STAT News

SEPTEMBER 12, 2022

In the life sciences industry, where company names derived from “biology” or “genetics” are a dime a dozen, it helps to have a brand that stands out. The company is developing treatments for diseases caused by broken mitochondria — the main energy source for human cells.

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Pharmaceutical Technology

MAY 24, 2023

The AMP programme is a public-private partnership between a number of biopharmaceutical and life sciences companies, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH), along with non-profit and other organisations.

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Scienmag

JUNE 7, 2021

Credit: Autobahn Labs Cold Spring Harbor Laboratory (CSHL) is collaborating with Autobahn Labs, a new life sciences incubator, to catalyze the Laboratory’s early-stage discovery programs into spin-out companies that commercialize transformational new therapies. Autobahn will provide […].

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Drug Discovery World

JUNE 27, 2024

The funding was co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, JP Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight.

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XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). Nobelpharma America, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, has launched an educational website called Face Forward.

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World of DTC Marketing

JANUARY 24, 2022

A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3, The panel was hosted by the life sciences venture capital coalition Incubate and held at the National Press Club. Only about $2.2 3, known as the Lower Drug Costs Now Act.

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Drug Discovery World

JUNE 19, 2023

Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs. Anoat will use the funds to conduct additional in vivo and in vitro pharmacology assessments and select a lead candidate.

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XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency.

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Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases.

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Drug Discovery World

MAY 11, 2023

Fundraising and investment are a core pillar of not just BioTrinity events but of the drug discovery industry at large, so events such as Tuesday’s keynote, Investment Trends for the Life Sciences Industry, are critical for members of the industry.

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XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases. HS: Absolutely.

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XTalks

MARCH 21, 2024

MLD is a rare genetic disease affecting the brain and nervous system caused by a deficiency of the enzyme arylsulfatase A (ARSA), which results in the accumulation of sulfatides (fatty substances) in cells. The transaction has proven fruitful with the milestone US approval of the gene therapy just a few months later.

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Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. It is a chimeric endonuclease, which consists of a custom-designed ZF DNA-binding domain and the non-specific nuclease domain from the FokI restriction enzyme. They are the smallest type of programmable nuclease.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

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XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company.

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pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. The post Rare diseases, repurposing and the role of AI appeared first on.

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XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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XTalks

FEBRUARY 27, 2023

Related: Interview with the Leaders at Amylyx, Maker of New FDA Approved ALS Drug Relyvrio – Xtalks Life Science Podcast Ep. They also cannot be pregnant at the onset of treatment, as Lamzede can harm the embryo or fetus.

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XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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Drug Discovery World

NOVEMBER 17, 2022

Researchers working across the entire life sciences community, including academia, charities, industry and the NHS will be able to apply to use this information to discover more effective ways to prevent, detect and treat diseases and conditions such as cancer, Alzheimer’s, heart disease, arthritis, diabetes and stroke. .

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

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XTalks

AUGUST 29, 2022

The team’s approach to cellular engineering with non-viral CRISPR technology could be used to replace the genes IL2RA and CTLA4 entirely, which are associated with rare genetic diseases. Scientists have previously shown that they could replace small sections of the IL2RA gene where mutations occur in patients.

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XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. They found that as cells transitioned from stem cells to differentiated adult cells, which involves significant epigenetic changes, the methylation marks made by CRISPRoff were inherited in a significant fraction of cells.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

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Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. “They called him ‘The Boy.’ Who wasn’t supposed to sit up. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S.

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pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. Results from a phase 3 trial of blarcamesine are due next year.

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XTalks

SEPTEMBER 22, 2022

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The novel, selective monoclonal antibody spesolimab behind Spevigo reduces flares in individuals by inhibiting interleukin-36 (IL-36) signalling.

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XTalks

AUGUST 16, 2021

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency. According to the CDC, approximately 2.7

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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XTalks

AUGUST 19, 2020

Cystic fibrosis (CF) patients must keep a six feet distance from others at all times to keep germs away that could exacerbate their disease. CF is a progressive, genetic disease that causes persistent lung infection and could limit a person’s ability to breathe over time.

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XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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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. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. Conclusion. About the author.

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XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”.

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