The Pharma Data

DECEMBER 17, 2020

Most recently, Cadavid served as senior vice president and Head of Clinical Development at Fulcrum Therapeutics where he led the development of multiple small molecules for the treatment of genetically defined rare diseases. Smith joined SpringWorks at the company’s inception in 2017 and was a founding member of the management team.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. In Dravet Syndrome, the approach is to target the SCN1a gene, which has mutations that cause the condition, explains Ferraro.

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XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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pharmaphorum

JANUARY 29, 2021

Since Macrae joined the company just four years ago, Sangamo has more than tripled its staff and raised $1.6 It has also built its own manufacturing site and launched partnerships with six big pharma companies. And ultimately we will be able to add genetic influences to diseases that don’t have a genetic cause.

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies.

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World of DTC Marketing

JANUARY 31, 2021

The venture capitalists funding them are looking for a big payday when a pharma company acquires them. I have been recruited by these companies who promise huge payouts when they are acquired if their drug shows promise in clinical trials. In pharma R&D returns have declined to 1.8 percent—a slight decrease of 0.1

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Pharmaceutical Technology

MARCH 20, 2023

Pharma companies and world health stand to benefit from new technologies, but it’s a tough time to be an investor – and an even tougher one for firms seeking investment. Research – and money – going into even more advanced gene therapies is ramping up. Further rate hikes could be on the way this year. Source: GlobalData.

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Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

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Delveinsight

AUGUST 4, 2021

Similarly, genetics, immune response, and environmental factors also influence the occurrence of Neurological Conditions. With the advancement in technology, today diagnostics testing such as neurological examination, brain scans, genetic screening, and other tests are available to examine the occurrence of Neurological Conditions.

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XTalks

DECEMBER 20, 2023

Check out this article to learn about the biotech companies that went public this year. The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy.

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pharmaphorum

SEPTEMBER 1, 2020

This knowledge of genetics and the underlying cause of many diseases, including cancer, has resulted in powerful new drugs. In cancer, the pharma industry has begun producing immunotherapies, which do not act directly against the disease but instead enlist the immune system to fight against malignant tissues.

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The Pharma Data

DECEMBER 30, 2020

It is a strong potential player in the gene therapy space. Its lead candidate is AMT-061, an AAV5-based gene therapy being evaluated for severe and moderately severe hemophilia B in the Phase III HOPE-B study. billion, Regenxbio is also a gene therapy company. UniQure has a market cap of $2.1

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XTalks

JANUARY 6, 2025

Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future. Cell and Gene Therapies Cell and gene therapies (C&GT) remain at the forefront of healthcare innovation, representing one of the fastest-growing therapeutic areas. billion in 2021, is projected to reach $42.56

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XTalks

FEBRUARY 11, 2025

Pediatric cancer care has traditionally relied on chemotherapy, radiation and surgery, but emerging therapies are expanding possibilities. While obstacles such as delivery barriers, immune responses and regulatory approvals remain, clinical trials are refining these approaches, bringing hope for more effective and less toxic therapies.

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