Discovery World
JULY 11, 2022
David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.
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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel
XTalks
NOVEMBER 3, 2023
After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. I think in this case, there is a huge unmet need.”
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This week in drug discovery (3-7 July)
Drug Discovery World
JULY 14, 2023
awarded to progress ovarian cancer cell therapy NK:IO has been awarded £1.6m ($2.1m) in grant funding from Innovate UK’s New Cancer Therapeutics programme. £1m The post This week in drug discovery (3-7 July) appeared first on Drug Discovery World (DDW).
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The challenges and trends of cell & gene therapies
Drug Discovery World
SEPTEMBER 6, 2022
DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. Cancer is king .
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