Medical Xpress

MARCH 27, 2023

Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases. The article is published in the journal Human Gene Therapy.

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Medical Xpress

MARCH 15, 2023

Scientists have developed a test that could greatly improve quality of life for infants with homocystinuria (HCU), a congenital disease that—if not treated early—causes serious complications. Research demonstrating the efficacy of this test was published today in Clinical Chemistry.

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STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

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Pharmaceutical Technology

MARCH 1, 2023

Tevard Biosciences and Vertex Pharmaceuticals have announced a four-year global research partnership to develop new tRNA-based therapies to treat Duchenne muscular dystrophy (DMD) patients. The deal offers access to the platforms of Tevard to discover and develop tRNA-based therapies.

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Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. . The origins . The current landscape .

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STAT News

APRIL 19, 2023

But after Cheung herself developed a genetic condition so rare it doesn’t have an official name, causing her to start losing her vision, HHMI decided to stop funding her. “I was asked to take a medical retirement,” Cheung told STAT. “I felt that I was being judged for having a disability.”

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Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge.

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Drug Discovery World

DECEMBER 15, 2022

Metrion will develop cell lines for both wild type and variant Kv3.1, The KCNC1 Foundation was created by the parents of a child diagnosed with KCNC1-related DEE to support research aimed at accelerating the development of a cure, as well as to raise awareness to identify and connect patients and provide support for families. .

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Pharmaceutical Technology

APRIL 6, 2023

RespireRx Pharmaceuticals has signed a material transfer agreement with University College London (UCL) to conduct a joint study to research the potential of AMPAkine to treat GRIA disorder. GRIA disorder is a family of rare genetic diseases.

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Scienmag

FEBRUARY 17, 2021

A leading cause of SCD in young athletes is arrhythmogenic cardiomyopathy (ACM), a genetic disease in which healthy heart muscle is replaced over time […]. Credit: Mark Bauer/FSU College of Medicine Nearly a half-million people a year die from sudden cardiac death (SCD) in the U.S.

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Scienmag

SEPTEMBER 13, 2021

Philadelphia, September 13, 2021—Researchers at Children’s Hospital of Philadelphia (CHOP) have demonstrated that an experimental device can improve blood sugar control in patients who developed diabetes after their pancreas was removed to treat their hyperinsulinism, a genetic disease in which the pancreas produces too much insulin.

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Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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Scienmag

APRIL 6, 2021

Researchers developed a new animal model to study a rare genetic disease that can lead to blindness at the age of 40-50.

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Scienmag

SEPTEMBER 29, 2021

JUPITER, FL – The American Chemical Society announced that its prestigious 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry honors a student from the Skaggs Graduate School of Chemical and Biological Sciences at Scripps Research and her faculty advisor, for their pioneering work developing RNA-targeting medications for genetic (..)

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Viruses and bacteria can be first modified to prevent them from causing infectious diseases and then implemented into human tissues as therapeutic gene vectors. PK Planning for Gene Therapy Development Programs. Formulation Considerations.

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Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

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Drug Discovery World

APRIL 17, 2024

DS: CAMP4 leverages research from co-founder Richard Young on how regulatory RNA (regRNA) molecules control the expression of genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What diseases are you currently targeting?

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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. AdBio Partners’ mission is to support founding scientists in the development of new therapeutic approaches,” said Alain Huriez, Managing Partner of AdBio Partners. “We

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Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘ parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’).

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Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity.

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Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

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Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis. In the FA study, teams worked with patients to identify key movement patterns and predict genetic markers of disease. Image shows: Luchen Li, wearing the sensor suit, and Professor Aldo Faisal.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

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Scienmag

JUNE 23, 2021

A Canadian research team has uncovered a new mechanism involved in Bourneville tuberous sclerosis (BTS), a genetic disease of childhood. The team hypothesizes that a mutation in the TSC1 gene causes neurodevelopmental disorders that develop in conjunction with the disease.

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Drug Discovery World

NOVEMBER 7, 2022

A new study led by Medical Research Council-funded researchers from UCL has found that tailoring the analysis of whole genome sequencing to individual patients could double the diagnostic rates of rare diseases. . It can also permit targeted screening of known disease complications and access to drug studies. Context

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Scienmag

SEPTEMBER 23, 2020

Progress in overcoming spinocerebellar ataxia, an intractable genetic disease Credit: Associate Professor Takahiro Seki A research team from Kumamoto University, Japan has developed an animal model that reproduces motor dysfunction and cerebellar neurodegeneration similar to that in spinocerebellar ataxia (SCA) by inhibiting chaperone-mediated autophagy (..)

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The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. The call will feature a presentation by KOL John Greinwald Jr., To register for the call, please click here.

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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. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’

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The Pharma Data

OCTOBER 21, 2020

NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass., 22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc.

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World of DTC Marketing

JANUARY 24, 2022

Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8

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Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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XTalks

MAY 17, 2023

The FNIH will focus on advancing clinical trials for the eight diseases through gene therapy research partnerships with the goal of accelerating the path of new therapies to clinical trials. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.

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Drug Discovery World

AUGUST 28, 2023

And we’ve shown that, at least in animal models of these diseases, our drug molecules work really well. DS: Your research has so far focused on Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS). What are the challenges of research in this area? DS: What role does genetics play in autophagy?

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Roots Analysis

FEBRUARY 26, 2024

Whether it was a common cold or a genetic disease, prayers and sacrifices to the gods were the only hope of a remedy at such times. It is possible that the legend of Big Foot may be based on an unfortunate person suffering from elephantiasis or gigantism or some other genetic predisposition that would have rendered him abnormal.

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Drug Discovery World

FEBRUARY 15, 2024

The RNA technology company conducted the work in collaboration with the lab of Professor De Geest at Ghent University, and describes how delivery of mRNA encoded antigens in LNPs comprising a novel class of ionisable lipids enhances the induction of antigen-specific immune responses while reducing undesired off-target expression in the liver, thereby (..)

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Drug Discovery World

JULY 11, 2022

However, the speed at which CGT products can impact healthcare depends on how the industry will navigate novel challenges and risks from the nascent field of CGT development. Complexities arise while trying to establish a standard, cost-efficient, and expert process for research and manufacturing. 1: TBRC Business Research PVTLTD.

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pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. Instead of traditional CRISPR- based gene therapy, the company uses homologous recombination as the basis of its genome editing platform, ‘GeneRide’.

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