genetic disorders Archives

Best in Biotech 29 Apr 2025

Six biotech companies leading the charge in hemophilia treatment

Discover six hemophilia companies developing new treatments for the genetic bleeding disorder, from gene therapies to RNAi therapies.

April 29, 2025 - 10 minutesmins - By Willow Shah-Neville

In Depth 7 Apr 2025

Will tRNA therapy be the next big thing in genetic disease treatment?

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

April 7, 2025 - 12 minutesmins - By Willow Shah-Neville

Best in Biotech 20 Feb 2025

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Discover three promising approaches being investigated to treat Hunter syndrome, a rare genetic disorder affecting children.

February 20, 2025 - 10 minutesmins - By Willow Shah-Neville

In Depth 21 Nov 2024

Huntington’s disease: a therapeutic field on a bumpy ride

For years, biotechs have been trying to develop medicines to cure the disease but much to no avail. Now, as clinical studies advance, there is hope.

November 21, 2024 - 9 minutesmins - By Roohi Mariam Peter

Best in Biotech 21 Nov 2024

Eight areas where gene therapy shines

Delve into the numerous applications of gene therapy, from cancer to hematological disorders and respiratory diseases.

November 21, 2024 - 12 minutesmins - By Jules Adam

News and Trends 23 Oct 2024

RNA editing in humans: the race begins with first successful trial

Wave Life Sciences achieves RNA editing in the clinic, crossing a major milestone in the space.

October 23, 2024 - 6 minutesmins - By Roohi Mariam Peter

Best in Biotech 3 Oct 2024

Six biotechs driving progress in Duchenne muscular dystrophy

In this article, we take a look at six clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space.

October 3, 2024 - 8 minutesmins - By Roohi Mariam Peter

News and Trends 27 Sep 2024

Aptadir Therapeutics launches to develop new class of RNA inhibitors against cancer and genetic disorders

Italian startup Aptadir Therapeutics has launched with $1.6 million to bring a new class of drugs to the clinic and a technology to be reckoned with.

September 27, 2024 - 6 minutesmins - By Roohi Mariam Peter

In Depth 10 Jul 2024

Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder

As treatments for skeletal dysplasia are intended to alleviate painful symptoms, two drugs are battling out in the clinic to prove their effectiveness.

July 10, 2024 - 7 minutesmins - By Roohi Mariam Peter

In Depth 6 Jun 2024

CRISPR: A promising path to treating blindness

Rigorous research, which has been going on for more than a decade now, suggests that CRISPR could treat genetic diseases including blindness.

June 6, 2024 - 7 minutesmins - By Roohi Mariam Peter

Best in Biotech 6 Jun 2024

Eight biotech companies advancing the field of siRNA

Explore the growing sector of siRNA and discover eight biotech companies spearheading this innovative field.

June 6, 2024 - 11 minutesmins - By Jules Adam

In Depth 23 May 2024

Is prime editing ready for prime time?

Prime editing, a more advanced version of CRISPR/Cas9 technology, has been greenlit by the FDA to begin the first-ever clinical trial for this technology.

May 23, 2024 - 7 minutesmins - By Roohi Mariam Peter

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

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genetic disorders

Six biotech companies leading the charge in hemophilia treatment

Will tRNA therapy be the next big thing in genetic disease treatment?

3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder

Huntington’s disease: a therapeutic field on a bumpy ride

Eight areas where gene therapy shines

RNA editing in humans: the race begins with first successful trial

Six biotechs driving progress in Duchenne muscular dystrophy

Aptadir Therapeutics launches to develop new class of RNA inhibitors against cancer and genetic disorders

Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder

CRISPR: A promising path to treating blindness

Eight biotech companies advancing the field of siRNA

Is prime editing ready for prime time?

Subscribe to our newsletter to get the latest biotech news!

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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